Latest news with #CTX310
Business Upturn
19-05-2025
- Business
- Business Upturn
CRISPR Therapeutics and Sirius Therapeutics Announce Multi-Target Collaboration to Develop Novel siRNA Therapies
-Collaboration brings together complementary capabilities to co-develop and co-commercialize SRSD107, a next generation, long-acting Factor XI (FXI) small interfering RNA (siRNA) for the treatment of thromboembolic disorders- -SRSD107 demonstrated peak reductions in FXI activity >93% and increases in activated partial thromboplastin time (aPTT) >2x with maintained efficacy up to 6 months post-dosing in a Phase 1 clinical trial- -Under the agreement, CRISPR Therapeutics will make an upfront payment of $25 million in cash and $70 million in equity to Sirius Therapeutics; CRISPR Therapeutics also has rights to exclusively license up to two additional siRNA programs- -Expands CRISPR's therapeutic toolkit to develop a broader range of transformative gene-based medicines in addition to the gene-editing programs in the clinic- ZUG, Switzerland and BOSTON and SAN DIEGO and SHANGHAI, May 19, 2025 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Sirius Therapeutics, a clinical stage biotech company developing innovative small interfering RNA (siRNA) therapies for global markets, today announced a strategic partnership to develop and commercialize siRNA therapies. 'We are excited to partner with Sirius, and broaden our cardiovascular medicine portfolio, on the heels of promising top-line data that we recently shared for CTX310, which targets ANGPTL3,' said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. 'Coagulation Factor XI represents an innovative and highly compelling target for treating thrombotic diseases that affect millions worldwide. SRSD107, which targets Factor XI, has the potential to be a best-in-class therapy, offering infrequent dosing and improved patient outcomes. Sirius' siRNA platform complements our existing capabilities and expands our therapeutic toolkit, enabling us to develop a broader range of transformative gene-based medicines.' 'We are pleased to collaborate with CRISPR Therapeutics, a recognized leader in the development of gene-based medicines,' said Qunsheng Ji, MD, Ph.D. Chief Executive Officer of Sirius Therapeutics. 'Thrombotic diseases represent a significant unmet need, and our promising Phase 1 data highlights the potential of SRSD107 as a best-in-class Factor XI-targeted therapy. Sirius is committed to addressing the needs of these patients, as we work with CRISPR Therapeutics to advance novel siRNA therapies globally.' 'There is a large population of patients who are at risk for potentially life-threatening thromboembolic events due to underlying co-morbid diseases such as malignancy, cardiovascular disease, and hyper-coagulability. A significant percentage of these patients are inadequately treated due to concerns for bleeding risk, or challenges with compliance,' said Christian T. Ruff, M.D., M.P.H., senior investigator of TIMI Group, director General Cardiology, Brigham and Women's Hospital, and associate professor, Harvard Medical School. 'SRSD107 offers the potential for a therapy with lower bleeding risk, infrequent dosing for better compliance, without concerns for renal clearance or drug interactions, and reversibility to further mitigate bleeding risks that could be differentiated from currently available therapies and other Factor XI modalities.' SRSD107 is a next generation, long-acting siRNA designed to selectively inhibit Factor XI (FXI), a key driver of pathological thrombosis with minimal impact on normal hemostasis. By targeting FXI, SRSD107 aims to reduce thrombotic events while minimizing the risk of bleeding – representing a differentiated approach compared to Factor Xa inhibitors. In addition, SRSD107 may offer the potential for reversibility not observed with other anti-Factor XI modalities. The addressable population includes patients with atrial fibrillation, venous thromboembolism (VTE), cancer-associated thrombosis, chronic Coronary Artery Disease (CAD), chronic Peripheral Vascular Disease (PVD), end-stage renal disease requiring hemodialysis, and patients undergoing major orthopedic surgery, where bleeding risk limits existing therapies. The clinical program for SRSD107 includes two promising Phase 1 clinical trials, where single doses of SRSD107 were found to be safe and well tolerated. In addition, SRSD107 demonstrated robust pharmacodynamic effects, including reductions of over 93% in FXI levels and FXI activity (FXIa), along with more than a twofold increase in activated partial thromboplastin time (aPTT) relative to baseline. These effects were sustained, with responses maintained for up to 6 months post-dosing. SRSD107 has the potential to be a best-in-class FXI inhibitor, showing deep reductions in FXI via semi-annual subcutaneous injection. Results from the Phase 1 trials were presented at both the 2025 Annual Scientific Sessions of the American College of Cardiology and the 2024 Annual Meeting of the American Society of Hematology. Figure 1. SRSD107 Phase 1 Clinical Results: Sustained, dose-dependent pharmacodynamic response to therapy A Phase 2 clinical trial of SRSD107 is being initiated to evaluate its safety and efficacy for the prevention of VTE in patients undergoing total knee arthroplasty. The trial aims to confirm the anticoagulant benefits of SRSD107 and to inform dose selection for future pivotal trials. Collaboration Details Under the terms of the agreement, CRISPR Therapeutics will make an upfront payment of $25 million in cash and $70 million in equity to Sirius Therapeutics. The companies will jointly develop SRSD107 under a 50-50 cost and profit-sharing structure. CRISPR Therapeutics will lead commercialization in the U.S., while Sirius will be responsible for commercialization in Greater China. Additionally, CRISPR Therapeutics will have the option to nominate up to two siRNA targets for research and development. For each target, CRISPR Therapeutics will fund research and retain opt-in rights to lead clinical development and commercialization. Sirius will be eligible to receive milestone payments, as well as tiered royalties ranging from high single to low-double digits. About CRISPR Therapeutics Since its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic approval of the first-ever CRISPR-based therapy. The Company has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. In 2018, CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic to investigate the treatment of sickle cell disease and transfusion-dependent beta thalassemia. Beginning in late 2023, CASGEVY® (exagamglogene autotemcel [exa-cel]) was approved in several countries to treat eligible patients with either of these conditions. The Nobel Prize-winning CRISPR technology has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit CRISPR THERAPEUTICS® standard character mark and design logo, CTX310™ and CTX320™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. CRISPR Therapeutics Forward-Looking Statement Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements made by Drs. Kulkarni, Ji and Ruff in this press release, as well as regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, expectations regarding data, safety and efficacy generally; (ii) data included in this press release, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; (iii) the status and clinical progress of the SRSD107 clinical program and development timelines for such program; (iv) CRISPR Therapeutics strategy and goals; (v) the future activities of the parties pursuant to the collaboration and the expected benefits of CRISPR Therapeutics' collaboration with Sirius Therapeutics; and (vi) the therapeutic value, development, and commercial potential of gene editing and delivery technologies and therapies, including CRISPR/Cas9. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading 'Risk Factors' in its most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. This press release discusses investigational therapies and is not intended to convey conclusions about efficacy or safety as to those investigational therapies or uses of such investigational therapies. There is no guarantee that any investigational therapy will successfully complete clinical development or gain approval from applicable regulatory authorities. About Thromboembolic Disorders Thrombosis, or blood clot formation, is the common underlying mechanism of most cases of myocardial infarction, ischemic stroke, and venous thromboembolism. According to a trial in The Lancet1 of regional and global mortality rates, thromboembolic disorders are estimated to cause as many as 1 in 4 deaths worldwide. About SRSD107 SRSD107 is a novel double-stranded small interfering ribonucleic acid (siRNA). SRSD107 specifically targets the human coagulation factor XI (FXI) mRNA and inhibits FXI protein expression, thereby blocking the intrinsic coagulation pathway and promoting anticoagulant/anti-thrombotic effects. SRSD107 has been engineered for the potential to enable twice-a-year dosing. About Sirius Therapeutics Sirius is a clinical stage biotech company developing innovative siRNA therapies for global markets. We are dedicated to discovering and developing new treatment options for cardiovascular and cerebrovascular disease and translating siRNA technology into transformative medicine for chronic disease patients. Sirius's most advanced products are SRSD107 for the treatment of thromboembolic disorders, SRSD216 for the treatment of hyperlipoproteinemia, and SRSD101 for the treatment of dyslipidemia. Founded in 2021 by a world-class leadership team and investors, Sirius has established an innovation center in the United States and translational medicine center in China. Sirius has raised nearly US$150 million funding to date from OrbiMed, Creacion Ventures, Hankang Capital, Delos Capital, and BioTrack Capital. Learn more at References: 1. Lozano R, Naghavi M, Foreman K, et al. Global and regional mortality from 235 causes of death for 20 age groups in 1990 and 2010: a systematic analysis for the Global Burden of Disease Study 2010. Lancet. 2012;380, 2095-1128. Investor Contact:+1-617-307-7503 [email protected]
Yahoo
19-05-2025
- Business
- Yahoo
CRISPR Therapeutics and Sirius Therapeutics Announce Multi-Target Collaboration to Develop Novel siRNA Therapies
-Collaboration brings together complementary capabilities to co-develop and co-commercialize SRSD107, a next generation, long-acting Factor XI (FXI) small interfering RNA (siRNA) for the treatment of thromboembolic disorders- -SRSD107 demonstrated peak reductions in FXI activity >93% and increases in activated partial thromboplastin time (aPTT) >2x with maintained efficacy up to 6 months post-dosing in a Phase 1 clinical trial- -Under the agreement, CRISPR Therapeutics will make an upfront payment of $25 million in cash and $70 million in equity to Sirius Therapeutics; CRISPR Therapeutics also has rights to exclusively license up to two additional siRNA programs- -Expands CRISPR's therapeutic toolkit to develop a broader range of transformative gene-based medicines in addition to the gene-editing programs in the clinic- ZUG, Switzerland and BOSTON and SAN DIEGO and SHANGHAI, May 19, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Sirius Therapeutics, a clinical stage biotech company developing innovative small interfering RNA (siRNA) therapies for global markets, today announced a strategic partnership to develop and commercialize siRNA therapies. 'We are excited to partner with Sirius, and broaden our cardiovascular medicine portfolio, on the heels of promising top-line data that we recently shared for CTX310, which targets ANGPTL3,' said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. 'Coagulation Factor XI represents an innovative and highly compelling target for treating thrombotic diseases that affect millions worldwide. SRSD107, which targets Factor XI, has the potential to be a best-in-class therapy, offering infrequent dosing and improved patient outcomes. Sirius' siRNA platform complements our existing capabilities and expands our therapeutic toolkit, enabling us to develop a broader range of transformative gene-based medicines.' 'We are pleased to collaborate with CRISPR Therapeutics, a recognized leader in the development of gene-based medicines,' said Qunsheng Ji, MD, Ph.D. Chief Executive Officer of Sirius Therapeutics. 'Thrombotic diseases represent a significant unmet need, and our promising Phase 1 data highlights the potential of SRSD107 as a best-in-class Factor XI-targeted therapy. Sirius is committed to addressing the needs of these patients, as we work with CRISPR Therapeutics to advance novel siRNA therapies globally.' 'There is a large population of patients who are at risk for potentially life-threatening thromboembolic events due to underlying co-morbid diseases such as malignancy, cardiovascular disease, and hyper-coagulability. A significant percentage of these patients are inadequately treated due to concerns for bleeding risk, or challenges with compliance,' said Christian T. Ruff, M.D., M.P.H., senior investigator of TIMI Group, director General Cardiology, Brigham and Women's Hospital, and associate professor, Harvard Medical School. 'SRSD107 offers the potential for a therapy with lower bleeding risk, infrequent dosing for better compliance, without concerns for renal clearance or drug interactions, and reversibility to further mitigate bleeding risks that could be differentiated from currently available therapies and other Factor XI modalities.' SRSD107 is a next generation, long-acting siRNA designed to selectively inhibit Factor XI (FXI), a key driver of pathological thrombosis with minimal impact on normal hemostasis. By targeting FXI, SRSD107 aims to reduce thrombotic events while minimizing the risk of bleeding – representing a differentiated approach compared to Factor Xa inhibitors. In addition, SRSD107 may offer the potential for reversibility not observed with other anti-Factor XI modalities. The addressable population includes patients with atrial fibrillation, venous thromboembolism (VTE), cancer-associated thrombosis, chronic Coronary Artery Disease (CAD), chronic Peripheral Vascular Disease (PVD), end-stage renal disease requiring hemodialysis, and patients undergoing major orthopedic surgery, where bleeding risk limits existing therapies. The clinical program for SRSD107 includes two promising Phase 1 clinical trials, where single doses of SRSD107 were found to be safe and well tolerated. In addition, SRSD107 demonstrated robust pharmacodynamic effects, including reductions of over 93% in FXI levels and FXI activity (FXIa), along with more than a twofold increase in activated partial thromboplastin time (aPTT) relative to baseline. These effects were sustained, with responses maintained for up to 6 months post-dosing. SRSD107 has the potential to be a best-in-class FXI inhibitor, showing deep reductions in FXI via semi-annual subcutaneous injection. Results from the Phase 1 trials were presented at both the 2025 Annual Scientific Sessions of the American College of Cardiology and the 2024 Annual Meeting of the American Society of Hematology. Figure 1. SRSD107 Phase 1 Clinical Results: Sustained, dose-dependent pharmacodynamic response to therapyA Phase 2 clinical trial of SRSD107 is being initiated to evaluate its safety and efficacy for the prevention of VTE in patients undergoing total knee arthroplasty. The trial aims to confirm the anticoagulant benefits of SRSD107 and to inform dose selection for future pivotal trials. Collaboration DetailsUnder the terms of the agreement, CRISPR Therapeutics will make an upfront payment of $25 million in cash and $70 million in equity to Sirius Therapeutics. The companies will jointly develop SRSD107 under a 50-50 cost and profit-sharing structure. CRISPR Therapeutics will lead commercialization in the U.S., while Sirius will be responsible for commercialization in Greater China. Additionally, CRISPR Therapeutics will have the option to nominate up to two siRNA targets for research and development. For each target, CRISPR Therapeutics will fund research and retain opt-in rights to lead clinical development and commercialization. Sirius will be eligible to receive milestone payments, as well as tiered royalties ranging from high single to low-double digits. About CRISPR TherapeuticsSince its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic approval of the first-ever CRISPR-based therapy. The Company has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. In 2018, CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic to investigate the treatment of sickle cell disease and transfusion-dependent beta thalassemia. Beginning in late 2023, CASGEVY® (exagamglogene autotemcel [exa-cel]) was approved in several countries to treat eligible patients with either of these conditions. The Nobel Prize-winning CRISPR technology has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit CRISPR THERAPEUTICS® standard character mark and design logo, CTX310™ and CTX320™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. CRISPR Therapeutics Forward-Looking StatementStatements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements made by Drs. Kulkarni, Ji and Ruff in this press release, as well as regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, expectations regarding data, safety and efficacy generally; (ii) data included in this press release, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; (iii) the status and clinical progress of the SRSD107 clinical program and development timelines for such program; (iv) CRISPR Therapeutics strategy and goals; (v) the future activities of the parties pursuant to the collaboration and the expected benefits of CRISPR Therapeutics' collaboration with Sirius Therapeutics; and (vi) the therapeutic value, development, and commercial potential of gene editing and delivery technologies and therapies, including CRISPR/Cas9. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading 'Risk Factors' in its most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. This press release discusses investigational therapies and is not intended to convey conclusions about efficacy or safety as to those investigational therapies or uses of such investigational therapies. There is no guarantee that any investigational therapy will successfully complete clinical development or gain approval from applicable regulatory authorities. About Thromboembolic DisordersThrombosis, or blood clot formation, is the common underlying mechanism of most cases of myocardial infarction, ischemic stroke, and venous thromboembolism. According to a trial in The Lancet1 of regional and global mortality rates, thromboembolic disorders are estimated to cause as many as 1 in 4 deaths worldwide. About SRSD107SRSD107 is a novel double-stranded small interfering ribonucleic acid (siRNA). SRSD107 specifically targets the human coagulation factor XI (FXI) mRNA and inhibits FXI protein expression, thereby blocking the intrinsic coagulation pathway and promoting anticoagulant/anti-thrombotic effects. SRSD107 has been engineered for the potential to enable twice-a-year dosing. About Sirius TherapeuticsSirius is a clinical stage biotech company developing innovative siRNA therapies for global markets. We are dedicated to discovering and developing new treatment options for cardiovascular and cerebrovascular disease and translating siRNA technology into transformative medicine for chronic disease patients. Sirius's most advanced products are SRSD107 for the treatment of thromboembolic disorders, SRSD216 for the treatment of hyperlipoproteinemia, and SRSD101 for the treatment of dyslipidemia. Founded in 2021 by a world-class leadership team and investors, Sirius has established an innovation center in the United States and translational medicine center in China. Sirius has raised nearly US$150 million funding to date from OrbiMed, Creacion Ventures, Hankang Capital, Delos Capital, and BioTrack Capital. Learn more at References:1. Lozano R, Naghavi M, Foreman K, et al. Global and regional mortality from 235 causes of death for 20 age groups in 1990 and 2010: a systematic analysis for the Global Burden of Disease Study 2010. Lancet. 2012;380, 2095-1128. Investor Contact:+1-617-307-7503ir@ Media Contact:+1-617-315-4493media@ A photo accompanying this announcement is available at in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
19-05-2025
- Business
- Yahoo
CRISPR Therapeutics and Sirius Therapeutics Announce Multi-Target Collaboration to Develop Novel siRNA Therapies
-Collaboration brings together complementary capabilities to co-develop and co-commercialize SRSD107, a next generation, long-acting Factor XI (FXI) small interfering RNA (siRNA) for the treatment of thromboembolic disorders- -SRSD107 demonstrated peak reductions in FXI activity >93% and increases in activated partial thromboplastin time (aPTT) >2x with maintained efficacy up to 6 months post-dosing in a Phase 1 clinical trial- -Under the agreement, CRISPR Therapeutics will make an upfront payment of $25 million in cash and $70 million in equity to Sirius Therapeutics; CRISPR Therapeutics also has rights to exclusively license up to two additional siRNA programs- -Expands CRISPR's therapeutic toolkit to develop a broader range of transformative gene-based medicines in addition to the gene-editing programs in the clinic- ZUG, Switzerland and BOSTON and SAN DIEGO and SHANGHAI, May 19, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Sirius Therapeutics, a clinical stage biotech company developing innovative small interfering RNA (siRNA) therapies for global markets, today announced a strategic partnership to develop and commercialize siRNA therapies. 'We are excited to partner with Sirius, and broaden our cardiovascular medicine portfolio, on the heels of promising top-line data that we recently shared for CTX310, which targets ANGPTL3,' said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. 'Coagulation Factor XI represents an innovative and highly compelling target for treating thrombotic diseases that affect millions worldwide. SRSD107, which targets Factor XI, has the potential to be a best-in-class therapy, offering infrequent dosing and improved patient outcomes. Sirius' siRNA platform complements our existing capabilities and expands our therapeutic toolkit, enabling us to develop a broader range of transformative gene-based medicines.' 'We are pleased to collaborate with CRISPR Therapeutics, a recognized leader in the development of gene-based medicines,' said Qunsheng Ji, MD, Ph.D. Chief Executive Officer of Sirius Therapeutics. 'Thrombotic diseases represent a significant unmet need, and our promising Phase 1 data highlights the potential of SRSD107 as a best-in-class Factor XI-targeted therapy. Sirius is committed to addressing the needs of these patients, as we work with CRISPR Therapeutics to advance novel siRNA therapies globally.' 'There is a large population of patients who are at risk for potentially life-threatening thromboembolic events due to underlying co-morbid diseases such as malignancy, cardiovascular disease, and hyper-coagulability. A significant percentage of these patients are inadequately treated due to concerns for bleeding risk, or challenges with compliance,' said Christian T. Ruff, M.D., M.P.H., senior investigator of TIMI Group, director General Cardiology, Brigham and Women's Hospital, and associate professor, Harvard Medical School. 'SRSD107 offers the potential for a therapy with lower bleeding risk, infrequent dosing for better compliance, without concerns for renal clearance or drug interactions, and reversibility to further mitigate bleeding risks that could be differentiated from currently available therapies and other Factor XI modalities.' SRSD107 is a next generation, long-acting siRNA designed to selectively inhibit Factor XI (FXI), a key driver of pathological thrombosis with minimal impact on normal hemostasis. By targeting FXI, SRSD107 aims to reduce thrombotic events while minimizing the risk of bleeding – representing a differentiated approach compared to Factor Xa inhibitors. In addition, SRSD107 may offer the potential for reversibility not observed with other anti-Factor XI modalities. The addressable population includes patients with atrial fibrillation, venous thromboembolism (VTE), cancer-associated thrombosis, chronic Coronary Artery Disease (CAD), chronic Peripheral Vascular Disease (PVD), end-stage renal disease requiring hemodialysis, and patients undergoing major orthopedic surgery, where bleeding risk limits existing therapies. The clinical program for SRSD107 includes two promising Phase 1 clinical trials, where single doses of SRSD107 were found to be safe and well tolerated. In addition, SRSD107 demonstrated robust pharmacodynamic effects, including reductions of over 93% in FXI levels and FXI activity (FXIa), along with more than a twofold increase in activated partial thromboplastin time (aPTT) relative to baseline. These effects were sustained, with responses maintained for up to 6 months post-dosing. SRSD107 has the potential to be a best-in-class FXI inhibitor, showing deep reductions in FXI via semi-annual subcutaneous injection. Results from the Phase 1 trials were presented at both the 2025 Annual Scientific Sessions of the American College of Cardiology and the 2024 Annual Meeting of the American Society of Hematology. Figure 1. SRSD107 Phase 1 Clinical Results: Sustained, dose-dependent pharmacodynamic response to therapyA Phase 2 clinical trial of SRSD107 is being initiated to evaluate its safety and efficacy for the prevention of VTE in patients undergoing total knee arthroplasty. The trial aims to confirm the anticoagulant benefits of SRSD107 and to inform dose selection for future pivotal trials. Collaboration DetailsUnder the terms of the agreement, CRISPR Therapeutics will make an upfront payment of $25 million in cash and $70 million in equity to Sirius Therapeutics. The companies will jointly develop SRSD107 under a 50-50 cost and profit-sharing structure. CRISPR Therapeutics will lead commercialization in the U.S., while Sirius will be responsible for commercialization in Greater China. Additionally, CRISPR Therapeutics will have the option to nominate up to two siRNA targets for research and development. For each target, CRISPR Therapeutics will fund research and retain opt-in rights to lead clinical development and commercialization. Sirius will be eligible to receive milestone payments, as well as tiered royalties ranging from high single to low-double digits. About CRISPR TherapeuticsSince its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic approval of the first-ever CRISPR-based therapy. The Company has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. In 2018, CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic to investigate the treatment of sickle cell disease and transfusion-dependent beta thalassemia. Beginning in late 2023, CASGEVY® (exagamglogene autotemcel [exa-cel]) was approved in several countries to treat eligible patients with either of these conditions. The Nobel Prize-winning CRISPR technology has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit CRISPR THERAPEUTICS® standard character mark and design logo, CTX310™ and CTX320™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. CRISPR Therapeutics Forward-Looking StatementStatements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements made by Drs. Kulkarni, Ji and Ruff in this press release, as well as regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, expectations regarding data, safety and efficacy generally; (ii) data included in this press release, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; (iii) the status and clinical progress of the SRSD107 clinical program and development timelines for such program; (iv) CRISPR Therapeutics strategy and goals; (v) the future activities of the parties pursuant to the collaboration and the expected benefits of CRISPR Therapeutics' collaboration with Sirius Therapeutics; and (vi) the therapeutic value, development, and commercial potential of gene editing and delivery technologies and therapies, including CRISPR/Cas9. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading 'Risk Factors' in its most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. This press release discusses investigational therapies and is not intended to convey conclusions about efficacy or safety as to those investigational therapies or uses of such investigational therapies. There is no guarantee that any investigational therapy will successfully complete clinical development or gain approval from applicable regulatory authorities. About Thromboembolic DisordersThrombosis, or blood clot formation, is the common underlying mechanism of most cases of myocardial infarction, ischemic stroke, and venous thromboembolism. According to a trial in The Lancet1 of regional and global mortality rates, thromboembolic disorders are estimated to cause as many as 1 in 4 deaths worldwide. About SRSD107SRSD107 is a novel double-stranded small interfering ribonucleic acid (siRNA). SRSD107 specifically targets the human coagulation factor XI (FXI) mRNA and inhibits FXI protein expression, thereby blocking the intrinsic coagulation pathway and promoting anticoagulant/anti-thrombotic effects. SRSD107 has been engineered for the potential to enable twice-a-year dosing. About Sirius TherapeuticsSirius is a clinical stage biotech company developing innovative siRNA therapies for global markets. We are dedicated to discovering and developing new treatment options for cardiovascular and cerebrovascular disease and translating siRNA technology into transformative medicine for chronic disease patients. Sirius's most advanced products are SRSD107 for the treatment of thromboembolic disorders, SRSD216 for the treatment of hyperlipoproteinemia, and SRSD101 for the treatment of dyslipidemia. Founded in 2021 by a world-class leadership team and investors, Sirius has established an innovation center in the United States and translational medicine center in China. Sirius has raised nearly US$150 million funding to date from OrbiMed, Creacion Ventures, Hankang Capital, Delos Capital, and BioTrack Capital. Learn more at References:1. Lozano R, Naghavi M, Foreman K, et al. Global and regional mortality from 235 causes of death for 20 age groups in 1990 and 2010: a systematic analysis for the Global Burden of Disease Study 2010. Lancet. 2012;380, 2095-1128. Investor Contact:+1-617-307-7503ir@ Media Contact:+1-617-315-4493media@ A photo accompanying this announcement is available at in to access your portfolio
Yahoo
13-05-2025
- Business
- Yahoo
2 Biotech Stocks That Are Screaming Buys in May
These players share a product, one set to generate significant growth over time. Both of these stocks offer investors a bargain at their prices today. 10 stocks we like better than CRISPR Therapeutics › It's very tempting to pile into the stocks with the strongest positive momentum of the moment -- they've proven themselves and could continue along this path. But in many cases, you could set yourself up for a bigger win if you look to quality companies that have seen their stocks stumble in recent times. This is because you'll buy at a reasonable price and potentially win as the stock recovers and goes on to gain. Two biotech stocks make perfect candidates for this sort of strategy right now. One has declined over the past four years, while the other just slipped in recent weeks. Each represents a bargain buy right now. It's also important to note that these two players share a product, one that should start generating significant revenue in the quarters to come. Let's take a close look at these two biotech stocks that are screaming buys in May. CRISPR Therapeutics (NASDAQ: CRSP) focuses on the exciting field of gene editing, a technology that "fixes" faulty genes responsible for disease. The CRISPR technique involves cutting DNA at a particular location to launch a natural repair process. Importantly, this biotech has proven that its technology works, scoring the first ever product approval for a CRISPR-based therapy. This approval was for blood disorders treatment Casgevy, and rollout began last year. The launch and revenue growth processes take longer for such a product than for standard medicines because gene editing involves several steps. All this means that we'll just start seeing revenue pick up momentum later this year. CRISPR Therapeutics partnered with big biotech Vertex Pharmaceuticals (NASDAQ: VRTX) on Casgevy. Although Vertex takes a larger share of profit at 60%, this is worthwhile because Vertex has the solid commercial experience and infrastructure that could help make the product successful. Vertex also bears the biggest share of the costs. Meanwhile, CRISPR Therapeutics has other promising candidates involved in clinical trials. It just presented encouraging phase 1 data for CTX310. This candidate could address a huge patient population, as it aims to treat people with high cholesterol. The company said that potentially 40 million people in the U.S. alone could be helped by such a product. CRISPR Therapeutics also expects trial updates from other candidates in the areas of oncology and autoimmune disease this year. So, this could be a catalyst-rich year for this biotech, making now, after the stock's 80% four-year decline, a great time to buy and hold on as this story moves into its next chapters. Vertex, as mentioned, partners with CRISPR Therapeutics on Casgevy, and that product should represent a new stream of revenue growth for the company as of this year. On top of this, it's important to remember that Vertex dominates the global cystic fibrosis (CF) treatment market with blockbuster drug Trikafta -- and Vertex just launched a new CF drug, Alyftrek, that's even better than its predecessor. Now Vertex is shifting its Trikafta patients to Alyftrek. In its recent earnings call, the company said the launch of this product, as well as the rollout of another new release -- painkiller Journavx -- are going well. These drugs represent billion-dollar opportunities for Vertex, and Journavx is particularly interesting because it opens the door to an entirely new treatment area for the company. Journavx is a non-opioid pain drug approved for moderate to severe acute pain, and Vertex continues to usher it through clinical trials for other pain indications, including chronic pain. The Journavx approval shows that Vertex not only can be successful in CF, but in other major treatment areas as well. Vertex shares climbed 83% over three years through the start of May. But since Vertex's earnings report last week, when it spoke of a temporary illegal generic competition problem in Russia, the stock has retreated 15%. This has left Vertex trading for 23x forward earnings estimates, down from more than 28x just a few days ago. Considering Vertex's leadership in CF and its new growth drivers, biotech investors won't want to miss out on this top biotech at this bargain price. Before you buy stock in CRISPR Therapeutics, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and CRISPR Therapeutics wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $614,911!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $714,958!* Now, it's worth noting Stock Advisor's total average return is 907% — a market-crushing outperformance compared to 163% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of May 12, 2025 Adria Cimino has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends CRISPR Therapeutics and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy. 2 Biotech Stocks That Are Screaming Buys in May was originally published by The Motley Fool Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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06-05-2025
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CRISPR Therapeutics Provides First Quarter 2025 Financial Results and Announces Positive Top-Line Data from Phase 1 Clinical Trial of CTX310™ Targeting ANGPTL3
A single dose of CTX310 demonstrated dose-dependent decreases in ANGPTL3, TGs, and LDL. Based upon ANGPTL3 knockdown, DL1 and DL2 were minimally active doses, whereas treatment at DL3 and DL4 resulted in reductions of up to 75% of baseline levels in ANGPTL3. CTX310 has been well-tolerated, with no treatment-related severe adverse events (SAEs) and no grade ≥3 adverse events (AEs) reported. No clinically significant changes in alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, or platelets were observed at any dose level. There were no dose-dependent trends in any of these laboratory measurements. Top-line data reported today are from the first 10 patients across the first four cohorts (lean body weight-based doses of DL1 [0.1 mg/kg], DL2[0.3 mg/kg], DL3 [0.6 mg/kg] and DL4 [0.8 mg/kg]) with at least 30 days of follow-up for each participant as of a data cutoff date of April 16, 2025. CTX310 is in an ongoing Phase 1 first-in-human dose escalation clinical trial targeting ANGPTL3 in four patient groups with elevated LDL, TG or both including homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (sHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias (MDL) with levels of TG (>300 mg/dL) and/or LDL-C (>100 mg/dL); >70 mg/dL for subjects with ASCVD. TG and LDL, both of which are validated as surrogate endpoints for clinical benefit and accepted by regulatory agencies, were assessed at various timepoints. CTX310™ targets ANGPTL3, a gene that encodes for key protein involved in the regulation of low-density lipoprotein (LDL) and triglyceride (TG) levels – both well-established risk factors for atherosclerotic heart disease (ASCVD). Loss-of-function mutations in ANGPTL3 are associated with significantly reduced levels of LDL and TGs, as well as reduced risk of ASCVD, without adverse effects on overall health. In the U.S. alone, more than 40 million patients are affected by elevated LDL, severely elevated TGs or both – representing a large addressable patient population. CTX310 is initially focused on a high-risk subset of this group with the greatest unmet medical need and limited effective treatment options. 'CRISPR Therapeutics remains focused on executing our strategic priorities and advancing our portfolio of innovative therapies. We are highly encouraged by the initial data from our Phase 1 trial for CTX310, which demonstrates the power of our in vivo gene editing platform to deliver paradigm changing medicines to patients with serious cardiovascular disease,' said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. 'Additionally, we are pleased with the continued progress of Casgevy and the broader pipeline, and we look forward to sharing further clinical updates in the months ahead.' ZUG, Switzerland and BOSTON, May 06, 2025 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the first quarter ended March 31, 2025. -Clinical trials ongoing for next-generation CAR T product candidates, CTX112™ and CTX131™, targeting CD19 and CD70 across multiple indications; broad updates for CTX112 in oncology and autoimmune diseases expected mid-2025 with CTX131 updates also expected in 2025- -CASGEVY ® continues to gain momentum; more than 65 authorized treatment centers (ATCs) activated globally for CASGEVY, and more than 90 patients have had cells collected across all regions; new patient initiations expected to grow significantly in 2025- -Initial CTX310™ Phase 1 clinical data demonstrates dose-dependent decreases in triglycerides (TG) and low-density lipoprotein (LDL), with peak reduction of up to 82% in TG and up to 81% in LDL, with a well-tolerated safety profile; presentation anticipated at a medical meeting in the second half of 2025- Story Continues Mean % Change from Baseline at Day 30 post-infusion (+/- SEM) Dose Level (DL) DL1 + DL2 0.1 + 0.3 mg/kg (n=6) DL3 0.6 mg/kg (n=3) DL4 0.8 mg/kg (n=1) Patient type HeFH (4), MDL, sHTG MDL (2), HeFH sHTG Triglycerides -10.6% ± 13.1% -55.7% ± 8.0% -81.9% LDL 34.8% ± 27.0% -28.5% ± 24.4% -64.6% Compelling individual patient responses highlight the therapeutic potential of CTX310: a DL4 patient with sHTG had an 82% reduction in triglycerides from a baseline of 1073 mg/dL at day 30, and a DL3 patient with HeFH had an 81% reduction in LDL-C from a baseline of 256 mg/dL at day 90 – supporting the potential for targeted efficacy in high-risk populations. These initial results represent a significant milestone in the advancement of CRISPR Therapeutics' proprietary lipid nanoparticle (LNP) delivery technologies for gene editing in the liver. The Company plans to present the CTX310 Phase 1 data at a medical meeting in the second half of 2025. CTX320 is in an ongoing Phase 1 clinical trial targeting the LPA gene in patients with elevated lipoprotein(a) [Lp(a)], a genetically determined risk factor associated with increased incidence of major adverse cardiovascular events (MACE). Elevated Lp(a) levels are prevalent in up to 20% of the global population. Dose escalation is ongoing, with an update expected in the second quarter of 2025. CRISPR Therapeutics continues to advance two preclinical programs: CTX340™, targeting angiotensinogen (AGT) for the treatment of refractory hypertension, and CTX450™, targeting 5' aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHP). Both candidates are currently in IND/CTA-enabling studies. Hemoglobinopathies and CASGEVY® (exagamglogene autotemcel [exa-cel]) CASGEVY is approved in the U.S., Great Britain, the EU, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain (Bahrain), Canada, Switzerland and the United Arab Emirates (UAE) for the treatment of both SCD and TDT, and launches are ongoing. Building on the foundational launch in 2024, significant progress is being made to bring this transformative therapy to patients worldwide. As of May 1, more than 65 authorized treatment centers (ATCs) have been activated globally and approximately 90 patients have had their first cell collection. The number of new patients initiating cell collection is expected to grow significantly throughout 2025. Vertex has secured a formal reimbursement agreement with NHS England, enabling access to CASGEVY for patients with SCD. This follows an earlier agreement, reaching in August 2024, providing access for eligible patients with TDT. A similar reimbursement agreement has been established in Wales for eligible SCD and TDT patients. Following a positive assessment, national reimbursement was finalized in Austria. In the Middle East, reimbursement was also finalized across the majority of Emirates, following regulatory approval in the UAE. A manufacturing license application has been submitted to the U.S. Food and Drug Administration (FDA), with commercial production in Portsmouth, New Hampshire expected to begin in the second half of 2025. This submission is part of the planned ramp-up of CASGEVY manufacturing capacity as demand for the therapy increases. CRISPR Therapeutics continues to advance its next-generation approaches designed to significantly broaden the addressable patient population for SCD and TDT. The Company's internally developed targeted conditioning program, an anti-CD117 (c-Kit) antibody-drug conjugate (ADC), remains on track in preclinical development. In parallel, the Company is making continued progress in its in vivo editing platform aimed at enabling direct editing of hematopoietic stem cells (HSC) without the need for conditioning. By potentially eliminating the need for conditioning, this approach could unlock access to transformative therapies for a significantly larger patient population. Immuno-Oncology and Autoimmune Disease Programs Clinical trials are ongoing for its next-generation allogeneic CAR T product candidates, CTX112™ and CTX131™, targeting CD19 and CD70, respectively, across multiple indications. Both candidates incorporate novel potency edits which can lead to significantly higher CAR T cell expansion and cytotoxicity, potentially establishing them as best-in-class allogeneic CAR T products for their respective targets. CTX112 is being developed for hematologic malignancies and autoimmune diseases and has the potential to be best-in-class based on preliminary data. Encouraging clinical data from the ongoing Phase 1/2 clinical trial of CTX112 in relapsed or refractory B-cell malignancies supported the FDA's decision to grant Regenerative Medicine Advanced Therapy (RMAT) designation for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma. CTX112 is also in an ongoing Phase 1 clinical trial in autoimmune diseases, including indications such as systemic lupus erythematosus (SLE), systemic sclerosis and inflammatory myositis. Preliminary safety, pharmacokinetic, and pharmacodynamic data from oncology trials support its potential in autoimmune indications. The Company plans to provide an update for both oncology and autoimmune disease in mid-2025. Clinical trials for CTX131™ are ongoing in both solid tumors and hematologic malignancies, with updates expected in 2025. In parallel, an Investigational New Drug (IND) application for glypican-3 (GPC3)-targeted gene-edited autologous CAR T program for the treatment of hepatocellular carcinoma has been opened by our partner, Roswell Park Comprehensive Cancer Center. CRISPR Therapeutics' immuno-oncology and autoimmune disease efforts are supported by a wholly-owned, U.S. manufacturing facility located in Framingham, MA. This investment enables the production of clinical and commercial-stage good manufacturing practice (GMP) materials across the Company's allogeneic cell therapy programs. Regenerative Medicine Programs CRISPR Therapeutics continues to advance its regenerative medicine efforts in Type 1 diabetes (T1D). In addition to CTX211, the Company continues to advance next-generation programs focusing on induced pluripotent stem cell (iPSC) derived, allogeneic, gene-edited, beta islet cell precursors. These approaches aim to achieve insulin independence in T1D patients without the need for chronic immunosuppression. The Company expects to provide an update in 2025. Upcoming Events The Company will participate in the following events in May: 3rd Annual H.C. Wainwright BioConnect Investor Conference, May 20 2025 RBC Capital Markets Global Healthcare Conference, May 20 First Quarter 2025 Financial Results Cash Position: Cash, cash equivalents, and marketable securities were $1,855.3 million as of March 31, 2025, compared to $1,903.8 million as of December 31, 2024. The decrease in cash was primarily driven by operating expenses, offset by proceeds from interest income and employee option exercises. R&D Expenses: R&D expenses were $72.5 million for the first quarter of 2025, compared to $76.2 million for the first quarter of 2024. The decrease in R&D expense was primarily driven by a decrease in employee-related expenses, including stock-based compensation expenses. G&A Expenses: General and administrative expenses were $19.3 million for the first quarter of 2025, compared to $18.0 million for the first quarter of 2024. Collaboration Expense: Collaboration expense, net, was $57.5 million for the first quarter of 2025, compared to $47.0 million for the first quarter of 2024. The increase in collaboration expense, net, was primarily attributable to costs related to CASGEVY and collaboration expenses related to in vivo HSC editing, offset by CASGEVY product sales. Net Loss: Net loss was $136.0 million for the first quarter of 2025, compared to a net loss of $116.6 million for the first quarter of 2024. About CASGEVY® (exagamglogene autotemcel [exa-cel]) CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient's own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate recurrent vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for certain indications in multiple jurisdictions for eligible patients. About the CRISPR Collaboration and Vertex CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CASGEVY represents the first potential treatment to emerge from the joint research program. Under an amended collaboration agreement, Vertex now leads global development, manufacturing, and commercialization of CASGEVY and splits program costs and profits worldwide 60/40 with CRISPR Therapeutics. Vertex is the manufacturer and exclusive license holder of CASGEVY. About CTX112 CTX112 is being developed for both oncology and autoimmune indications. CTX112 is a next-generation, wholly-owned, allogeneic CAR T product candidate targeting Cluster of Differentiation 19, or CD19, which incorporates edits designed to evade the immune system, enhance CAR T potency, and reduce CAR T exhaustion. CTX112 is being investigated in an ongoing clinical trial designed to assess safety and efficacy of the product candidate in adult patients with relapsed or refractory B-cell malignancies who have received at least two prior lines of therapy. In addition, CTX112 is being investigated in an ongoing clinical trial designed to assess the safety and efficacy of the product candidate in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis. About CTX131 CTX131 is being developed for both solid tumors and hematologic malignancies, including T cell lymphomas (TCL). CTX131 is a next-generation, wholly-owned, allogeneic CAR T product candidate targeting Cluster of Differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX131 incorporates edits designed to evade the immune system, prevent fratricide, enhance CAR T potency, and reduce CAR T exhaustion. CTX131 is being investigated in ongoing clinical trials designed to assess the safety and efficacy of the product candidate in adult patients with relapsed or refractory solid tumors and hematologic malignancies, including TCL. About In Vivo Programs CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) platform for the delivery of CRISPR/Cas9 to the liver. The Company's in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards LPA, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company's research and preclinical development candidates include CTX340 and CTX450, targeting angiotensinogen (AGT) for refractory hypertension and 5'-aminolevulinate synthase 1 (ALAS1) for acute hepatic porphyria (AHP), respectively. About CTX211 CTX211 is an allogeneic, gene-edited, stem cell-derived investigational therapy for the treatment of type 1 diabetes (T1D), which incorporates gene edits that aim to make cells hypoimmune and enhance cell fitness. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin in response to glucose. A Phase 1 clinical trial for CTX211 for the treatment of T1D is ongoing. About CRISPR Therapeutics Since its inception over a decade ago, CRISPR Therapeutics has evolved from a research-stage company advancing gene editing programs into a leader that celebrated the historic approval of the first-ever CRISPR-based therapy. The Company has a diverse portfolio of product candidates across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine, cardiovascular, autoimmune, and rare diseases. In 2018, CRISPR Therapeutics advanced the first-ever CRISPR/Cas9 gene-edited therapy into the clinic to investigate the treatment of sickle cell disease and transfusion-dependent beta thalassemia. Beginning in late 2023, CASGEVY® (exagamglogene autotemcel [exa-cel]) was approved in several countries to treat eligible patients with either of these conditions. The Nobel Prize-winning CRISPR technology has revolutionized biomedical research and represents a powerful, clinically validated approach with the potential to create a new class of potentially transformative medicines. To accelerate and expand its efforts, CRISPR Therapeutics has formed strategic partnerships with leading companies including Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Boston, Massachusetts and San Francisco, California. To learn more, visit CRISPR THERAPEUTICS® standard character mark and design logo, CTX112™, CTX131™, CTX211™, CTX310™, CTX320™, CTX340™ and CTX450™ are trademarks and registered trademarks of CRISPR Therapeutics AG. CASGEVY® and the CASGEVY logo are registered trademarks of Vertex Pharmaceuticals Incorporated. All other trademarks and registered trademarks are the property of their respective owners. CRISPR Special Note Regarding Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements made by Dr. Kulkarni in this press release, as well as regarding any or all of the following: (i) CRISPR Therapeutics preclinical studies, clinical trials and pipeline products and programs, including, without limitation, manufacturing capabilities, status of such studies and trials, potential expansion into new indications and expectations regarding data, safety and efficacy generally; (ii) data included in this press release, as well as the ability to use data from ongoing and planned clinical trials for the design and initiation of further clinical trials; (iii) CRISPR Therapeutics strategy, goals, anticipated financial performance and the sufficiency of its cash resources; (iv) plans and expectations for the commercialization of, and anticipated benefits of, CASGEVY, including anticipated patient access to CASGEVY; (v) regulatory submissions and authorizations, including timelines for and expectations regarding additional regulatory agency decisions; (vi) the expected benefits of its collaborations; and (vii) the therapeutic value, development, and commercial potential of gene editing and delivery technologies and therapies, including CRISPR/Cas9. Risks that contribute to the uncertain nature of the forward-looking statements include, without limitation, the risks and uncertainties discussed under the heading 'Risk Factors' in its most recent annual report on Form 10-K and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. We disclaim any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law. This press release also contains information regarding our industry, our business and the markets for certain of our product candidates, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Unless otherwise expressly stated, we obtained this industry, business, market and other data from market research firms and other third parties, including medical publications, government data and similar sources. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. This press release discusses CRISPR/Cas9 gene editing investigational therapies and is not intended to convey conclusions about efficacy or safety as to those investigational therapies or uses of such investigational therapies. There is no guarantee that any investigational therapy will successfully complete clinical development or gain approval from applicable regulatory authorities. Investor Contact: +1-617-307-7503 ir@ Media Contact: +1-617-315-4493 media@ CRISPR Therapeutics AG Condensed Consolidated Statements of Operations (Unaudited, In thousands except share data and per share data) Three Months Ended March 31, 2025 2024 Revenue: Collaboration revenue $ — $ — Grant revenue 865 504 Total revenue $ 865 504 Operating expenses: Research and development 72,484 76,172 General and administrative 19,296 17,953 Collaboration expense, net 57,509 46,966 Total operating expenses 149,289 141,091 Loss from operations (148,424 ) (140,587 ) Total other income, net 13,537 24,720 Net loss before income taxes (134,887 ) (115,867 ) Provision for income taxes (1,109 ) (724 ) Net loss (135,996 ) (116,591 ) Foreign currency translation adjustment 41 (11 ) Unrealized gain (loss) on marketable securities 2,254 (3,454 ) Comprehensive loss $ (133,701 ) $ (120,056 ) Net loss per common share — basic $ (1.58 ) $ (1.43 ) Basic weighted-average common shares outstanding 85,938,720 81,794,630 Net loss per common share — diluted $ (1.58 ) $ (1.43 ) Diluted weighted-average common shares outstanding 85,938,720 81,794,630 CRISPR Therapeutics AG Condensed Consolidated Balance Sheets Data (Unaudited, in thousands) As of March 31, 2025 December 31, 2024 Cash and cash equivalents $ 235,184 $ 298,257 Marketable securities 1,620,101 1,605,569 Working capital 1,748,164 1,849,350 Total assets 2,166,102 2,242,034 Total shareholders' equity 1,829,160 1,932,080
