Latest news with #CIRM
Yahoo
22-05-2025
- Business
- Yahoo
Senti Bio Receives Additional $1.0 Million Tranche from California Institute for Regenerative Medicines (CIRM) Grant for Advancing Clinical Development of SENTI-202
SENTI-202 currently being evaluated in Phase 1 study for the treatment of relapsed/refractory hematologic malignancies, including acute myeloid leukemia SOUTH SAN FRANCISCO, Calif., May 22, 2025 (GLOBE NEWSWIRE) -- Senti Biosciences, Inc. (Nasdaq: SNTI) ('Senti Bio' or the 'Company'), a clinical-stage biotechnology company developing next-generation cell and gene therapies using its proprietary Gene Circuit platform, today reported the receipt of an additional $1.0 million from the California Institute of Regenerative Medicine (CIRM) upon the achievement of clinical study enrollment milestones. As previously announced, the CIRM awarded an $8 million grant to Senti Bio to support the ongoing clinical development of SENTI-202, a potential first-in-class Logic Gated off-the-shelf chimeric antigen receptor natural killer (CAR-NK) investigational cell therapy, for the treatment of relapsed/refractory hematologic malignancies including acute myeloid leukemia (AML). To date, the Company has received a total of $7.4 million of the $8.0 million available under the grant. 'We are grateful for the continued support of the CIRM as we continue to rapidly enroll into our SENTI-202 clinical trial. We are committed to advancing this important program forward and based on the recently presented positive preliminary results, we are becoming more confident in its potential to provide a much-needed treatment option for patients,' commented Timothy Lu, MD, PhD, Co-Founder and CEO of Senti Biosciences. SENTI-202 is the Company's first-in-class off-the-shelf Logic Gated CD33 OR FLT3 NOT EMCN CAR NK cell therapy product candidate designed to selectively target and eliminate CD33 and/or FLT3-expressing hematologic malignancies, such as AML and myelodysplastic syndrome (MDS), while sparing healthy bone marrow cells. SENTI-202 is currently being evaluated in a Phase 1 clinical trial (NCT06325748). Positive preliminary results from the ongoing Phase 1 trial as well as correlative data from patients and preclinical data supporting Logic Gate mechanism of action were recently presented at the American Association for Cancer Research (AACR) Annual Meeting 2025. The data showed that SENTI-202 was well-tolerated in the patients whose data was presented with no dose limiting toxicities and a maximum tolerated dose was not reached. The preliminary recommended Phase 2 dose (RP2D) was identified based on the totality of clinical data, including efficacy, as 1.5 x 109 CAR NK cells administered on Days 0,7,14 in 28-day Cycles following lymphodepleting chemotherapy. Two of three patients in the preliminary RP2D cohort achieved a composite Complete Remission (cCR); 5 of the 7 best overall response evaluable patients across all dose levels achieved an ORR (cCR + morphologic leukemia-free state) outcome and 4 of the 7 achieved cCR (3 CR with full hematologic recovery, and 1 CRh (CR with partial hematologic recovery)). Four of four cCR patients were MRD- (Measurable Residual Disease Negative) as assessed by local standard of care, with longest cCR duration of 8+ months ongoing. About Senti BioSenti Bio is a biotechnology company developing a new generation of cell and gene therapies for patients living with incurable diseases. To achieve this, Senti Bio is leveraging its synthetic biology platform to engineer Gene Circuits into new medicines with enhanced precision and control. These Gene Circuits are designed to precisely kill cancer cells, to spare healthy cells, to increase specificity to target tissues, and/or to be controllable even after administration. The Company's wholly-owned pipeline is comprised of cell therapies engineered with Gene Circuits to target challenging liquid and solid tumor indications. Senti's Gene Circuits have been shown preclinically to work in both NK and T cells. Senti Bio has also preclinically demonstrated the potential breadth of Gene Circuits in other modalities and diseases outside of oncology, and continues to advance these capabilities through partnerships. Forward-Looking Statements This press release and document contain certain statements that are not historical facts and are considered forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements generally are identified by the words 'believe,' 'could,' 'predict,' 'continue,' 'ongoing,' 'project,' 'expect,' 'anticipate,' 'estimate,' 'intend,' 'strategy,' 'future,' 'opportunity,' 'plan,' 'may,' 'should,' 'will,' 'would,' 'will be,' 'will continue,' 'will likely result,' 'forecast,' 'seek,' 'target' and similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Forward-looking statements are predictions, projections, and other statements about future events that are based on current expectations of Senti Bio's management and assumptions, whether or not identified in this document, and, as a result, are subject to risks and uncertainties. Forward-looking statements include, but are not limited to, expectations regarding Senti Bio's progress future results from its clinical trials for SENTI-202; and the ability of any product candidate to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study data. These forward-looking statements are provided for illustrative purposes only and are not intended to serve as and must not be relied on by any investor as, a guarantee, an assurance, a prediction, or a definitive statement of fact or probability. Actual events and circumstances are difficult or impossible to predict and will differ from assumptions. Many actual events and circumstances are beyond the control of Senti Bio. Many factors could cause actual future results to differ materially from the forward-looking statements in this document, including but not limited to: (i) changes in domestic and foreign business, market, financial, political and legal conditions, (ii) changes in the competitive and highly regulated industries in which Senti Bio operates, variations in operating performance across competitors, changes in laws and regulations affecting Senti Bio's business, (iii) the ability to implement business plans, forecasts and other expectations, (iv) the risk of downturns and a changing regulatory landscape in Senti Bio's highly competitive industry, (v) risks relating to the uncertainty of any projected financial information with respect to Senti Bio, (vi) risks related to uncertainty in the timing or results of Senti Bio's clinical trial start up, clinical studies, patient enrollment, and GMP manufacturing startup activities, (vii) Senti Bio's dependence on third parties in connection with clinical trial startup, clinical studies, and GMP manufacturing activities, (viii) risks related to delays and other impacts from macroeconomic and geopolitical events, increasing rates of inflation and rising interest rates on business operations, (ix) risks related to the timing and utilization of the grant from CIRM, and (x) the success of any future research and development efforts by Senti Bio. The foregoing list of factors is not exhaustive. You should carefully consider the foregoing factors and the other risks and uncertainties described in the 'Risk Factors' section of Senti Bio's most recent periodic report filed with the U.S. Securities and Exchange Commission ('SEC'), and other documents filed by Senti Bio from time to time with the SEC. These filings identify and address other important risks and uncertainties that could cause actual events and results to differ materially from those contained in the forward-looking statements in this document. There may be additional risks that Senti Bio does not presently know, or that Senti Bio currently believes are immaterial that could also cause actual results to differ from those contained in the forward-looking statements in this document. Forward-looking statements speak only as of the date they are made. Senti Bio anticipates that subsequent events and developments may cause Senti Bio's assessments to change. Except as required by law, Senti Bio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events, or of Other Information About Senti Biosciences, more information, please visit the Senti Bio website at or follow Senti Bio on X (@SentiBio) and LinkedIn (Senti Biosciences). Investors and others should note that we communicate with our investors and the public using our company website ( including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X and LinkedIn. The information that we post on our website or on X or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. Investor Contact:JTC Team, LLCJenene Thomas(908) 824-0775SNTI@ 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
Business Wire
12-05-2025
- Business
- Business Wire
Genascence Phase 1b DONATELLO Trial Evaluating Potential First-in-Class Gene Therapy for Knee Osteoarthritis (OA) Meets Primary Endpoint Showing GNSC-001 Was Safe and Well Tolerated Across Multiple Dosing Arms
PALO ALTO, Calif.--(BUSINESS WIRE)--Genascence Corporation ('Genascence'), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced positive 12-month safety and biomarker results from the Phase 1b DONATELLO clinical trial evaluating GNSC-001, a potential first-in-class gene therapy blocking interleukin 1 (IL-1) for the treatment of knee osteoarthritis (OA). Results from the 12-month analysis showed the study met the primary endpoint, demonstrating continued safety and tolerability across all doses tested, as well as the key secondary endpoint showing sustained IL-1Ra expression in synovial fluid, building on data reported through the six-month visit. GNSC-001 is a genetic medicine – a recombinant adeno-associated viral vector expressing an optimized human interleukin-1 receptor antagonist (IL-1Ra), a naturally occurring protein that blocks IL-1 signaling. IL-1 is considered one of the key mediators involved in the pathogenesis of OA, causing inflammation, joint pain, and cartilage destruction. GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single intra-articular injection into the affected joint. The U.S. Food and Drug Administration (FDA) granted GNSC-001 Fast Track designation in the fourth quarter of 2024. Genascence recently completed a successful meeting with the FDA on the design of the Phase 2b/3 clinical trial of GNSC-001 focused on clinical efficacy and plans to initiate the Phase 2b/3 study in 2026. 'Osteoarthritis is incapacitating, causing years of pain and disability for people living with the disease. Current treatment options are limited to managing pain and do not treat the underlying disease itself,' said Thomas Chalberg, Ph.D., founder and CEO of Genascence. 'The 12-month safety and sustained IL-1Ra expression data affirms the promise of GNSC-001 to potentially transform the treatment paradigm for OA. We are pleased by the successful meeting with the FDA, and look forward to initiating the study, transitioning GNSC-001 to late-stage clinical development so we can bring a new treatment option to people suffering from this disabling disease.' 'GNSC-001 is the first IL-1 inhibitor that has been shown to generate IL-1Ra expression levels that reach and maintain therapeutic thresholds long-term following a single administration,' said Annahita Keravala, Ph.D., chief scientific officer (CSO) of Genascence. 'Results from the DONATELLO clinical trial suggest that our novel therapeutic approach, a local gene therapy can potentially have therapeutic benefit in knee OA, a disease for which there are no treatments beyond management of symptoms. This would be transformative for people suffering from this debilitating condition, and thus warrants further development.' "These results from the DONATELLO trial reflect the kind of innovation CIRM was created to support,' said Lisa Kadyk, Ph.D., CIRM Fellow, Clinical Development at the California Institute for Regenerative Medicine (CIRM), which supported the DONATELLO clinical trial with a $12 million award. 'By harnessing the power of gene therapy, GNSC-001 represents a novel and potentially disease-modifying approach to treating osteoarthritis. We are encouraged by the 12-month safety and biomarker data and proud to have supported this important step toward a more effective, long-term treatment for people living with knee OA." Dr. Keravala will present data from the six-month interim analysis of the DONATELLO clinical trial at the 28 th Annual American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting, being held May 13-17, 2025 in New Orleans, LA and virtually. The poster presentation details are provided below. Title: A Randomized, Double-Blind, Placebo-Controlled Dose-Ranging Phase 1b Study Evaluating Safety, Tolerability, and Pharmacodynamics of a Local AAV-Mediated Anti-Interluekin-1 Gene Therapy in Subjects with Knee Osteoarthritis: 6-Month Interim Results Date/Time: Thursday, May 15, 2025, 5:30-7:00 pm CT Location: Poster Hall Abstract Number: AMA616 Poster Number: 1849 Abstracts can be found at About the DONATELLO Clinical Trial The DONATELLO Phase 1b clinical trial (NCT05835895) is a double-blind, placebo-controlled dose-ranging study designed to evaluate the safety, tolerability, and pharmacodynamics of a single intra-articular injection of GNSC-001 in patients with OA of the knee. The study enrolled 67 participants with OA at 10 centers across the U.S. The first five groups were randomized to receive GNSC-001 at doses of 110 12 vg or 110 13 vg, with or without a short course of oral steroids for immune-conditioning, or a placebo (saline) injection. The trial was expanded to enroll an additional, non-randomized arm. In this arm, pre-treatment synovial fluid sampling was required for entry, and subjects received 110 13 vg GNSC-001 with an abbreviated three-day course of oral steroids plus a local, intra-articular steroid injection. Data and Safety Summary The primary endpoints of the DONATELLO clinical trial are safety and tolerability. Through 12 months of follow-up, data show that GNSC-001 was well tolerated, with no treatment-emergent or treatment-related deaths, serious adverse events (SAEs), or adverse event (AE)-related withdrawals reported. The most common target knee AEs included arthralgia, joint swelling, and joint effusion. The study's secondary endpoints include expression levels of interleukin-1 receptor antagonist (IL-1Ra) in synovial fluid at Month 12, as well as change from baseline to Month 12. Results revealed that mean expression of IL-1Ra reached target therapeutic levels in multiple arms of the study and remained above the target threshold throughout the 12-month follow up period. Immune-conditioning with a short course of steroids generally supported higher levels of prolonged IL-1Ra expression. The DONATELLO clinical trial was supported by a $12 million award from the California Institute for Regenerative Medicine (CLIN2-14265). About Osteoarthritis (OA) of the Knee Osteoarthritis (OA) is a progressive joint disease that is a leading cause of disability. It is characterized by destruction of cartilage and structural changes in bone within the joint, which contribute to pain and loss of joint function. Osteoarthritis affects more than 30 million Americans and is increasing as a result of the aging population and increasing prevalence of obesity. Osteoarthritis represents a major economic burden, owing to direct medical costs and loss of productivity. Each year, millions of patients are treated for knee OA with NSAIDs, opioids, and steroid injections into the knee to manage their knee pain. There are no currently available therapies known to alter or slow down OA progression. About Genascence Corporation Genascence, a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, is developing life-changing treatments for highly prevalent conditions affecting millions of people. The company was founded in 2017 with technology licensed from three leading U.S. research institutions: Mayo Clinic, University of Florida, and NYU Langone Health. Headquartered in Palo Alto, California, Genascence's founders and leadership team have deep experience in the design, development, and manufacturing of successful gene therapies and biological medicines. For more information, please visit
Business Wire
24-04-2025
- Business
- Business Wire
Ray Therapeutics Awarded $8M CIRM Grant to Advance RTx-015 Gene Therapy for Retinitis Pigmentosa
BERKELEY, Calif.--(BUSINESS WIRE)-- Ray Therapeutics, a biotechnology company developing optogenetic gene therapies for vision restoration, today announced it has been awarded an $8 million grant from the California Institute for Regenerative Medicine (CIRM). The grant will support the company's ongoing clinical development of RTx-015 for the treatment of retinitis pigmentosa (RP), a progressive and debilitating inherited retinal disease that leads to blindness. Ray Therapeutics' approach uses an optimized optogenetic gene therapy to deliver light-sensitive proteins to the retina of the eye to restore visual function to patients with RP, regardless of the underlying genetic cause. 'Retinitis pigmentosa remains a devastating condition with no approved treatments for the vast majority of patients,' said Paul Bresge, CEO & Co-Founder, Ray Therapeutics. 'We are deeply grateful to CIRM for their belief in our science and their continued support of our programs. We are honored to partner with CIRM as we advance therapies that have the potential to transform the lives of patients.' 'Restoring vision is one of the most powerful ways we can improve quality of life,' said Jonathan Thomas, PhD, JD, President and CEO, CIRM. 'Ray Therapeutics is advancing a potential breakthrough treatment for a high unmet medical need for people in California and around the world with advanced RP, for whom there are currently no treatment options. We are proud to support this exciting program.' Ray Therapeutics' gene therapy program received a unanimous vote of support from CIRM's scientific and patient advocate reviewers. It was recognized by CIRM's independent Grants Working Group (GWG) as having exceptional scientific merit and a high potential for impact, with all 15 reviewers scoring the application at the highest level. About Retinitis Pigmentosa RP is a genetic disease in which the photoreceptors gradually degenerate resulting in complete, or nearly complete blindness for most patients. The symptoms of RP include night blindness, reduced visual fields, and eventual loss of visual acuity. Patients are typically diagnosed in the first decades of life. It is estimated that more than half a million people are affected by RP worldwide. At present, no effective treatment is available for RP. About Ray Therapeutics Ray Therapeutics is a clinical-stage biopharmaceutical company advancing optogenetic therapies to restore vision in patients with severe retinal degeneration. By delivering a bioengineered, highly light-sensitive protein to targeted retinal cells, the approach is designed to improve visual function regardless of the underlying genetic mutation. The company's lead candidate, RTx-015, targets retinal ganglion cells and is currently being evaluated in a Phase 1 clinical trial for patients with retinitis pigmentosa and choroideremia. A second program, RTx-021, which targets retinal bipolar cells, is in late-stage preclinical development for Stargardt disease and geographic atrophy secondary to age-related macular degeneration. Ray Therapeutics is headquartered in Berkeley, California. For more information, visit About the California Institute for Regenerative Medicine (CIRM) The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to accelerate regenerative medicine research to deliver treatments for patients with unmet medical needs. Established in 2004 through the passage of Proposition 71, CIRM was initially funded with $3 billion from the state of California to support ongoing research, and in 2020, was funded again with another $5.5 billion through Proposition 14 to continue the Agency's important work. CIRM has provided billions in funding to support stem cell, genetic research, and development programs in its portfolio. Through the Agency's research, infrastructure, and education programs, CIRM aims to transform the field of regenerative medicine, stimulate economic growth, and improve the lives of diverse communities throughout the state. For more information, go to
Associated Press
04-04-2025
- Business
- Associated Press
Tr1X Awarded $8 Million CIRM Grant to Support TRX103 Phase 1/2a Clinical Trial for Graft-Versus-Host Disease
SAN DIEGO, April 04, 2025 (GLOBE NEWSWIRE) -- Tr1X, Inc. (pronounced 'Trix'), a clinical-stage biopharmaceutical company developing first-in-class allogeneic engineered Treg and CAR-Treg cell therapies with the potential to cure autoimmune and inflammatory diseases, today announced that the California Institute for Regenerative Medicine (CIRM) has awarded the Company an $8 million grant to support its ongoing Phase 1/2a clinical trial of TRX103, an engineered Tr1 Treg cell therapy for use in the prevention of graft-versus-host disease (GvHD) in blood cancer patients undergoing mismatched stem cell transplants. This award follows a $4 million CIRM grant received by Tr1X in early 2024 for late-stage preclinical research that helped to advance TRX103 to human trials. The TRX103 Phase 1/2a clinical trial is being led by Maria Grazia Roncarolo, MD, co-founder, president and head of R&D at Tr1X, with patients enrolling at leading stem cell transplant centers across the country. The trial is designed to treat patients receiving a mismatched or haploidentical transplant with a one-time infusion of TRX103. The Company has reported positive initial persistence and safety data in the first two patient cohorts, with additional data anticipated to be reported in 2025. TRX103, which is the Company's lead program, is also being tested in a Phase 1/2a trial for patients with treatment-refractory Crohn's Disease. 'With CIRM's support, TRX103 has rapidly advanced from promising preclinical findings into clinical trials, bringing us closer to transforming mismatched stem cell transplants into safer, life-saving options,' said Dr. Roncarolo. 'By harnessing the unique power of engineered Tr1 cells, our goal is to prevent graft-versus-host disease, improve immune reconstitution and induce tolerance to host cells—therefore expanding access to curative therapies for patients who urgently need them.' GvHD occurs when donor cells attack the recipient's tissue and organs, often causing life-threatening complications. Current treatments for GvHD, which suppress the immune system, can lead to infections and hinder cancer treatment effectiveness. TRX103, an allogeneic engineered regulatory T cell product, may improve transplant access for patients without a matched donor while reducing GvHD risks. Dr. Roncarolo added, 'This trial represents a hope for all of us transplanters—not only for better outcomes but also for a future where effective, personalized treatments like hematopoietic stem cell transplant become accessible to all patients, regardless of donor match.' About TRX103 TRX103 is an investigational allogeneic off-the-shelf engineered T cell product generated from CD4+ cells sourced from healthy donors. These CD4+ cells are engineered to become cells that mimic the function of type 1 regulatory (Tr1) cells, called TRX cells. Tr1X is developing TRX103 for the treatment of several immune and inflammatory disorders. Multiple preclinical studies have shown TRX103 to be tolerable and effective and to have the potential to reset the immune system to a healthy state. TRX103 has the potential to overcome major limitations of current cell therapies for autoimmune diseases, which include limited persistence and side effects including cytokine release syndrome (CRS) and neurotoxicity. About Tr1X Tr1X is a clinical-stage private biotechnology company pioneering a new class of curative cell therapies designed to fundamentally reset the immune system in patients with autoimmune and inflammatory diseases. Founded by leading scientists who discovered Tr1 cells, Tr1X develops off-the-shelf, allogeneic Treg and CAR-Treg therapies engineered for superior safety, scalability and accessibility, eliminating the need for standard lymphodepletion and enabling durable restoration of immune tolerance and a potential cure. Supported by top-tier investors including The Column Group, NEVA SGR and Alexandria Venture Investments, and backed by grant funding from the California Institute for Regenerative Medicine (CIRM), Tr1X aims to transform treatment paradigms from lifelong disease management to permanent cures. For more information, visit Media Contact:
Associated Press
05-02-2025
- Business
- Associated Press
Entos Pharmaceuticals Awarded $4 Million USD in Funding from the California Institute for Regenerative Medicine (CIRM) for its Congenital Generalized Lipodystrophy Program
SAN DIEGO--(BUSINESS WIRE)--Feb 5, 2025-- Entos ® Pharmaceuticals (Entos), a clinical-stage genetic medicines company committed to using its Fusogenix™ PLV™ technology to develop cures and improve the lives of patients and their families, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4M USD grant. The funds will support the completion of IND-enabling activities for the development of ENTLEP001, a durable genetic medicine for the treatment of congenital generalized lipodystrophy (CGL). CGL is a rare genetic disorder caused by mutations in the human leptin gene. People with CGL are leptin-deficient and cannot make adipose tissue. The disorder is associated with severe, hard-to-treat diabetes, hypertriglyceridemia, and potential complications affecting the liver and heart. ENTLEP001 is a systemically administered investigational gene therapy using the Fusogenix PLV platform to express unmodified human leptin in the body. The treatment is designed to be durable, redosable, and provide biologically relevant levels of native human leptin expression. Preclinical studies with ENTLEP001 using a leptin-deficient mouse model resulted in clinically relevant leptin expression, disease-modifying activity including normalization of glucose and insulin levels, and decreased weight gain and food intake. The Fusogenix PLV platform is a fusion-powered delivery system for genetic medicines that combines the best aspects of viral and non-viral approaches. Using a completely new mechanism for intracellular delivery of RNA, DNA and gene editing therapies, Fusogenix PLV is suitable for a wide range of best-in-class genetic medicines. Details regarding the platform's safety, efficacy, and biodistribution in various animal models were published in Cell in September 2024. 'Despite advancements in CGL treatment with FDA-approved metreleptin, there are still significant unmet needs,' said Steve Chen, MD, CMO of Entos Pharmaceuticals. 'Metreleptin requires daily subcutaneous injections and in some patients is associated with significant injection site adverse events. Although rare, there are reported cases of neutralizing anti-leptin antibody in patients receiving metreleptin. In contrast, ENTLEP001 is a genetic medicine that expresses native human leptin and may require dosing only once or twice a year, eliminating injection site reactions, simplifying patient compliance, and improving outcomes.' 'This award from CIRM is an important validation for our lead program in CGL as we work to advance our second product into the clinic using the Fusogenix PLV platform,' said John Lewis, CEO of Entos Pharmaceuticals. 'We deeply appreciate their support and recognition of the crucial need to address rare genetic disorders like congenital generalized lipodystrophy and the potential for novel genetic medicine approaches like the Fusogenix PLV to create life-changing treatments for patients and their families.' About Congenital Generalized Lipodystrophy (CGL) Congenital generalized lipodystrophy (CGL), also known as Berardinelli-Seip syndrome, is a rare genetic disorder characterized by the near total loss of adipose tissue associated with extreme muscularity that is often present at birth or soon thereafter. CGL is associated with metabolic complications related to insulin resistance such as severe diabetes and hypertriglyceridemia. Diabetes associated with CGL is often very difficult to treat. Additional complications affecting the liver and heart can also occur. The symptoms and severity of CGL can vary greatly from one person to another. CGL is caused by mutations in the human leptin gene LEP. About Entos Pharmaceuticals Inc. A new reality in genetic medicine lies ahead, one that will be ushered in with the advent of safe, effective, and re-dosable nucleic acid delivery technologies. Since its inception in 2016, Entos® has been dedicated to advancing next-generation genetic medicines using our proprietary Fusogenix™ PLV™ drug delivery system. The Fusogenix PLV platform is formulated with FAST proteins to enable the delivery of nucleic acid to target cells through direct fusion. Entos is pioneering the development of life-changing medicines for patients and has partnered with global companies, such as Eli Lilly, to accelerate and expand the impact of our platform. Entos Pharmaceuticals Inc. is headquartered in Edmonton, Canada, with its wholly owned U.S. and U.K. subsidiaries based in San Diego, California and London, United Kingdom, respectively. For more information, visit or follow Entos on LinkedIn. About California Institute for Regenerative Medicine (CIRM) At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is the world's largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality. For more information go to Forward Looking Statements This press release contains forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements contained in this press release include statements regarding Entos and its belief as to the mode of action and potential and benefits of the Fusogenix proteolipid vehicle platform and other statements related to anticipated developments in the Company's business and technologies. In any forward-looking statement in which Entos expresses an expectation or belief as to future results, such expectations or beliefs are expressed in good faith and are believed to have a reasonable basis, but there can be no assurance that the statement or expectation, or belief will be achieved. Such forward-looking statements involve known and unknown risks and uncertainties, which could cause the Company's actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue research and development projects, the efficacy of Fusogenix as a nucleic acid delivery vehicle, uncertainties related to the research and development of pharmaceuticals, uncertainties related to the regulatory process and general changes to the economic environment. We may incur expenses or delays relating to such events outside of our control, which could have a material adverse impact on our business, operating results, and financial condition. Investors should consult with the U.S. Securities Commission for additional information on risks and uncertainties relating to the forward-looking statements. Investors are cautioned against placing undue reliance on forward-looking statements. The Company does not undertake any obligation to update these forward-looking statements except as required by applicable laws. This press release contains forward-looking statements, within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements contained in this press release include statements regarding Entos and its belief as to the mode of action and potential and benefits of the Fusogenix proteolipid vehicle platform and other statements related to anticipated developments in the Company's business and technologies. In any forward-looking statement in which Entos expresses an expectation or belief as to future results, such expectations or beliefs are expressed in good faith and are believed to have a reasonable basis, but there can be no assurance that the statement or expectation, or belief will be achieved. Such forward-looking statements involve known and unknown risks and uncertainties, which could cause the Company's actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue research and development projects, the efficacy of Fusogenix as a nucleic acid delivery vehicle, uncertainties related to the research and development of pharmaceuticals, uncertainties related to the regulatory process and general changes to the economic environment. We may incur expenses or delays relating to such events outside of our control, which could have a material adverse impact on our business, operating results, and financial condition. Investors should consult with the U.S. Securities Commission for additional information on risks and uncertainties relating to the forward-looking statements. Investors are cautioned against placing undue reliance on forward-looking statements. The Company does not undertake any obligation to update these forward-looking statements except as required by applicable laws. Entos ® word mark and design logo, Fusogenix™ and PLV™ are registered trademarks of Entos Pharmaceuticals Inc. All other trademarks and registered trademarks are the property of their respective owners. John Lewis, Ph.D., Founder and CEO [email protected] Pharmaceuticals Media Contact: Perrin Beatty, Ph.D. 1-800-727-0884 SOURCE: Entos Pharmaceuticals Inc. Copyright Business Wire 2025. PUB: 02/05/2025 11:10 AM/DISC: 02/05/2025 11:10 AM
