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Blind Bay Area woman will be part of group summiting Mount Kilimanjaro
Blind Bay Area woman will be part of group summiting Mount Kilimanjaro

CBS News

time12-08-2025

  • CBS News

Blind Bay Area woman will be part of group summiting Mount Kilimanjaro

A blind Mountain View woman is training to be part of a group that will hike to the top of Mount Kilimanjaro. "You know, a lot of people think that when you hike a mountain, you do it so you can get the view at the summit. Obviously, for someone like me, when we hike up Mount Kilimanjaro, I'm not going to be able to see the view at the very top," Kristie Colton said. "But there is so much more that can be taken away from this journey." Colton is blind because of a condition that she has lived with since she was 9, called Stargardt disease. It's a form of macular degeneration that affects central vision. "So, if I hold my finger out here, I can see it because it's within my peripheral vision. But then as it comes in towards the center, my finger essentially disappears," Colton said. But that is not enough to stop her from reaching new heights. In the fall, she will be part of a history-making expedition in which 11 blind hikers, with guides, will summit Mt. Kilimanjaro. "I realized that I couldn't turn this opportunity down. It's so once-in-a-lifetime," Colton said. In order to get there, she has to train — a lot. She has been doing intense workouts designed to build strength and stamina. "I need to make sure I have a little bit of extra strength in my ankles to counteract the fact that I don't always see exactly where my feet are landing," Colton told KPIX. Each step of the process is fueled by her determination not to let her disability drive her away from experiencing life to the fullest. "When I was in high school, I was really embarrassed to be seen as disabled – it was something that I would go out of my way to hide," Colton said. "Sharing this vulnerable side of me, I think, has made me so much closer with people." That includes her friends Grace Eysenbach and Jungyeon Park, who will be guiding her every step of the way up Kilimanjaro. "Kristie is so much more than just how much she can see. She's funny, she's spunky, she's a dreamer, she inspires everyone else around her to dream really big about themselves," Eysenbach said. Colton said that what was once her soft spot has now become a source of strength. "I might not experience the world in the same way, but there are so many things that I still love to do and enjoy doing because vision is

Alkeus Pharmaceuticals Announces Presentation of Positive Interim TEASE-3 Study Data in Early-Stage Stargardt Disease Patients Treated with Oral Gildeuretinol
Alkeus Pharmaceuticals Announces Presentation of Positive Interim TEASE-3 Study Data in Early-Stage Stargardt Disease Patients Treated with Oral Gildeuretinol

Yahoo

time07-08-2025

  • Business
  • Yahoo

Alkeus Pharmaceuticals Announces Presentation of Positive Interim TEASE-3 Study Data in Early-Stage Stargardt Disease Patients Treated with Oral Gildeuretinol

Gildeuretinol acetate demonstrated less loss in ellipsoid zone (EZ) area compared to historical sibling controls. Early-stage Stargardt disease patients treated with gildeuretinol for two to seven years exhibited relatively stable disease over the course therapy. Gildeuretinol was well tolerated and had a safety profile consistent with prior gildeuretinol studies, with the majority of adverse events being mild or moderate. CAMBRIDGE, Mass., Aug. 07, 2025 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc. today announced the presentation of an interim data update from its TEASE-3 study demonstrating that five early-stage Stargardt disease patients treated with investigational oral gildeuretinol acetate experienced less loss in ellipsoid zone (EZ) area compared to historical sibling controls and overall disease remained relatively stable while on therapy ranging between two to seven years. The study update was presented during the 43rd American Society of Retina Specialists (ASRS) Annual Scientific Meeting, held July 30 – Aug. 2 in Long Beach, Calif. 'We are encouraged by this new interim data from the TEASE-3 study of gildeuretinol in early-stage Stargardt patients with confirmed disease-causing ABCA4 genetic mutations,' said Seemi Khan, M.D., M.P.H., M.B.A., Chief Medical Officer of Alkeus Pharmaceuticals. 'These additional data further support the potential of investigational gildeuretinol to slow disease progression and stabilize vision in individuals living with Stargardt disease. We are excited to continue advancing our efforts towards delivering a treatment for Stargardt disease.' TEASE-3, the first clinical trial in early-stage Stargardt disease, is an open-label study of gildeuretinol in genetically confirmed patients who exhibit early retinal changes on imaging but have not yet developed meaningful symptoms of vision loss. TEASE-3 study participants each have a sibling who was previously diagnosed with Stargardt disease. The disease progression over two years is assessed by retinal imaging and functional outcome measures. After the initial two-year treatment period, patients continue to receive gildeuretinol while enrolled in an open label long-term extension study. In the TEASE-3 study, five patients have completed two years of treatment with once-daily oral gildeuretinol. Gildeuretinol was well tolerated and demonstrated a safety profile consistent with prior gildeuretinol studies, with the majority of adverse events being mild or moderate in severity. 'Time is of the essence for patients losing vision from early-onset Stargardt disease,' said Kenneth Fan, M.D., M.B.A., Retina Consultants of Texas. 'With no approved treatments available, these data inform our understanding of retinal health in early-stage disease and underscore the importance of ongoing clinical evaluation.' About Stargardt Disease Stargardt disease is a serious cause of blindness in children and adults, with an estimated 30,000 people affected in the U.S. and more than 150,000 worldwide. There is no approved treatment. In individuals with Stargardt disease, the ABCA4 protein is defective. This defect in the protein results in the accelerated dimerization of vitamin A, forming toxic by-products that irreversibly damage the retina, resulting in progressive vision loss. About Alkeus Pharmaceuticals Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases. Based in Cambridge, Mass., Alkeus is backed by institutional investors led by Bain Capital Life Sciences. Alkeus is developing therapies for serious diseases of the eye with high unmet need. Alkeus' breakthrough-designated lead candidate, oral gildeuretinol acetate (ALK-001), is being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy secondary to age-related macular degeneration. About Gildeuretinol Acetate (ALK-001) Oral gildeuretinol acetate (ALK-001) is a new molecular entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. Gildeuretinol is being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy secondary to age-related macular degeneration. Gildeuretinol has received Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for Stargardt disease from the U.S. Food and Drug Administration. About the TEASE Program The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol acetate (ALK-001) in Stargardt disease, denoted as TEASE-1, TEASE-2, TEASE-3 and TEASE-4. The TEASE-1 study was a randomized, double-masked, placebo-controlled trial in 50 patients with advanced Stargardt disease and is complete. The TEASE-2 trial is a randomized, double-masked, placebo-controlled trial in 80 patients with moderate Stargardt disease, expected to read out topline data in 2025. TEASE-3, a clinical trial in early-stage Stargardt disease, is an ongoing open-label study of gildeuretinol in genetically confirmed patients with early signs of disease visible on retinal imaging, but who have not begun experiencing meaningful symptoms of vision loss. TEASE-4 is an ongoing open-label extension study. For further information, please contact: Media RelationsMedia@ 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

Alkeus Pharmaceuticals Announces Presentation of Positive Interim TEASE-3 Study Data in Early-Stage Stargardt Disease Patients Treated with Oral Gildeuretinol
Alkeus Pharmaceuticals Announces Presentation of Positive Interim TEASE-3 Study Data in Early-Stage Stargardt Disease Patients Treated with Oral Gildeuretinol

Yahoo

time07-08-2025

  • Business
  • Yahoo

Alkeus Pharmaceuticals Announces Presentation of Positive Interim TEASE-3 Study Data in Early-Stage Stargardt Disease Patients Treated with Oral Gildeuretinol

Gildeuretinol acetate demonstrated less loss in ellipsoid zone (EZ) area compared to historical sibling controls. Early-stage Stargardt disease patients treated with gildeuretinol for two to seven years exhibited relatively stable disease over the course therapy. Gildeuretinol was well tolerated and had a safety profile consistent with prior gildeuretinol studies, with the majority of adverse events being mild or moderate. CAMBRIDGE, Mass., Aug. 07, 2025 (GLOBE NEWSWIRE) -- Alkeus Pharmaceuticals, Inc. today announced the presentation of an interim data update from its TEASE-3 study demonstrating that five early-stage Stargardt disease patients treated with investigational oral gildeuretinol acetate experienced less loss in ellipsoid zone (EZ) area compared to historical sibling controls and overall disease remained relatively stable while on therapy ranging between two to seven years. The study update was presented during the 43rd American Society of Retina Specialists (ASRS) Annual Scientific Meeting, held July 30 – Aug. 2 in Long Beach, Calif. 'We are encouraged by this new interim data from the TEASE-3 study of gildeuretinol in early-stage Stargardt patients with confirmed disease-causing ABCA4 genetic mutations,' said Seemi Khan, M.D., M.P.H., M.B.A., Chief Medical Officer of Alkeus Pharmaceuticals. 'These additional data further support the potential of investigational gildeuretinol to slow disease progression and stabilize vision in individuals living with Stargardt disease. We are excited to continue advancing our efforts towards delivering a treatment for Stargardt disease.' TEASE-3, the first clinical trial in early-stage Stargardt disease, is an open-label study of gildeuretinol in genetically confirmed patients who exhibit early retinal changes on imaging but have not yet developed meaningful symptoms of vision loss. TEASE-3 study participants each have a sibling who was previously diagnosed with Stargardt disease. The disease progression over two years is assessed by retinal imaging and functional outcome measures. After the initial two-year treatment period, patients continue to receive gildeuretinol while enrolled in an open label long-term extension study. In the TEASE-3 study, five patients have completed two years of treatment with once-daily oral gildeuretinol. Gildeuretinol was well tolerated and demonstrated a safety profile consistent with prior gildeuretinol studies, with the majority of adverse events being mild or moderate in severity. 'Time is of the essence for patients losing vision from early-onset Stargardt disease,' said Kenneth Fan, M.D., M.B.A., Retina Consultants of Texas. 'With no approved treatments available, these data inform our understanding of retinal health in early-stage disease and underscore the importance of ongoing clinical evaluation.' About Stargardt Disease Stargardt disease is a serious cause of blindness in children and adults, with an estimated 30,000 people affected in the U.S. and more than 150,000 worldwide. There is no approved treatment. In individuals with Stargardt disease, the ABCA4 protein is defective. This defect in the protein results in the accelerated dimerization of vitamin A, forming toxic by-products that irreversibly damage the retina, resulting in progressive vision loss. About Alkeus Pharmaceuticals Alkeus Pharmaceuticals, Inc. is a private biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases. Based in Cambridge, Mass., Alkeus is backed by institutional investors led by Bain Capital Life Sciences. Alkeus is developing therapies for serious diseases of the eye with high unmet need. Alkeus' breakthrough-designated lead candidate, oral gildeuretinol acetate (ALK-001), is being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy secondary to age-related macular degeneration. About Gildeuretinol Acetate (ALK-001) Oral gildeuretinol acetate (ALK-001) is a new molecular entity designed to reduce the dimerization of vitamin A without modulating the visual cycle. Gildeuretinol is being evaluated in clinical trials for the treatment of Stargardt disease and for geographic atrophy secondary to age-related macular degeneration. Gildeuretinol has received Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for Stargardt disease from the U.S. Food and Drug Administration. About the TEASE Program The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol acetate (ALK-001) in Stargardt disease, denoted as TEASE-1, TEASE-2, TEASE-3 and TEASE-4. The TEASE-1 study was a randomized, double-masked, placebo-controlled trial in 50 patients with advanced Stargardt disease and is complete. The TEASE-2 trial is a randomized, double-masked, placebo-controlled trial in 80 patients with moderate Stargardt disease, expected to read out topline data in 2025. TEASE-3, a clinical trial in early-stage Stargardt disease, is an ongoing open-label study of gildeuretinol in genetically confirmed patients with early signs of disease visible on retinal imaging, but who have not begun experiencing meaningful symptoms of vision loss. TEASE-4 is an ongoing open-label extension study. For further information, please contact: Media RelationsMedia@ 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

Ocugen, Inc. Announces U.S. FDA Clearance of Investigational New Drug Amendment to Initiate Phase 2/3 Pivotal Confirmatory Clinical Trial of OCU410ST—Modifier Gene Therapy Candidate for Stargardt Disease
Ocugen, Inc. Announces U.S. FDA Clearance of Investigational New Drug Amendment to Initiate Phase 2/3 Pivotal Confirmatory Clinical Trial of OCU410ST—Modifier Gene Therapy Candidate for Stargardt Disease

Yahoo

time16-06-2025

  • Business
  • Yahoo

Ocugen, Inc. Announces U.S. FDA Clearance of Investigational New Drug Amendment to Initiate Phase 2/3 Pivotal Confirmatory Clinical Trial of OCU410ST—Modifier Gene Therapy Candidate for Stargardt Disease

MALVERN, Pa., June 16, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all Stargardt disease (ABCA4-associated retinopathies). The FDA previously granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. 'We have had a highly productive and collaborative engagement with the FDA's Center for Biologics Evaluation and Research (CBER) in establishing the pivotal confirmatory trial for OCU410ST,' said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. 'It's evident that there is a real sense of urgency by the agency in providing treatment options for patients who currently have nothing available to them. As we initiate the Phase 2/3 registration trial, we are expediting the clinical development of OCU410ST by two to three years and potentially providing an innovative gene therapy to patients desperate for a treatment option.' Positive data from the Phase 1 GARDian trial for OCU410ST demonstrated: A favorable safety and tolerability profile with no serious adverse events related to OCU410ST, including no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization and no adverse events of special interest Considerably slower lesion growth—48% at 12-month follow up in evaluable treated eyes when compared to untreated eyes Statistically significant (p=0.031) improvement with clinically meaningful, nearly 2-line gain in visual function (BCVA) at 12-month follow-up in evaluable treated eyes when compared to untreated eyes The Phase 2/3 clinical trial for OCU410ST will enroll 51 participants diagnosed with Stargardt disease. Of these, 34 will receive a one-time subretinal injection of OCU410ST (200 μL at a concentration of 1.5 × 10¹¹ vector genomes/mL) in the eye with poorer visual acuity, while 17 will be assigned to an untreated control group. The primary objective of the trial is to evaluate the reduction in atrophic lesion size. Key secondary endpoints include improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA), compared to controls. Data from the one-year follow-up will be used to support the company's Biologics License Application (BLA). 'The initiation of this pivotal Phase 2/3 study represents a significant milestone in our commitment to bringing transformative genetic therapies to individuals affected by Stargardt disease—a progressive and debilitating condition,' said Dr. Huma Qamar, Chief Medical Officer at Ocugen. 'The recent RPDD granted by the FDA for this program further underscores the urgent need for innovative treatment options for children living with Stargardt disease. OCU410ST, developed through our proprietary modifier gene therapy platform, is designed to target the underlying biological mechanisms of the disease.' Approximately 100,000 patients in U.S. and Europe combined and 1 million patients globally live with Stargardt disease. Stargardt and ABCA4-associated retinopathies are genetically complex, involving more than 1,200 known mutations and addressing this condition with traditional gene therapy or gene editing approaches remains highly challenging. 'Stargardt disease represents a significant unmet medical need, particularly among children and young adults,' said Lejla Vajzovic, MD, FASRS, Director of the Duke Surgical Vitreoretinal Fellowship Program and Professor of Ophthalmology, Pediatrics, and Biomedical Engineering with Tenure at Duke University Eye Center. 'The Phase 2/3 study of OCU410ST is thoughtfully designed with scientific rigor and a patient-centered focus to evaluate both structural and functional outcomes. We are optimistic that this approach will move us closer to a meaningful therapeutic solution for affected families.' The OCU410ST Phase 2/3 pivotal confirmatory trial represents a major advancement as Ocugen's second late-stage clinical program. Ocugen plans to submit a BLA for OCU410ST in 2027 in alignment with its strategic goal of filing three BLAs over the next three years. About OCU410STOCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR-Related Orphan Receptor A) gene. It represents Ocugen's modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathophysiological pathways linked to Stargardt disease, such as lipofuscin formation, oxidative stress, complement formation, inflammation, and cell survival Stargardt DiseaseStargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula. Decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. Some peripheral vision is usually preserved. Stargardt disease typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in people with Stargardt Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies to address major blindness diseases and offer hope for patients across the globe. We are making an impact on patient's lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Discover more at and follow us on X and LinkedIn. Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as 'predicts,' 'believes,' 'potential,' 'proposed,' 'continue,' 'estimates,' 'anticipates,' 'expects,' 'plans,' 'intends,' 'may,' 'could,' 'might,' 'will,' 'should,' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; the ability of OCU410ST to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled 'Risk Factors' in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release. Contact:Tiffany HamiltonAVP, Head of

SpliceBio Secures $135 Million Series B Financing to Advance Lead Program SB-007 in Stargardt Disease and Expand Pipeline of Genetic Medicines
SpliceBio Secures $135 Million Series B Financing to Advance Lead Program SB-007 in Stargardt Disease and Expand Pipeline of Genetic Medicines

Yahoo

time11-06-2025

  • Business
  • Yahoo

SpliceBio Secures $135 Million Series B Financing to Advance Lead Program SB-007 in Stargardt Disease and Expand Pipeline of Genetic Medicines

Financing co-led by new investors EQT Life Sciences and Sanofi Ventures, with participation from Roche Venture Fund, as well as all existing investors Proceeds will support clinical development of lead program SB-007 in Stargardt disease Funding will also advance a broader pipeline of genetic medicines targeting indications in ophthalmology, neurology, and other undisclosed therapeutic areas BARCELONA, Spain, June 11, 2025 /PRNewswire/ -- SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today announced the close of a $135 million Series B financing co-led by new investors EQT Life Sciences and Sanofi Ventures, with participation from Roche Venture Fund, as well as all existing investors: New Enterprise Associates, UCB Ventures, Ysios Capital, Gilde Healthcare, Novartis Venture Fund, and Asabys Partners. The funding will be used to advance the clinical development of SpliceBio's lead gene therapy candidate, SB-007 for Stargardt disease, including the ongoing interventional Phase 1/2 ASTRA study and the observational POLARIS study. SB-007 is the first dual adeno-associated viral (AAV) gene therapy cleared by the Food and Drug Administration (FDA) to enter clinical development for Stargardt disease. SB-007 has also received regulatory clearance for clinical development from the UK Medicines and Healthcare products Regulatory Agency (MHRA). Stargardt disease is an inherited retinal disorder caused by mutations in the ABCA4 gene that leads to progressive vision loss and blindness, with no approved treatments available. SB-007 is designed to address the underlying genetic cause of the disease by producing a functional copy of the full-length ABCA4 protein with the potential to treat all patients, regardless of their specific ABCA4 mutation. The proceeds will also be used to accelerate SpliceBio's pipeline of AAV gene therapy programs in ophthalmology, neurology, and other undisclosed indications that utilise the company's proprietary Protein Splicing platform. "This financing marks a pivotal milestone for SpliceBio as we advance the clinical development of SB-007 for Stargardt disease and continue to expand our pipeline across ophthalmology, neurology and beyond," said Miquel Vila-Perelló, Ph.D., Chief Executive Officer and Co-Founder of SpliceBio. "The support from such high-quality investors underscores the strength of our programs and our unique Protein Splicing platform and its potential to unlock gene therapies for diseases that remain untreatable today. We are building a company positioned to lead the next wave of genetic medicines." SpliceBio is redefining and expanding the scope of diseases that can be tackled with gene therapies by addressing a fundamental limitation of AAV vectors in their inability to deliver genes that exceed their limited packaging capacity of 4.7 kilobases. Many genetic disorders remain untreatable because the necessary gene is too large to fit into the AAV vectors. SpliceBio's unique Protein Splicing platform leverages the use of a family of proprietary, engineered proteins called inteins, originally developed at Princeton University. The company's technology enables the splitting of the gene into two (or more) transgenes that are then delivered using dual AAV vectors. Once inside the cell, the DNA of each transgene is transcribed into messenger RNA and translated into protein. SpliceBio's engineered inteins are designed to then assemble the full-length protein that is needed to treat the disease. Daniela Begolo, Managing Director at EQT Life Sciences, commented: "We are proud to support SpliceBio, a pioneer among the next-generation of genetic medicine companies. Its Protein Splicing platform is designed to offer a novel solution to deliver large genes with AAV, one of the field's most pressing challenges, and exemplifies our commitment to backing transformational science that can meaningfully benefit patients' lives." Laia Crespo, Partner at Sanofi Ventures, remarked: "With compelling data for its lead program, SB-007, and a highly differentiated platform, we are excited to support SpliceBio as it tackles a fundamental challenge for genetic medicines. By enabling the delivery of large and complex genes through its novel AAV vector Protein Splicing technology, SpliceBio has the potential to make a significant impact on the field of gene therapy and to deliver best-in-class therapies to patients." Carole Nuechterlein, Head of Roche Venture Fund, added: "We are impressed by the team's strong execution, the momentum behind SB-007 in Stargardt disease, and the platform's potential to unlock a new class of genetic medicines. We are proud to support SpliceBio at this pivotal stage of growth as they advance their lead program through clinical development and explore additional high-impact indications." In connection with the financing, Daniela Begolo, Managing Director at EQT Life Sciences, Laia Crespo, Partner at Sanofi Ventures, and Carole Nuechterlein, Head of Roche Venture Fund, will join the SpliceBio Board of Directors. About SpliceBioSpliceBio is a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes. The Company's lead program, SB-007, targets the root cause of Stargardt disease, a genetic eye disease that causes blindness in children and adults. SpliceBio's pipeline comprises additional gene therapy programs across therapeutic areas, including ophthalmology and neurology. SpliceBio's platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein, Protein Splicing, and protein engineering research. For additional information, please visit About SB-007SB-007 is an investigational Protein Splicing dual AAV gene therapy in development for the treatment of Stargardt disease. It is designed to restore expression of the native full-length ABCA4 protein in the retina. SB-007 has been granted Orphan Drug Designation from both the FDA in the US and the European Commission in Europe. In December 2024, SB-007 received FDA IND clearance, marking the first-ever clearance for a dual AAV gene therapy in Stargardt disease. Alongside initiation of the Phase 1/2 ASTRA study, with the announcement of the first patient dosed in March 2025, SpliceBio continues to advance POLARIS, a natural history study of the disease. Both studies are actively recruiting. For more information or to enquire about participation in the studies, please visit About EQT Life Sciences EQT Life Sciences was formed in 2022 following an integration of LSP, a leading European life sciences and healthcare venture capital firm, into the EQT platform. As LSP, the firm raised over EUR 3.0 billion (USD 3.5 billion) and supported the growth of more than 150 companies since it started to invest over 30 years ago. With a dedicated team of highly experienced investment professionals, coming from backgrounds in medicine, science, business, and finance, EQT Life Sciences backs the smartest inventors who have ideas that could truly make a difference for patients. More info: Follow EQT on LinkedIn, Twitter, YouTube and Instagram. About Sanofi VenturesSanofi Ventures is the corporate venture capital arm of Sanofi, focused on investing in promising early-stage healthcare companies. The firm supports pioneering innovations in biotechnology, digital health, and life sciences aligning with Sanofi's mission to bring life-changing treatments to patients worldwide. For more information visit: About Roche Venture FundThe Roche Venture Fund is the corporate venture fund of Roche and invests in innovative life science companies. Over the past 20 years, the Roche Venture Fund has invested in over 60 companies globally and currently has a portfolio of around 30 companies located in 10 countries. As part of a multinational healthcare company, the Roche Venture Fund has access to considerable expertise both internally and externally and co-invests with leading venture funds, including other corporate venture funds, on a regular View original content: SOURCE SpliceBio 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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