Gaucher Disease, Type 1

CAP-003 achieved best-in-class GCase increases in NHPs exceeding levels needed for normalizing activity in patients Brainwide RNA expression greater than 200-fold higher than AAV9, with significant detargeting of liver and DRGs with proprietary engineered capsid Company on track to file IND in first half of 2025 THOUSAND OAKS, CA, USA I October 7, 2024 I Capsida Biotherapeutics (“Capsida”) presented new IND-enabling data on its intravenously (IV) delivered gene therapy for Parkinson’s disease associated with GBA mutations (PD-GBA), CAP-003, that demonstrate its substantial therapeutic potential. Capsida’s next-generation therapy, using a proprietary engineered capsid, demonstrates best-in-class potential to effectively and safely treat PD-GBA. CAP-003 achieves unprecedented levels of GCase protein and activity while simultaneously detargeting the liver and dorsal root ganglion (DRGs). These data were presented in a late-breaking poster presentation at the Society for Neuroscience “Neuroscience 2024” annual meeting being held October 5-9, 2024 in Chicago, IL. Mutations in GBA, the gene expressing the GCase enzyme, affect up to 15% of PD patients and are the most common genetic risk factor for PD. Post-mortem studies demonstrate an approximate 30% GCase activity deficit in patients 1 . Other potential treatments for PD-GBA have been limited by their inability to cross the blood-brain barrier and supplement GCase enzyme activity in sufficient quantities to overcome the deficit in patients and impact the disease. In an attempt to overcome these challenges, they have utilized invasive direct brain or cerebrospinal fluid administration, with limited results, but significant burden for patients. Results from Capsida’s IND-enabling non-human primate (NHP) studies show brainwide RNA expression more than 200-fold greater than IV administered AAV9 and substantial increases in GCase protein and enzyme activity compared to untreated animals. A single IV infusion of CAP-003 resulted in up to six-fold increase in GCase protein compared to untreated animals in relevant brain regions. Across doses and brain regions, including the substantia nigra, the levels of GCase activity following infusion of CAP-003 was increased by up to approximately 250% compared to untreated animals. These data support the potential for meaningful clinical benefit in patients. Increases of 30% are expected to normalize GCase activity in patients with PD-GBA. NHPs dosed with CAP-003 IV have 19- and 17-fold less biodistribution to the liver and DRG, respectively, compared to IV administered wild-type AAV9. CAP-003 is well tolerated with no notable safety findings in NHPs. “Capsida’s wholly owned novel gene therapy offers the potential to normalize GCase activity in patients with a single IV infusion safely, enabling the potential for long-term disease modification and substantial slowing of disease progression,” said Peter Anastasiou, Capsida’s Chief Executive Officer. “These data give us confidence that we are on track to enter the clinic with CAP-003 in the first half of 2025.” Capsida to Present at Cell & Gene Meeting on the Mesa In addition, Capsida will present these data as part of a corporate presentation at the Cell & Gene Meeting on the Mesa taking place October 7-9, 2024, in Phoenix, AZ. Mr. Anastasiou will present a corporate update on Tuesday, October 8, 2024 at 3:45 PM local time. The Capsida corporate presentation can be accessed by visiting the News section of the Company’s website at www.capsida.com . About Capsida Biotherapeutics Capsida Biotherapeutics is a fully integrated gene therapy company with a central nervous system (CNS) pipeline consisting of disease modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida’s intravenously (IV) administered gene therapies utilize proprietary engineered capsids that enable high transduction levels to desired tissues and cells, while limiting tropism to non-target organs, such as the liver. Capsida has three wholly owned programs, including potential best-in-class treatments for genetic epilepsy due to STXBP1 mutations and Parkinson’s disease associated with GBA mutations, both of which are in IND-enabling studies and on track to enter the clinic in the first half of 2025. In addition to its wholly owned programs, the Company has validating CNS partnerships with AbbVie, Lilly, CRISPR Therapeutics, and the AbbVie partnership was expanded to include ophthalmology disorders. Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. Visit us at www.capsida.com . 1 Gegg, M. E, et al (2012). Annals of neurology, 72(3), 455-463. SOURCE: Capsida Biotherapeutics

CAP-003 achieved best-in-class GCase increases in NHPs exceeding levels needed for normalizing activity in patients Brainwide RNA expression greater than 200-fold higher than AAV9, with significant detargeting of liver and DRGs with proprietary engineered capsid Company on track to file IND in first half of 2025 THOUSAND OAKS, Calif., Oct. 7, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") presented new IND-enabling data on its intravenously (IV) delivered gene therapy for Parkinson's disease associated with GBA mutations (PD-GBA), CAP-003, that demonstrate its substantial therapeutic potential. Capsida's next-generation therapy, using a proprietary engineered capsid, demonstrates best-in-class potential to effectively and safely treat PD-GBA. CAP-003 achieves unprecedented levels of GCase protein and activity while simultaneously detargeting the liver and dorsal root ganglion (DRGs). These data were presented in a late-breaking poster presentation at the Society for Neuroscience "Neuroscience 2024" annual meeting being held October 5-9, 2024 in Chicago, IL. Mutations in GBA, the gene expressing the GCase enzyme, affect up to 15% of PD patients and are the most common genetic risk factor for PD. Post-mortem studies demonstrate an approximate 30% GCase activity deficit in patients1. Other potential treatments for PD-GBA have been limited by their inability to cross the blood-brain barrier and supplement GCase enzyme activity in sufficient quantities to overcome the deficit in patients and impact the disease. In an attempt to overcome these challenges, they have utilized invasive direct brain or cerebrospinal fluid administration, with limited results, but significant burden for patients. Results from Capsida's IND-enabling non-human primate (NHP) studies show brainwide RNA expression more than 200-fold greater than IV administered AAV9 and substantial increases in GCase protein and enzyme activity compared to untreated animals. A single IV infusion of CAP-003 resulted in up to six-fold increase in GCase protein compared to untreated animals in relevant brain regions. Across doses and brain regions, including the substantia nigra, the levels of GCase activity following infusion of CAP-003 was increased by up to approximately 250% compared to untreated animals. These data support the potential for meaningful clinical benefit in patients. Increases of 30% are expected to normalize GCase activity in patients with PD-GBA. NHPs dosed with CAP-003 IV have 19- and 17-fold less biodistribution to the liver and DRG, respectively, compared to IV administered wild-type AAV9. CAP-003 is well tolerated with no notable safety findings in NHPs. "Capsida's wholly owned novel gene therapy offers the potential to normalize GCase activity in patients with a single IV infusion safely, enabling the potential for long-term disease modification and substantial slowing of disease progression," said Peter Anastasiou, Capsida's Chief Executive Officer. "These data give us confidence that we are on track to enter the clinic with CAP-003 in the first half of 2025." Capsida to Present at Cell & Gene Meeting on the Mesa In addition, Capsida will present these data as part of a corporate presentation at the Cell & Gene Meeting on the Mesa taking place October 7-9, 2024, in Phoenix, AZ. Mr. Anastasiou will present a corporate update on Tuesday, October 8, 2024 at 3:45 PM local time. The Capsida corporate presentation can be accessed by visiting the News section of the Company's website at . About Capsida Biotherapeutics Capsida Biotherapeutics is a fully integrated gene therapy company with a central nervous system (CNS) pipeline consisting of disease modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida's intravenously (IV) administered gene therapies utilize proprietary engineered capsids that enable high transduction levels to desired tissues and cells, while limiting tropism to non-target organs, such as the liver. Capsida has three wholly owned programs, including potential best-in-class treatments for genetic epilepsy due to STXBP1 mutations and Parkinson's disease associated with GBA mutations, both of which are in IND-enabling studies and on track to enter the clinic in the first half of 2025. In addition to its wholly owned programs, the Company has validating CNS partnerships with AbbVie, Lilly, CRISPR Therapeutics, and the AbbVie partnership was expanded to include ophthalmology disorders. Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. Visit us at . 1Gegg, M. E, et al (2012). Annals of neurology, 72(3), 455-463. SOURCE Capsida Biotherapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

LONDON, Oct. 03, 2024 (GLOBE NEWSWIRE) -- Spur Therapeutics, formerly Freeline Therapeutics, today announced that it will present updated clinical data from its GALILEO-1 first-in-human clinical trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease Type 1, at the European Society of Gene & Cell Therapy (ESGCT) 31st Annual Congress being held October 22-25, 2024, in Rome, Italy. Details of the oral presentation are below: Abstract Title:Results from GALILEO-1, a First-In-Human Clinical Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1Session:3A – AAV Vectors as Tools in Gene Therapy of Rare Diseases - Recent Development to Improve Efficacy and SafetyDate & Time:Wednesday, October 23 from 08:30-10:30 a.m. CEST (02:30-04:30 a.m. ET)Presenter:Francesca Ferrante, MD, Spur Therapeutics Additional details on the meeting can be found at the ESGCT 31st Annual Congress website. About Spur TherapeuticsSpur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions. By optimizing every component of its product candidates, Spur aims to unlock the true potential of gene therapy to realize outsized clinical results.Spur is advancing a breakthrough gene therapy candidate for Gaucher disease and a potential first-in-class gene therapy candidate for adrenomyeloneuropathy, as well as a research strategy to move gene therapy into more prevalent diseases, including forms of Parkinson’s, dementia, and cardiovascular disease. Expanding our impact, and advancing the practice of genetic medicine. Toward life-changing therapies, and brighter futures. Toward More™ For more information, visit www.spurtherapeutics.com or connect with Spur on LinkedIn and X. ContactNaomi Aokinaomi.aoki@spurtherapeutics.com + 1 617 283 4298