Latest news with #AAV9
Yahoo
3 days ago
- Business
- Yahoo
William Blair Reaffirms Outperform Rating on Rocket Pharma Despite Trial Setback
Sami Corwin, an analyst at William Blair, maintained his Outperform rating on Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) on May 27 with no price target, despite the company's recent difficulties. The biotechnology company's trial for the gene therapy medicine RP-A501 was clinically halted and a patient tragically passed away. Corwin stated that the delay was required due to the capillary leak syndrome (CLS) safety event, which had never been observed with RP-A501 or comparable treatments before. Rather than the AAV9 vector utilized in the treatment, Corwin expressed hope that the serious adverse event could be related to the recent addition of a C3 inhibitor to the immunosuppressive protocol. Only individuals who received the C3 inhibitor had CLS cases documented, the analyst said. Although the occurrences of thrombotic microangiopathy (TMA) are alarming, the analyst also pointed out that the afflicted patients are making a full recovery. Despite the RCKT stock's price tanking by around 80% year-to-date, Corwin thinks that investors are primarily concerned about the safety of the program and when the clinical hold would be lifted. He went on to say that Rocket's other clinical cardiovascular program, RP-A601, which employs a different AAV vector and does not include the C3 inhibitor, remains unaffected, implying that the recent occurrences shouldn't impact how this distinct program is viewed. While we acknowledge the potential of RCKT to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than RCKT and that has 100x upside potential, check out our report about the cheapest AI stock. Read More: and . Disclosure: None. 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
Yahoo
3 days ago
- Business
- Yahoo
William Blair Reaffirms Outperform Rating on Rocket Pharma Despite Trial Setback
Sami Corwin, an analyst at William Blair, maintained his Outperform rating on Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) on May 27 with no price target, despite the company's recent difficulties. The biotechnology company's trial for the gene therapy medicine RP-A501 was clinically halted and a patient tragically passed away. Corwin stated that the delay was required due to the capillary leak syndrome (CLS) safety event, which had never been observed with RP-A501 or comparable treatments before. Rather than the AAV9 vector utilized in the treatment, Corwin expressed hope that the serious adverse event could be related to the recent addition of a C3 inhibitor to the immunosuppressive protocol. Only individuals who received the C3 inhibitor had CLS cases documented, the analyst said. Although the occurrences of thrombotic microangiopathy (TMA) are alarming, the analyst also pointed out that the afflicted patients are making a full recovery. Despite the RCKT stock's price tanking by around 80% year-to-date, Corwin thinks that investors are primarily concerned about the safety of the program and when the clinical hold would be lifted. He went on to say that Rocket's other clinical cardiovascular program, RP-A601, which employs a different AAV vector and does not include the C3 inhibitor, remains unaffected, implying that the recent occurrences shouldn't impact how this distinct program is viewed. While we acknowledge the potential of RCKT to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than RCKT and that has 100x upside potential, check out our report about the cheapest AI stock. Read More: and . Disclosure: None. 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
Hans India
23-05-2025
- Health
- Hans India
New gene therapy to target airway and lungs via nasal spray
US researchers have engineered a novel gene therapy to target the airway and lungs via a nasal spray. For gene therapy to work well, therapeutic molecules need to be efficiently delivered to the correct locations in the body. It is commonly done by using adeno-associated viruses (AAV) gene therapy. To improve the AAV's ability to deliver therapeutics specifically to the lungs and airway, researchers at the Mass General Brigham engineered a new version, called which can be administered with a nasal spray. In preclinical models, outperformed previous versions by more effectively targeting the airway and lungs and showing promise for respiratory and lung gene therapy, said the researchers in the paper published in the journal Cell Reports Medicine. "We noticed that which we initially engineered to enter the central nervous system, also efficiently targeted lung cells," said senior author FengFeng Bei, from the Department of Neurosurgery at Brigham and Women's Hospital. "This prompted us to further investigate for intranasal gene delivery to the respiratory airways," Bei added. In the study, outperformed previous versions (AAV6 and AAV9) in cell culture, mouse models, and non-human primate models. 'Our findings highlight as a promising vector for respiratory and lung gene therapy,' the team said. They then used the more efficient tool to deliver scar-preventing gene therapy for pulmonary fibrosis, using a mouse model of the respiratory disease. They also used the tool to deliver gene therapy for a viral infection, where the therapy prevented the replication of the SARS-CoV-2 virus in a mouse model of Covid-19. "Although further research is needed, our findings suggest that intranasal has strong translational potential as a promising delivery tool for targeting the airway and lung," said Bei.
Business Wire
14-05-2025
- Business
- Business Wire
Capsida Presents New GLP Toxicology Data Supporting Recent FDA IND Clearance of Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Capsida Biotherapeutics ('Capsida') today announced the presentation of new non-human primate (NHP) GLP toxicology data for CAP-002, its wholly owned first-in-class, intravenously (IV) administered investigational gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). These data were the foundation for the Investigational New Drug (IND) application for CAP-002 that was recently cleared by the U.S. Food and Drug Administration (FDA). Capsida will deliver these data in an oral presentation today at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place May 13-17, 2025, in New Orleans and virtually. The three-month cohort data from the NHP GLP toxicology study demonstrate dose-dependent brain-wide expression of STXBP1 and simultaneous detargeting of the liver and dorsal root ganglia (DRGs). CAP-002 was delivered as a single dose via IV infusion at 3.8E13 vg/kg, 5.9E13 vg/kg, and 7.4E13 vg/kg. There was a dose-dependent increase in average cargo RNA levels in the brain, with the 3.8E13 vg/kg dose of CAP-002 demonstrating a 207-fold increase over cargo RNA levels previously shown for 2.5E13 vg/kg of IV-delivered adeno-associated virus 9 (AAV9). Together with pharmacology data in a mouse model of STXBP1-DEE, these NHP GLP toxicology results show that all doses of CAP-002 tested have the potential to fully correct seizures and meaningfully correct cognitive and motor dysfunction in patients. The 3.8E13 vg/kg dose of CAP-002 also demonstrated robust 10x and 13x detargeting of liver and DRGs respectively, compared to a 2.5E13 vg/kg dose of AAV9. CAP-002 was well tolerated with no adverse clinical pathology or histopathology findings throughout the central nervous system, DRGs, or peripheral organs, including the liver. CAP-002 is the first IV-administered, AAV blood brain barrier-crossing genetic medicine program entering a human clinical trial. CAP-002 is manufactured in Capsida's state-of-the-art wholly owned facility using a proprietary manufacturing process. Capsida received FDA Orphan Drug Designation in October 2024 and is now initiating study start-up activities for the SYNRGY Phase 1/2a clinical trial, with the first patient expected to be dosed in the third quarter of this year. 'These GLP toxicology data established the potential of CAP-002 to safely treat STXBP1-DEE and were a critical part of the data package to enable the FDA IND clearance of this first-in-class IV investigational gene therapy,' said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. 'Our focus is on initiating our Phase 1/2a SYNRGY clinical trial with the aim of providing a safe, disease-modifying treatment for STXBP1-DEE patients and their families.' Presentation Details Oral Presentation: Title: Systemic Gene Therapy CAP-002 Demonstrates Potential for Disease-Modifying Treatment of Seizures and Motor and Cognitive Deficits of STXBP1-DEE Using an Engineered, CNS-Targeted AAV Date and Time: Session: Viral Vectors in Large Animal Models Location: New Orleans Theater B Presenter: Nicholas Flytzanis, Ph.D., Founder, Chief Research and Innovation Officer, Capsida Abstracts can be found at and the presentation will be available under the 'Publications' section of the Capsida website. About STXBP1-DEE Syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE) is estimated to affect up to one in 26,000 births globally, equating to approximately 5,000 pediatric patients in the U.S. and Europe. The STXBP1 protein is present in every neuron and is essential for normal neurotransmission. Mutations in the STXBP1 gene are associated with early-onset seizures, severe developmental delay and intellectual disability, motor abnormalities, and a risk of sudden unexpected death in epilepsy (SUDEP). There are no approved treatments for STXBP1-DEE. About Capsida Biotherapeutics Capsida Biotherapeutics is a fully integrated next-generation genetic medicines 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 wholly owned pipeline includes its first-in-class treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), which has received Investigational New Drug (IND) clearance to initiate clinical trials from the U.S. Food and Drug Administration (FDA). Capsida's pipeline also includes potential best-in-class treatments for Parkinson's disease associated with GBA mutations (PD-GBA) and Friedreich's ataxia (FA). The PD-GBA program is also on track to enter clinical development this quarter. In addition to its wholly owned programs, the Company has validating partnerships with AbbVie, Lilly, and CRISPR Therapeutics. 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
Associated Press
14-05-2025
- Business
- Associated Press
Capsida Presents New GLP Toxicology Data Supporting Recent FDA IND Clearance of Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--May 14, 2025-- Capsida Biotherapeutics ('Capsida') today announced the presentation of new non-human primate (NHP) GLP toxicology data for CAP-002, its wholly owned first-in-class, intravenously (IV) administered investigational gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE). These data were the foundation for the Investigational New Drug (IND) application for CAP-002 that was recently cleared by the U.S. Food and Drug Administration (FDA). Capsida will deliver these data in an oral presentation today at the 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), taking place May 13-17, 2025, in New Orleans and virtually. The three-month cohort data from the NHP GLP toxicology study demonstrate dose-dependent brain-wide expression of STXBP1 and simultaneous detargeting of the liver and dorsal root ganglia (DRGs). CAP-002 was delivered as a single dose via IV infusion at 3.8E13 vg/kg, 5.9E13 vg/kg, and 7.4E13 vg/kg. There was a dose-dependent increase in average cargo RNA levels in the brain, with the 3.8E13 vg/kg dose of CAP-002 demonstrating a 207-fold increase over cargo RNA levels previously shown for 2.5E13 vg/kg of IV-delivered adeno-associated virus 9 (AAV9). Together with pharmacology data in a mouse model of STXBP1-DEE, these NHP GLP toxicology results show that all doses of CAP-002 tested have the potential to fully correct seizures and meaningfully correct cognitive and motor dysfunction in patients. The 3.8E13 vg/kg dose of CAP-002 also demonstrated robust 10x and 13x detargeting of liver and DRGs respectively, compared to a 2.5E13 vg/kg dose of AAV9. CAP-002 was well tolerated with no adverse clinical pathology or histopathology findings throughout the central nervous system, DRGs, or peripheral organs, including the liver. CAP-002 is the first IV-administered, AAV blood brain barrier-crossing genetic medicine program entering a human clinical trial. CAP-002 is manufactured in Capsida's state-of-the-art wholly owned facility using a proprietary manufacturing process. Capsida received FDA Orphan Drug Designation in October 2024 and is now initiating study start-up activities for the SYNRGY Phase 1/2a clinical trial, with the first patient expected to be dosed in the third quarter of this year. 'These GLP toxicology data established the potential of CAP-002 to safely treat STXBP1-DEE and were a critical part of the data package to enable the FDA IND clearance of this first-in-class IV investigational gene therapy,' said Swati Tole, M.D., Chief Medical Officer of Capsida Biotherapeutics. 'Our focus is on initiating our Phase 1/2a SYNRGY clinical trial with the aim of providing a safe, disease-modifying treatment for STXBP1-DEE patients and their families.' Presentation Details Oral Presentation: Abstracts can be found at and the presentation will be available under the 'Publications' section of the Capsida website. About STXBP1-DEE Syntaxin-binding protein 1 developmental and epileptic encephalopathy (STXBP1-DEE) is estimated to affect up to one in 26,000 births globally, equating to approximately 5,000 pediatric patients in the U.S. and Europe. The STXBP1 protein is present in every neuron and is essential for normal neurotransmission. Mutations in the STXBP1 gene are associated with early-onset seizures, severe developmental delay and intellectual disability, motor abnormalities, and a risk of sudden unexpected death in epilepsy (SUDEP). There are no approved treatments for STXBP1-DEE. About Capsida Biotherapeutics Capsida Biotherapeutics is a fully integrated next-generation genetic medicines 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 wholly owned pipeline includes its first-in-class treatment for STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), which has received Investigational New Drug (IND) clearance to initiate clinical trials from the U.S. Food and Drug Administration (FDA). Capsida's pipeline also includes potential best-in-class treatments for Parkinson's disease associated with GBA mutations (PD-GBA) and Friedreich's ataxia (FA). The PD-GBA program is also on track to enter clinical development this quarter. In addition to its wholly owned programs, the Company has validating partnerships with AbbVie, Lilly, and CRISPR Therapeutics. 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 View source version on CONTACT: Media Contact Greig Communications, Inc. Kathy Vincent [email protected] KEYWORD: UNITED STATES NORTH AMERICA CALIFORNIA LOUISIANA INDUSTRY KEYWORD: HEALTH GENETICS CLINICAL TRIALS RESEARCH SCIENCE PHARMACEUTICAL BIOTECHNOLOGY SOURCE: Capsida Biotherapeutics Copyright Business Wire 2025. PUB: 05/14/2025 07:45 AM/DISC: 05/14/2025 07:44 AM
