Capsida Biotherapeutics, (“Capsida”) a next-generation genetic medicines company, today provided corporate and pipeline updates related to its ocular and central nervous system (CNS) programs.
Capsida’s Next Generation Ocular Capsids Presented at ASGCT
Capsida will present a poster highlighting high-performing ocular capsids delivered by intravitreal (IVT) and suprachoroidal (SCS) administration (Abstract 2031) at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, taking place May 11-15, 2026, in Boston, Massachusetts. Traditional gene therapy for ocular diseases has been hampered by the procedural burden of subretinal surgery, as well as physiological and physical barriers that prevent efficient transduction of retinal cell types. Capsida’s engineered ocular capsids aim to deliver best in class therapies by improving expression profiles through less invasive IVT and SCS administration. In the data presented at ASGCT, these next-generation IVT and SCS capsids showed industry leading retinal distribution and expression compared with existing serotypes and clinical capsids. These capsids have the potential for an improved efficacy and safety profile achieved through lower dosing. Capsida is advancing these wholly owned ophthalmology programs towards the clinic.
Romuald “Romu” Corbau, Ph.D., Appointed as Chief Scientific Officer
To support this program as well as its CNS portfolio, Romu Corbau has joined Capsida as Chief Scientific Officer. Dr. Corbau has over 27 years of experience in gene therapy and drug discovery across startups and global organizations in the United States and Europe. He was most recently the Chief Scientific Officer at Adverum prior to its sale to Lilly in 2025, where he led second- and third-generation ixo-vec programs and broader ocular pipeline expansion. Prior to Adverum, Dr. Corbau was the Chief Scientific Officer of GenEdit (now BreezeBio) and previously Freeline (now Spur Therapeutics), where he advanced AAV programs for hemophilia B and Fabry disease into the clinic, and initiated the Gaucher disease program. Previously, Dr. Corbau led the translational R&D at Spark Therapeutics for Luxturna, a subretinal injection gene therapy for Leber’s congenital amaurosis and the first FDA-approved gene therapy for an inherited disease. While at Spark Therapeutics, Dr. Corbau held a concurrent appointment at the Children’s Hospital of Philadelphia. Prior to Spark, he was at CISTIM in Belgium, and earlier spent 13 years at Pfizer in the United Kingdom.
“Capsida has developed an impressive capsid engineering platform demonstrating clear progress in ophthalmology,” said Dr. Corbau. “The ability to achieve broad retinal distribution and expression through less invasive delivery approaches has been a well-known challenge for the field. The data being presented at ASGCT highlight the potential of these next-generation capsids to address those limitations, and I’m excited to help translate that into meaningful therapies for patients.”
Update on CNS Programs
Capsida recently shared an update directly to The STXBP1 Foundation regarding its ongoing investigation of the patient enrolled in the CAP-002 SYNRGY trial who passed away in September 2025 due to cerebral edema. In that communication, Capsida disclosed the closure of the trial. Capsida will also be closing the trial for CAP-003 in Parkinson’s disease associated with GBA mutations (PD-GBA) which used the same capsid. No patients were dosed in this trial.
As previously reported, the autopsy report did not identify the underlying cause of cerebral edema. Without access to tissue samples, the Company started additional preclinical work to investigate further. The ongoing experiments include studying the interaction between the engineered viral capsid contained in CAP-002 and the ADAM15 protein, its primary receptor for crossing the blood brain barrier (BBB). Based on the learning over the past six months, Capsida believes that the receptor is a key factor in designing CNS gene therapy.
Over the last seven years, Capsida’s platform has enabled the creation of a large library of novel capsids to cross the BBB with intravenous delivery, and capsids for ocular delivery. Drawing from this pool, Capsida is moving forward development of a next-generation capsid, which uses a distinct receptor, RECK, to cross the BBB. While these experiments are on-going, early data supports continued development of this new capsid for CNS gene therapies. Capsida plans to report data from the preclinical evaluation in an upcoming scientific forum.
“While the decision to close the SYNRGY trial was incredibly difficult, we remain committed to understanding the events observed in that study and to developing gene therapies for patients with CNS disorders,” said Mina Kim, Interim Chief Executive Officer of Capsida.
“The insights we are gaining are actively shaping our next-generation approach to IV delivery of CNS gene therapy. We are making progress with a new capsid that uses a distinct receptor, RECK, to cross the blood-brain barrier, and while still early, we are encouraged by the emerging data,” added Nicholas Flytzanis, Ph.D., Co-founder and Chief Research and Innovation Officer.
Capsida’s Poster Presentations at ASGCT 2026
Identification of high-performing ocular AAV capsids through directed engineering across intravitreal and suprachoroidal delivery routes
Date and Time: Wednesday, May 13 at 5:00-6:30 pm ET
Abstract Number: 2031
Presenter: Celeste Stephany, Ph.D., Executive Director of CNS and Ophthalmology Preclinical Research, Capsida
Improvements to the manufacturing process for engineered AAV9 viral vectors, increasing yields and robustness with performance scaling to a 200L production train
Date and Time: Thursday, May 14 at 5:00-6:30 pm ET
Abstract Number: 3233
Presenter: Hari Acharya, Ph.D., Executive Director of Process and Analytical Development, Capsida
About Capsida Biotherapeutics
Capsida Biotherapeutics is a next-generation genetic medicines company focused on rare and more common neurological and ocular diseases across all ages. Capsida’s wholly owned pipeline includes intravenously (IV) delivered gene therapies for the CNS that have broad, brain-wide neuronal expression, as well as engineered capsids with improved expression profiles through less invasive intravitreal (IVT) and suprachoroidal (SCS) administration for ocular diseases. 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.
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