Prevail Therapeutics
Develops gene- and RNA-based therapeutics targeting genetic drivers of neurodegenerative and rare diseases, with clinical programs focused on Parkinson’s disease (GBA1-linked) and an siRNA program targeting α-synuclein. Conducts preclinical through clinical-stage development and runs multi-site clinical trials with CNS-directed delivery and longitudinal safety and biomarker monitoring.
Industries
Nr. of Employees
medium (51-250)
Prevail Therapeutics
New York, New York, United States
Patents
Recombinant adeno-associated virus compositions and methods for producing same
US-12729385-B2
View Details
Recombinant adeno-associated virus compositions and methods for producing same
US-12729385-B2
View DetailsProducts
Gene therapy clinical trial for Parkinson’s disease linked to GBA1 variants
A clinical development program testing a one-time CNS-delivered gene therapy intended to deliver a functional copy of the GBA1 gene to slow progression in people with Parkinson’s disease who carry GBA1 variants. Protocol includes cisterna magna administration under anesthesia, peri-procedural immunosuppression, longitudinal follow-up to 5 years, and biomarker sampling.
siRNA therapy clinical trial for sporadic Parkinson’s disease
A first‑in‑human program testing an siRNA therapeutic designed to lower α-synuclein levels. The program uses intrathecal (lower spine) administration, includes randomized placebo-controlled cohorts, staged dose escalation across up to five dose levels, and short- and medium-term follow-up with PK/PD and biomarker assessments.
PR001
An AAV9 vector-based gene therapy designed to deliver a functional GBA1 gene to the brain to treat Parkinson's disease associated with GBA1 mutations.
Gene therapy clinical trial for Parkinson’s disease linked to GBA1 variants
A clinical development program testing a one-time CNS-delivered gene therapy intended to deliver a functional copy of the GBA1 gene to slow progression in people with Parkinson’s disease who carry GBA1 variants. Protocol includes cisterna magna administration under anesthesia, peri-procedural immunosuppression, longitudinal follow-up to 5 years, and biomarker sampling.
siRNA therapy clinical trial for sporadic Parkinson’s disease
A first‑in‑human program testing an siRNA therapeutic designed to lower α-synuclein levels. The program uses intrathecal (lower spine) administration, includes randomized placebo-controlled cohorts, staged dose escalation across up to five dose levels, and short- and medium-term follow-up with PK/PD and biomarker assessments.
PR001
An AAV9 vector-based gene therapy designed to deliver a functional GBA1 gene to the brain to treat Parkinson's disease associated with GBA1 mutations.
Services
Patient inquiry handling, eligibility screening workflows, and coordination with genetic testing initiatives to identify trial candidates.
Management of multi-site clinical operations including screening, dosing, monitoring, and long-term follow-up visits.
Patient inquiry handling, eligibility screening workflows, and coordination with genetic testing initiatives to identify trial candidates.
Management of multi-site clinical operations including screening, dosing, monitoring, and long-term follow-up visits.
Expertise Areas
- Gene therapy development
- siRNA therapeutic development
- Clinical trial management and operations
- CNS delivery for therapeutics
Key Technologies
- Gene therapy
- siRNA therapeutics
- Cisterna magna injection
- Intrathecal injection