Prevail Therapeutics
Developer of genetic medicines for neurodegenerative and rare diseases. Operates multiple genotype-directed clinical trials including CNS-directed and systemic gene therapies and an siRNA program for Parkinson’s disease, frontotemporal dementia, and Type 1 Gaucher disease. The site identifies the company as a wholly owned subsidiary of a larger pharmaceutical company.
Industries
Nr. of Employees
medium (51-250)
Prevail Therapeutics
New York, New York, United States, North America
Products
CNS-directed gene therapy clinical trial for Parkinson’s disease (GBA1 variants)
Genotype-directed clinical study delivering a one-time CNS administration of gene replacement material via cisterna magna injection for participants with Parkinson’s disease carrying GBA1 variants.
CNS-directed gene therapy clinical trial for frontotemporal dementia (GRN variants)
Single administration gene therapy administered into the cisterna magna intended to restore progranulin expression in participants with FTD associated with GRN variants; includes staged dose levels and long-term follow-up.
Systemic gene therapy clinical trial for Type 1 Gaucher disease
One-time intravenous infusion of a gene therapy product delivered via peripheral vein catheter to increase enzyme levels for Type 1 Gaucher disease; includes staged dose escalation and monitoring with some home visit options.
Intrathecal siRNA randomized clinical trial for sporadic Parkinson’s disease
Placebo-controlled, randomized study testing intrathecal siRNA injections to reduce alpha-synuclein levels, using a two-dose schedule across multiple dose levels and multinational sites.
CNS-directed gene therapy clinical trial for Parkinson’s disease (GBA1 variants)
Genotype-directed clinical study delivering a one-time CNS administration of gene replacement material via cisterna magna injection for participants with Parkinson’s disease carrying GBA1 variants.
CNS-directed gene therapy clinical trial for frontotemporal dementia (GRN variants)
Single administration gene therapy administered into the cisterna magna intended to restore progranulin expression in participants with FTD associated with GRN variants; includes staged dose levels and long-term follow-up.
Systemic gene therapy clinical trial for Type 1 Gaucher disease
One-time intravenous infusion of a gene therapy product delivered via peripheral vein catheter to increase enzyme levels for Type 1 Gaucher disease; includes staged dose escalation and monitoring with some home visit options.
Intrathecal siRNA randomized clinical trial for sporadic Parkinson’s disease
Placebo-controlled, randomized study testing intrathecal siRNA injections to reduce alpha-synuclein levels, using a two-dose schedule across multiple dose levels and multinational sites.
Services
Expanded access / compassionate use request review
Individual requests for investigational therapy access are reviewed under a published policy; requests must be submitted in writing by a licensed treating physician.
Patient engagement and genetic testing facilitation
Coordination with external genetic research programs and patient advocacy partners to provide free at-home genetic testing kits, counseling, and referrals for genotype-specific trial enrollment.
Expanded access / compassionate use request review
Individual requests for investigational therapy access are reviewed under a published policy; requests must be submitted in writing by a licensed treating physician.
Patient engagement and genetic testing facilitation
Coordination with external genetic research programs and patient advocacy partners to provide free at-home genetic testing kits, counseling, and referrals for genotype-specific trial enrollment.
Expertise Areas
- Gene therapy development for CNS and systemic diseases
- siRNA therapeutics development and early clinical testing
- Genotype-directed clinical trial recruitment
- CNS delivery methods (cisterna magna, intrathecal) and systemic infusion
Key Technologies
- Viral vector delivery
- Gene replacement therapy
- siRNA-mediated gene silencing
- Cisterna magna injection