Prevail Therapeutics

Name

Prevail Therapeutics

Legal name

Prevail Therapeutics Inc.

Website

https://prevailtherapeutics.com

Company type

Private

Founded year

2019

Operating status

Operating

Headcount range

51–100 employees

Ownership category

akta.pro rank

Product category

Gene Therapy / Biopharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

AAV Vector Gene Therapy Developers (HLAAACAA)

akta.pro secondary industries

Neurology/CNS Gene Therapies (HLAAACAH), siRNA Therapeutics (gene silencing) (HLAAADAB), Genome Editing Gene Therapies (CRISPR/TALEN/ZFN; in vivo/ex vivo) (HLAAACAD)

Keywords

• Gene therapy development
• Neurodegenerative disease therapeutics
• AAV vector delivery
• GBA1 gene replacement
• SiRNA therapeutics

Headquarters

HQ city

New York

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales

Revenue model

1. Gene Therapy Commercialization: Once clinical trials are completed and regulatory approvals are obtained, Prevail expects to generate revenue through commercialization of gene therapies for Parkinson's disease and Gaucher disease. The company operates as a wholly owned subsidiary of Eli Lilly and Company, which provides commercial infrastructure for eventual product launch.
2. Partnership Milestone Payments: Prevail receives milestone payments from collaboration partners such as Scribe Therapeutics for achievement of development, regulatory, and commercial milestones, plus royalties on future sales.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Prevail Therapeutics is a biotechnology company developing gene therapies and siRNA therapies that target the underlying genetic causes of neurodegenerative and rare diseases. Its pipeline includes AAV-based gene therapies for Parkinson's disease with GBA1 variants and Type 1 Gaucher disease, plus an siRNA therapy for sporadic Parkinson's disease, currently administered through three active clinical trial programs (PROPEL, PROCEED, PROSPECT).

Product overview

Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company, is developing three distinct clinical-stage genetic therapies targeting the root causes of neurodegenerative and rare diseases: PROPEL (gene therapy for Parkinson's disease with GBA1 variants, delivered via cisterna magna injection), PROCEED (gene therapy for Type 1 Gaucher disease, administered as a single intravenous infusion), and PROSPECT (siRNA therapy for sporadic Parkinson's disease, delivered via intrathecal injection). The company focuses on gene therapy and siRNA approaches that address underlying genetic causes rather than symptom management alone.

Differentiator

Problem solved

Functional benefit

Products and services

• PROPEL

Quantifiable outcome

• Clinical trials are evaluating safety, tolerability, and effectiveness of gene therapies designed to slow disease progression in Parkinson's and Gaucher disease patients

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

One partnership is on record.

• Scribe TherapeuticscoreTechnology or Integration · 1 January 2023Scribe Therapeutics and Prevail Therapeutics entered a strategic collaboration to develop in vivo CRISPR-based genetic medicines for neurological and neuromuscular diseases. Under the deal terms, Scribe receives $75 million upfront plus equity investment, with potential milestone payments exceeding $1.5 billion and tiered royalties on future sales. Scribe also retains the option to co-fund and share profits on one collaboration program in the United States. The collaboration focuses on leveraging Scribe's X-Editor (XE) CRISPR technology for in vivo genome editing in the CNS.

Scale indicators3 records

Recent moves5 records

Expansion highlights4 records

Direct peers

• uniQure: Gene therapy company developing AAV-based therapies with CNS programs including Huntington's disease. Highly comparable as a clinical-stage AAV developer focused on neurodegenerative rare diseases with similar regulatory pathway and manufacturing constraints.
• Sarepta Therapeutics: Commercial-stage gene therapy company with multiple approved AAV-delivered therapies and active pipeline in rare neurological diseases. Comparable as a fellow AAV gene therapy developer with operational scale in CNS and rare disease markets.
• REGENXBIO: AAV gene therapy platform company with CNS pipeline including Parkinson's disease programs. Comparable as a peer AAV developer pursuing neurodegenerative indications with proprietary delivery technology.
• Alnylam Pharmaceuticals: Leading siRNA therapeutics company with approved products and active CNS-pipeline development. Comparable for Prevail's PROSPECT program targeting α-synuclein in Parkinson's disease via siRNA approach.
• Voyager Therapeutics: Clinical-stage AAV gene therapy company focused on CNS/neurological diseases including Parkinson's disease programs. Most direct competitor to Prevail with overlapping AAV delivery approaches and Parkinson's disease as a lead indication.

Broad incumbents

• Roche/Genentech: Global pharmaceutical incumbent with significant gene therapy capabilities (including Spark Therapeutics) and active neurodegenerative disease programs. Comparable as a broad incumbent with overlapping CNS and gene therapy ambitions.
• Pfizer (Gene Therapy): Large pharmaceutical incumbent with a gene therapy division and CNS pipeline. Comparable as a broad incumbent pursuing AAV gene therapy for rare neurological diseases through a wider portfolio approach.
• Novartis (Gene Therapy): Global pharmaceutical incumbent with approved gene therapies (Zolgensma, Kymriah) and active CNS programs. Comparable as a broad incumbent with established gene therapy commercialization infrastructure.
• BioMarin Pharmaceutical: Specialty biopharmaceutical company with gene therapy programs targeting CNS diseases including Parkinson's. Comparable as an incumbent with overlapping AAV gene therapy ambitions in neurodegenerative rare diseases.

Emerging players

• Scribe Therapeutics: CRISPR-based in vivo genome editing company and Prevail's strategic partner. Comparable as a peer in CRISPR gene editing for neurological diseases via the X-Editor platform collaboration.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks4 records

Key highlights6 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds3 records

Investors11 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments1 record

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Prevail Therapeutics