Vivet Therapeutics

Name

Vivet Therapeutics

Legal name

Vivet Therapeutics

Website

https://vivet-therapeutics.com

Company type

Private

Founded year

2016

Operating status

Operating

Headcount range

11–50 employees

Ownership category

akta.pro rank

Product category

Gene Therapy

NAICS

Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

AAV Vector Gene Therapy Developers (HLAAACAA)

akta.pro secondary industries

Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Cardiometabolic & Other Systemic Gene Therapies (HLAAACAI), Gene Therapy Delivery Devices & Administration Systems (catheters, intrathecal, ocular delivery) (HLAAACAL)

Keywords

• Gene therapy development
• AAV gene therapy
• Rare disease therapeutics
• Liver-directed therapies
• Orphan disease treatments

Headquarters

HQ city

Paris

HQ country

France

HQ region

Europe

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Gene Therapy Development and Commercialization: Pre-revenue clinical-stage biotech. Development of investigational gene therapies for rare inherited metabolic liver diseases. Revenue expected from future product commercialization, with potential option fees and milestone payments from partnership agreements (e.g., Pfizer exclusive option to acquire, Mirum option for PFIC programs).

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Vivet Therapeutics is a clinical-stage biotechnology company developing a diversified pipeline of liver-directed adeno-associated virus (AAV) gene therapies for rare inherited metabolic disorders. Its investigational products include VTX-801 for Wilson's disease (in Phase 1/2 GATEWAY trial), VTX-802 and VTX-803 for Progressive Familial Intrahepatic Cholestasis types 2 and 3, VTX-806 for Cerebrotendinous Xanthomatosis, and the proprietary VTX-PID imlifidase-based technology platform designed to deplete anti-AAV neutralizing antibodies.

Product overview

Vivet Therapeutics is a clinical-stage biotech company developing a diversified pipeline of liver-directed AAV (adeno-associated virus) gene therapy products for rare inherited metabolic liver disorders. The portfolio includes VTX-801 for Wilson's disease (in Phase 1/2 GATEWAY trial with Orphan Drug and Fast Track designations), VTX-802 and VTX-803 for Progressive Familial Intrahepatic Cholestasis (PFIC2 and PFIC3), and VTX-806 for Cerebrotendinous Xanthomatosis (CTX). The company also operates VTX-PID, a proprietary technology platform using imlifidase to enable AAV gene therapy in patients with pre-existing neutralizing antibodies. Vivet has established partnerships with FIMA/CIMA (University of Navarra), Massachusetts Eye and Ear (Harvard), and collaborations with Pfizer and Mirum Pharmaceuticals.

Differentiator

Problem solved

Functional benefit

Brands

• VTX-801: AAV-based gene therapy for Wilson's disease - VTX-801 is Vivet's lead program, an investigational gene therapy for Wilson Disease that has received Orphan Drug Designation and Fast Track designation from FDA and European Commission
• VTX-PID
• VTX-806
• VTX-802
• VTX-803

Products and services

• VTX-801 Investigational AAV-based gene therapy delivering a functional mini-ATP7B copper transporter to liver cells to restore copper metabolism in patients with Wilson's disease. Lead clinical-stage program currently in Phase 1/2 GATEWAY trial at six leading centers in the US and Europe; holds Orphan Drug Designation from FDA and EC and FDA Fast Track designation.
• VTX-802 AAV-based gene therapy delivering a codon-optimized version of the BSEP gene to the liver for patients with Progressive Familial Intrahepatic Cholestasis Type 2 (PFIC2). Currently under preclinical testing and licensed to Amets Biotechnology Inc.
• VTX-803 AAV-based gene therapy delivering the human ABCB4 gene to restore MDR3 transporter function for treatment of Progressive Familial Intrahepatic Cholestasis Type 3 (PFIC3). Granted Orphan Drug Designation from both FDA and European Commission.
• VTX-806 AAV-based gene therapy delivering a codon-optimized version of the CYP27A1 gene to the liver for treatment of Cerebrotendinous Xanthomatosis (CTX), a rare neurodegenerative disease. Holds Orphan Drug Designation from both FDA and European Commission.
• VTX-PID Proprietary imlifidase formulation technology designed to deplete neutralizing antibodies (NAbs) against AAV serotypes, enabling AAV gene therapy in NAb-positive subjects who were previously ineligible for treatment and allowing potential gene therapy re-administration.

Quantifiable outcome

• VTX-PID reduced anti-AAV3B NAbs to non-inhibitory levels in significant portion of naturally immunized participants in clinical studies
• +1 more outcomes

Named customers1 record

Segments4 records

Ideal customer profiles5 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Three partnerships are on record, tiered core.

• Mirum PharmaceuticalscoreStrategic or Co-development Partner · 12 March 2021Exclusive worldwide option and license agreement for Vivet's PFIC gene therapy programs (VTX-802 for PFIC2 and VTX-803 for PFIC3). Mirum provides funding for preclinical development costs and gains exclusive option to license programs after IND/CTA-enabling studies, after which Mirum leads clinical development and commercialization.
• Fundación para la Investigación Médica Aplicada (FIMA) / CIMA, University of NavarracoreTechnology or IntegrationStrategic collaboration with not-for-profit foundation at Centro de Investigación Médica Aplicada (CIMA), University of Navarra, Pamplona, Spain. Vivet has exclusive licenses to novel proprietary AAV vector gene therapy technology to treat metabolic diseases. Gloria Gonzalez-Aseguinolaza serves as CSO and co-founder, originating from CIMA.
• Massachusetts Eye and Ear (MEE)coreTechnology or IntegrationExclusive license from MEE, a teaching hospital of Harvard Medical School in Boston, for patent-protected Anc80 AAV gene therapy vectors. Anc80 is a next-generation gene therapy technology designed to increase gene expression levels in the liver while reducing risk of undesired immunogenicity.

Scale indicators3 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• uniQure: Clinical and commercial AAV gene therapy company with programs in hemophilia, Huntington's disease, and other rare diseases. Comparable AAV manufacturing scale and rare disease commercial focus.
• Ultragenyx Pharmaceutical: Clinical-stage and commercial gene therapy company developing AAV-based therapies for rare metabolic diseases. Direct overlap with Vivet in liver-directed and rare disease gene therapy, similar indication breadth across ultra-rare populations.
• BioMarin Pharmaceutical: Established rare disease biotech with commercial gene therapy (Roctavian for hemophilia A) and pipeline in metabolic rare diseases. Direct overlap with Vivet in liver-directed AAV gene therapy for inherited metabolic disorders.
• Regenxbio: AAV gene therapy platform company developing its own pipeline plus licensing NAV vectors to partners. Highly comparable technology base to Vivet's AAV approach, with similar liver-directed and rare disease indications.
• Freeline Therapeutics: Clinical-stage AAV gene therapy company with liver-directed programs for Fabry disease and hemophilia B. Closely comparable in liver-tropic AAV approach and rare inherited metabolic disease focus.

Emerging players

• Passage Bio: Clinical-stage AAV gene therapy company focused on rare monogenic CNS diseases. Smaller pipeline with partial overlap in rare disease AAV gene therapy but primarily CNS-directed rather than liver-directed.
• Abeona Therapeutics: Clinical-stage gene and cell therapy company developing treatments for rare diseases. Comparable in rare disease gene therapy focus, with similar headcount and stage profile to Vivet.
• Taysha Gene Therapies: Clinical-stage AAV gene therapy company developing treatments for rare CNS diseases. Comparable stage and rare disease focus but different therapeutic area, both navigate similar regulatory and partnership dynamics.
• LogicBio Therapeutics: Clinical-stage gene therapy company developing AAV-mediated and genome editing approaches for rare diseases. Comparable in rare disease focus and AAV delivery technology base.

Others

• Mirum Pharmaceuticals: Commercial rare disease company that holds the exclusive option/license to Vivet's PFIC programs (VTX-802, VTX-803). Directly comparable as a PFIC-focused rare disease company and the most relevant downstream commercialization counterparty for Vivet's PFIC assets.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/Vivet_tx
• LinkedInlinkedin.com/company/vivettherapeutics

Compliance3 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Subsidiaries1 record

Funding overview

Funding rounds4 records

Investors9 records

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

M&A

Investments

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

Frequently asked questions about Vivet Therapeutics