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Discover 5 Top Biotech Startups advancing Gene Therapy

Curious about new technological advancements in the Biotech industry? Explore our analysis of 362 global gene therapy startups & scaleups and learn how their solutions impact your business!

Staying ahead of the technology curve means strengthening your competitive advantage. That is why we give you data-driven innovation insights into the Biotech industry. This time, you get to discover 5 hand-picked startups advancing gene therapy.

Out of 362, the Global Startup Heat Map highlights 5 Top Biotech Startups advancing Gene Therapy

The insights of this data-driven analysis are derived from the Big Data & Artificial Intelligence (AI)-powered StartUs Insights Discovery Platform, covering 2 093 000+ startups & scaleups globally. The platform gives you an exhaustive overview of emerging technologies & relevant startups within a specific field in just a few clicks.

The Global Startup Heat Map below reveals the distribution of the 362 exemplary startups & scaleups we analyzed for this research. Further, it highlights 5 Biotech startups that we hand-picked based on criteria such as founding year, location, funding raised, and more. You get to explore the solutions of these 5 startups & scaleups in this report. For insights on the other 357 gene therapy startups, get in touch.

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Lexeo Therapeutics offers Adeno-Associated Virus (AAV) Gene Therapy

Founding Year: 2018
Location: New York, US
Funding: USD 85 M
Partner for: Neurodegenerative Gene Therapy

US-based startup Lexeo Therapeutics provides gene therapy for degenerative disorders. The startup’s lead therapy, LX2006, is an AAV-mediated therapy that targets Friedreich’s ataxia (FA), a multi-system disorder affecting cardiovascular functions. Other therapies in the startup’s portfolio include LX1004 and LX1001. The former delivers neuronal ceroid lipofuscinosis 2 (CLN2) to the central nervous system whereas the latter delivers APOE2. The two therapies are in clinical development and target Batten disease and Alzheimer’s respectively.

DegenRx provides Antibody-based Gene Therapy

Founding Year: 2018
Location: Tilburg, Netherlands
Partner for: AAV-Mediated Gene Therapy

Dutch startup DegenRx designs gene therapies for Parkinson’s disease. Parkinson’s disease marks the loss of dopaminergic neurons in the substantia nigra and the formation of Lewy Bodies. The startup’s antibody-based, AAV-mediated gene therapy approach specifically targets alpha-synuclein oligomers. It requires only a single administration in the spinal cord or central nervous system (CNS) for extended treatment. The startup also provides gene therapy for Alzheimer’s disease.

Genespire treats Inherited Metabolic Disorders

Founding Year: 2020
Location: Milan, Italy
Funding: USD 17 M
Partner for: Gene Therapy for Liver Disorders

Genespire is an Italian startup that develops gene therapy for inherited metabolic disorders. X-linked hyper IgM syndrome (X-HIGM) is a rare and life-threatening immune deficiency disorder. The startup’s solution targets X-HIGM using an advanced gene-editing approach. This approach rescues the functional expression of the endogenous defective gene, improving the immune response of patients. The startup also offers lentiviral vector technology for off-the-shelf, in vivo gene therapies for liver disorders.

Vivet Therapeutics develops Gene Replacement Therapy

Founding Year: 2016
Location: Paris, France
Funding: USD 93 M
Partner for: Corrective Gene Therapy

French startup Vivet Therapeutics engineers gene replacement therapy for Wilson’s disease. The disease results from a mutation in ATP7B copper transporter, resulting in toxic accumulation of copper in the live and central nervous system. The startup’s therapy restores physiological copper metabolism by providing a single intravenous vector administration. This alleviates the need for multiple daily administration of current treatments. Furthermore, it eliminates the associated side effects with current treatment and optimizes the adherence to treatment. The startup also develops therapies for progressive familial intrahepatic cholestasis (PFIC) and Citrullinemia type I (CTLN1).

Sexton Biotechnologies offers Human Platelet Lysate Products

Founding Year: 2019
Location: Indianapolis, US
Funding: USD 5 M
Partner for: Automated Fluid Management Platform

Sexton Biotechnologies is a US-based startup that develops human platelet lysate products. The pathogen-reduced human platelet lysates (hPL) provide a media that reduces risks and improves downstream performance against alternatives. The startup’s platform for gene therapies, Signata, leverages closed-automation to reduce contamination risks, capture batch records, and control the processes with a flexible system.

Discover more BioTech Startups

BioTech startups such as the examples highlighted in this report focus on gene editing, off-the-shelf therapies, and bioinformatics solutions. While all of these technologies play a major role in advancing the BioTech industry, they only represent the tip of the iceberg. To explore more BioTech technologies, simply get in touch to let us look into your areas of interest. For a more general overview, you can download our free BioTech Innovation Report to save your time and improve strategic decision-making.

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