Myrtelle is a gene therapy company conducting clinical trials that utilize targeted gene delivery techniques to treat rare monogenic disorders. The company focuses on providing therapeutic options for patients with genetic conditions that currently have no effective treatments available.
Funding
$44.5M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many rare monogenic disorders lack effective treatments, leaving patients with limited or no therapeutic options. These genetic conditions, particularly those affecting the central nervous system, pose significant challenges for drug development due to their complexity and the need for targeted delivery.
Solution
Myrtelle is a gene therapy company focused on developing targeted gene delivery techniques to address rare monogenic disorders. The company's platform is designed to treat myelin-based diseases, including white matter disorders (Leukodystrophies), by enabling innovative approaches to disorders with white matter involvement. Myrtelle's approach aims to transform the treatment, abilities, and quality of life for individuals affected by devastating neurological disorders, particularly those with limited existing treatment options. The company has strategic partnerships to advance its mission through scientific research, development, and manufacturing, including an exclusive IP license with Pfizer Inc.
Target Audience
The primary target audience includes patients with rare monogenic disorders, particularly those affecting the central nervous system, and their families, as well as medical professionals specializing in gene therapy and neurological diseases.
Features
- Targeted gene delivery for monogenic disorders
- Platform designed to address an array of diseases with white matter involvement
- Focus on myelin-based diseases and white matter disorders (Leukodystrophies)
- Exclusive IP license with Pfizer Inc.
- Development of novel treatments for devastating disorders of the central nervous system
- Clinical trials for Canavan disease