The startup develops gene therapy aimed at restoring the function of defective proteins in patients with Friedreich's ataxia, a rare neurodegenerative disease. This approach targets the underlying genetic causes of the condition, providing a potential effective treatment for affected individuals.
Funding
$2.2M 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
Friedreich's ataxia, a rare inherited neurodegenerative disease, lacks effective treatments that address the underlying genetic cause, leading to progressive neurological and cardiac deficits in affected individuals. Current management strategies primarily focus on symptomatic relief and supportive care, failing to halt or reverse disease progression.
Solution
Biointaxis is developing a gene therapy, FRATAXAV®, designed to restore native levels of frataxin, the protein deficient in Friedreich's ataxia patients, by targeting the root genetic cause of the condition. The therapy aims to prevent, stop, and potentially reverse the neurological and cardiac deficits associated with the disease. Biointaxis focuses on early-stage product development, pre-clinical studies, and early clinical trials (Phase I/II), with the goal of licensing production and distribution rights to established pharmaceutical partners. Pre-clinical research has demonstrated the efficacy of the treatment in re-establishing neurological functions in mice models of Friedreich's ataxia.
Target Audience
The primary target audience includes individuals affected by Friedreich's ataxia, their families, and pharmaceutical companies specializing in gene therapies for rare neurodegenerative diseases.
Features
- AAV-based gene therapy approach for targeted delivery and expression of the frataxin gene.
- Designed to restore native levels of frataxin protein in affected cells.
- Demonstrated efficacy in pre-clinical studies using mouse models of Friedreich's ataxia.
- Focus on early-stage development, including R&D and pre-clinical regulatory work.
- Aims to license production and distribution rights to pharmaceutical partners for late-stage development and commercialization.
- Part of the PHOENIX project, developing Organs-on-Chip platforms for cardiac and neuro-muscular diseases.