Solid Biosciences is developing gene therapy candidates targeting Duchenne muscular dystrophy and other rare neuromuscular and cardiac diseases, utilizing advanced genetic engineering techniques. The company aims to improve patient outcomes by addressing the underlying genetic causes of these debilitating conditions through its diverse pipeline of therapies.
Funding
$109M 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
Duchenne muscular dystrophy, catecholaminergic polymorphic ventricular tachycardia (CPVT), BAG3-mediated dilated cardiomyopathy, and Friedreich’s ataxia are debilitating genetic diseases with limited effective treatments. These conditions result from underlying genetic defects that lead to progressive muscle degeneration or cardiac dysfunction. Current therapies often fail to address the root cause of these diseases, offering only symptomatic relief.
Solution
Solid Biosciences is developing a portfolio of gene therapy candidates designed to address the underlying genetic causes of rare neuromuscular and cardiac diseases. Their approach involves utilizing advanced genetic engineering techniques to deliver functional genes or modify existing genes, aiming to restore normal muscle or cardiac function. The company's pipeline includes SGT-003 for Duchenne muscular dystrophy, SGT-501 for CPVT, AVB-401 for BAG3-mediated dilated cardiomyopathy, AVB-202-TT for Friedreich’s ataxia, and additional assets targeting fatal cardiac diseases. By targeting the genetic basis of these diseases, Solid Biosciences seeks to provide more effective and potentially curative treatments.
Target Audience
The primary target audience includes patients suffering from Duchenne muscular dystrophy, catecholaminergic polymorphic ventricular tachycardia (CPVT), BAG3-mediated dilated cardiomyopathy, and Friedreich’s ataxia, as well as the healthcare providers who treat them.
Features
- SGT-003: Gene therapy candidate for Duchenne muscular dystrophy
- SGT-501: Gene therapy candidate for catecholaminergic polymorphic ventricular tachycardia (CPVT)
- AVB-401: Gene therapy candidate for BAG3-mediated dilated cardiomyopathy
- AVB-202-TT: Gene therapy candidate for Friedreich’s ataxia
- Utilizes advanced genetic engineering techniques for targeted gene delivery or modification
- Focus on restoring normal muscle or cardiac function by addressing underlying genetic defects