We are a clinical stage company developing a compelling portfolio of innovative AAV-based genetic medicines for people with serious rare neuromuscular diseases. We see the patient community as our partners, collaborators, and teachers. We leverage our AAV gene therapy technology platform and proprietary manufacturing expertise to progress programs across three modalities: gene replacement, vectorized exon skipping, and vectori...
We are a clinical stage company developing a compelling portfolio of innovative AAV-based genetic medicines for people with serious rare neuromuscular diseases. We see the patient community as our partners, collaborators, and teachers. We leverage our AAV gene therapy technology platform and proprietary manufacturing expertise to progress programs across three modalities: gene replacement, vectorized exon skipping, and vectorized RNA knockdown. We use a focused set of criteria to select target indications, which we believe increases the chances of success for each of our programs.
Sponsored Ad
Upload your Marketing & Sales content on your company Virtual Booth, click HERE.