Gene Therapy (2)

Challenge A private biotech company developing gene therapy products for several orphan diseases was in the process of initiating CMC development for their pipeline products. In order to move...

Challenge A privately-held biotechnology company developing and exploiting a proprietary technology platform for optimizing and controlling gene therapy expression was looking to evolve its business...

Challenge: Alacrita conducted an assessment of an AAV gene therapy for an ophthalmic indication, focusing on market potential, technical and regulatory issues and identification of key risks.