This is a video synopsis/summary of a Peer Exchange involving Ryan Haumschild, PharmD, MS, MBA; Jessica Nance, MD, MS; Kimberly C. Chen, DO, MSHLM; Emma Ciafaloni, MD, FAAN; and Mary Pak, MD, FACP.

Investigators headed by a team at Brigham and Women’s Hospital have developed an adeno-associated virus (AAV) vector that studies show can far more efficiently cross the blood-brain barrier (BBB), in ...

An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent cells and leading to syndromic hearing loss. In a new experimental study, ...

Kristofer Mussar, PhD, is COO of VectorBuilder and MD of VectorBuilder GmbH, leading global operations and innovation in gene delivery. What if business leaders approached strategy the way scientists ...

Gene therapies can correct diseases at their genetic core, but their success hinges on viral vector manufacturing. As gene therapy technology becomes more advanced, producing viral vectors becomes ...

Astellas Pharma is digging up some cash to use one of 4D Molecular Therapeutics’ gene therapy vectors for at least one rare eye disease—but the genetic medicines company is going to have to wait a ...

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a ...