Adeno-associated virus (AAV), valued for its low immunogenicity, broad tropism, and long-lasting in vivo gene expression, has emerged as the leading vector for gene therapy delivery. For AAV vectors intended for clinical use, the FDA requires stringent quality control...
Featured Jan 14 REGENXBIO and Nippon Shinyaku Announce Exclusive Partnership to Develop and Commercialize RGX-121 and RGX-111 for MPS Diseases read more » Jan 13 AbbVie and REGENXBIO Announce Updates on the ABBV-RGX-314 Clinical Program read more » Jan 13 JPM25:...
Kyverna Therapeutics, under new CEO Warner Biddle, has refined its strategic focus for its CAR-T cell therapy program, KYV-101, prioritizing stiff person syndrome (SPS) as its initial target for approval. Here’s a summary: New Strategic Focus: Kyverna aims to be...
REGENXBIO Inc. and Nippon Shinyaku Co., Ltd. have entered a strategic partnership to develop and commercialize RGX-121 for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome....
Featured Jan 08 First-ever gene edit of infant boy leads to apparent cure read more » Jan 07 Novartis gene therapy poised to expand SMA treatment landscape read more » Jan 07 Vertex pays Orna $65M to work on next-gen gene therapies for blood disorders read more » News...
