Inherited Disease
News on Mendelian diseases, hereditary cancer, variant classification, cystic fibrosis testing and more.
In a Phase I/II trial, patients with X-linked chronic granulomatous disease will receive a treatment created using a new approach to base editing.
Be Biopharma Raises $92M in Series C Financing to Advance Engineered B-Cell Therapies
The firm will use the proceeds to advance BE-101 for hemophilia B through clinical proof-of-concept testing and BE-102 for hypophosphatasia into the clinic.
JP Morgan Healthcare Conference, Day 1: Vertex, Bristol Myers Squibb, Gilead, Sarepta, and More
On the first day, Vertex discussed regulatory plans for a kidney disease drug, BMS and Merck talked about pipeline diversification, and Gilead offered updates on anito-cel.
The company anticipates launching three CRISPR products by 2030, including therapies for hereditary angioedema and transthyretin amyloidosis.
Regenxbio, Nippon Shinyaku Ink Deal to Advance Hunter, Hurler Syndrome Gene Therapies
In the $810 million deal, Nippon will commercialize RGX-121 and RGX-111 in the US and Asia, and Regenxbio will manufacture them.