Seeking Alpha

Andelyn Biosciences Selected by Ultragenyx for Late-Stage Process Performance Qualification (PPQ) Manufacturing of Novel Gene Therapy for Sanfilippo Syndrome

COLUMBUS, Ohio, Dec. 18, 2023 /PRNewswire/ -- Andelyn Biosciences, Inc., a pioneering and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), announced ...
Nasdaq

Ultragenyx Announces New Data For UX111 Gene Therapy In Treating Sanfilippo Syndrome Type A

(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company, on Wednesday reported encouraging data from its ongoing clinical program of UX111, a gene therapy for Sanfilippo ...
Business Wire

Spruce Biosciences Receives U.S. FDA Breakthrough Therapy Designation for Tralesinidase Alfa Enzyme Replacement Therapy (TA-ERT) in Sanfilippo Syndrome Type B (MPS IIIB)

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological ...
InvestorsHub on MSN

Spruce Biosciences Shares Surge After FDA Grants Breakthrough Therapy Designation

Shares of Spruce Biosciences (NASDAQ:SPRB) jumped 60% on Monday after the company announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for its ...
WFMY News2

Parents of children with rare genetic condition concerned about recent pharmaceutical delays

KNOXVILLE, Tenn. — Bryan and Blair Begbie said all they want for Rowan — their youngest daughter — is the chance to be a kid. However, the Food and Drug Administration issued what is called a Complete ...