资讯

Gene hunt leads to potential colorectal cancer treatment
Justin Taylor, M.D., a physician-scientist at Sylvester Comprehensive Cancer Center, part of the University of Miami Miller ...
BIRAC in pact with Miltenyi Biotec India to boost India's capabilities in cell & gene therapy
BIRAC and Miltenyi Biotec India have joined forces to elevate India's standing in cell and gene therapy (CGT). The ...
Verve: Eli Lilly Acquisition Based On One-Time GalNAc PCSK9 Gene Editing Program
Eli Lilly acquires Verve Therapeutics, Inc. for $1B to advance gene-editing therapies like VERVE-102, targeting ASCVD. Click ...
Sarepta shares crash as second patient dies after receiving its gene therapy
Shares of Sarepta Therapeutics closed down as much as 42% to hit a nine-year low of $18.30 on Monday after a second death of ...
Managed Healthcare Executive3 天
Sarepta Stops Shipments of Elevidys For Non-Ambulatory DMD Patients
A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys ...
Fierce Pharma3 天
Sarepta reports 2nd death after DMD gene therapy Elevidys, stops dosing in half of patients
A second patient has died following treatment with Sarepta Therapeutics’ Elevidys, raising more doubts about the Duchenne muscular dystrophy (DMD) gene therapy’s safety profile. | A second patient has ...
90.5 WESA3 天
Penn researchers identify new potential gene therapy for glioblastoma brain cancer
Glioblastoma has been deadly and hard to treat, but researchers say they’ve made progress with bivalent CAR T-cell therapy ...
Sarepta Therapeutics: Rebuilding The Thesis After Hitting Rock Bottom
Despite setbacks, Sarepta’s gene therapy pipeline and market leadership in DMD present a high-risk, high-reward setup. Read ...
STAT4 天
FDA reassures rare disease advocates that ‘being flexible’ is plan for gene therapy
Trump health officials signal eased FDA rules for rare disease gene therapy, but concerns linger over safety, oversight, and ...
Saints Wire6 天
Sickle cell disease cured? Ohio State celebrates big win via innovative gene therapy
Some doctors told him he'd live with the disease forever, but Khaled Alsheebani never stopped believing treatments would ...
BioWorld14 天
Regenxbio’s Duchenne gene therapy data positive as shares falter
Regenxbio Inc.’s gene therapy in treating Duchenne muscular dystrophy (DMD) produced positive initial phase I/II results from its first five patients. However, the company’s stock (NASDAQ:RGNX) ...
Nasdaq14 天
FDA Grants Platform Technology Tag to Sarepta's Gene Therapy Vector
Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, used in the investigational gene therapy SRP-9003. This therapy is being ...