CGTLive
Online/Digital
The Mission of CGTLive™ is to deliver highly relevant, quality information about cell, gene, regenerative, and engineered medicines to healthcare professionals in order to facilitate the best patient care possible. CGTLive™ offers an independent, comprehensive digital platform that covers practice-changing news and insight directly from top medical conferences and researchers. The platform is intended for busy healthcare professionals who are considering or currently treating patients with The Mission of CGTLive™ is to deliver highly relevant, quality information about cell, gene, regenerative, and engineered medicines.
The news platform offers an in-depth look at the multitude of new products and procedures in development with ever-expanding targets and mechanisms, to help provide clarity during an unprecedented time of growing demand for expertise. The CGTLive™ platform connects visitors with the most up-to-date clinical trial results, FDA updates and approvals, practice-changing research, expert insight, and more. Additionally, through a unique mix of video editorial, CGTLive™ offers dynamic discussions with authoritative insights, opinions, and perspectives on critical issues facing today's healthcare professionals.
CGTLive™ delivers direct access to expert insight to improve the lives of patients with genetic disorders and targetable cancers, such as lymphoma, Duchenne muscular dystrophy, spinal muscular atrophy, lysosomal storage disorders, retinitis pigmentosa, GM1 and GM2 gangliosidosis, mucopolysaccharidosis, sickle cell anemia, hemophilia, and much more. Source
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Media Outlet details
| Scope | National, Consumer |
|---|---|
| Language | English |
| Country | United States of America |
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Recent Articles
Search ArticlesFDA Places New Clinical Hold On RGX-121 for Hunter Syndrome
The FDA has placed a new clinical hold on REGENXBIO's RGX-121 (clemidsogene lanparvovec), an investigational one-time gene therapy for Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, after asymptomatic spine MRI findings were identified in 5 participants in the CAMPSIITE study (NCT03566043).1 REGENXBIO noted in a release that it does not expect to resubmit the RGX-121 biologics license application (BLA) in the near term.
FDA Extends Deramiocel Review for Duchenne Muscular Dystrophy
The FDA has extended the Prescription Drug User Fee Act (PDUFA) target action date for Capricor Therapeutics' deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), from August 22 to November 22, 2026.1 The 3-month extension follows the agency's acceptance of additional phase 3 HOPE-3 (NCT05126758) data as a major amendment to the biologics license application (BLA).2 The amendment includes 24-month open-label extension findings and additional robustness analyses...
PTC Wins Sangamo's Fabry Gene Therapy in Bankruptcy Auction
According to a new announcement, PTC Therapeutics has been selected as the winning bidder to acquire isaralgagene civaparvovec (ST-920), a Biologics License Application-stage adeno-associated virus vector-based gene therapy for Fabry disease, from Sangamo Therapeutics in a court-supervised bankruptcy auction.
Pariglasgene Brecaparvovec-opnr Approved for Glycogen Storage Disease 1a
According to a new announcement, the FDA has granted accelerated approval to pariglasgene brecaparvovec-opnr (Genglycos; Ultragenyx Pharmaceutical, Inc), a one-time, liver-directed AAV8 gene therapy, to reduce daily cornstarch intake as an adjunct to nutritional management in patients 8 years and older with glycogen storage disease type Ia (GSD1a).1 The approval, announced August 19, 2026, makes pariglasgene brecaparvovec-opnr the first approved treatment for GSD1a.1 "Patients with GSD1a face...
Miv-cel Earns RMAT Designation for Progressive Multiple Sclerosis
According to a new announcement, the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to mivocabtagene autoleucel (miv-cel; Kyverna Therapeutics) for non-active secondary progressive multiple sclerosis (naSPMS). Miv-cel is an autologous CD19-targeting CAR T-cell therapy. The designation, granted in August 2026, was based on what the company described as compelling clinical data from an ongoing Stanford University investigator-initiated trial (IIT).
J&J Secures $2.58B Option to Acquire Sail Biomedicines
Johnson & Johnson has entered a collaboration and equity investment agreement with Sail Biomedicines, a Flagship Pioneering company, to advance in vivo CAR T-cell therapies for immune-mediated diseases. As part of the agreement, Johnson & Johnson secured an exclusive option to acquire Sail for $2.58 billion. The companies will jointly advance Sail's lead in vivo CAR T program along with its broader platform technology.
FDA Panel Votes Against Deramiocel for Duchenne cardiomyopathy
The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) voted 9 to 3, with no abstentions, that available evidence does not support the effectiveness of deramiocel for treating cardiomyopathy in patients with Duchenne muscular dystrophy (DMD). Deramiocel (Capricor Therapeutics) is an allogeneic cardiosphere-derived cell therapy administered by intravenous infusion every 3 months.
Rugonersen's Phase 3 BEACON Trial Doses First Participant
Oak Hill Bio announced that the first participant has been dosed in BEACON (NCT07605429), a pivotal phase 3 trial evaluating rugonersen, an investigational antisense oligonucleotide (ASO), for Angelman syndrome.
Cell Therapy TG-C Misses Primary Endpoints in Phase 3 Knee Osteoarthritis Trial
Kolon TissueGene announced topline results from ACTiVION-II (NCT03291470), the first of two US phase 3 trials evaluating TG-C (TissueGene-C), an allogeneic cell and gene therapy for knee osteoarthritis.¹ The randomized, double-blind, placebo-controlled trial missed both co-primary endpoints, change from baseline in VAS pain and WOMAC total score at month 12, and none of the key secondary endpoints were met.
Liver-Directed Gene Therapy Shows Durable Effect in MMA Mouse Model
Genespire, in collaboration with the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), reported findings published in the Journal of Hepatology showing that liver-directed delivery of an immune-shielded lentiviral vector (ISLV) encoding the MMUT gene produced durable therapeutic benefits in a validated mouse model of methylmalonic acidemia (MMA).1,2 Systemic administration to 2-week-old MMA mice produced a rapid and long-lasting effect, sustained for more than a year, or roughly...