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 ArticlesMivocabtagene Autoleucel Shows Durable 1-year Benefit in SPS, gMG
According to a new announcement, Kyverna Therapeutics reported positive one-year data from the KYSA-8 trial (NCT06588491) of mivocabtagene autoleucel (miv-cel; KYV-101; Kyverna Therapeutics) in stiff person syndrome (SPS) and updated longer-term data from the KYSA-6 trial (NCT06193889) in generalized myasthenia gravis (gMG).¹ In the 26-patient KYSA-8 cohort, median improvement in the Timed 25-Foot Walk (T25FW) reached 49% at month 12, compared with 46% at week 16 (P <.0001).¹ All seven...
FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A
According to a new announcement, the FDA has approved rebisufligene etisparvovec-hopf (Fayuvi) for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A.
Ocugen Doses First Patient in Phase 3 Trial of OCU410 for GA
Ocugen has dosed the first patient in ArMaDa3 (NCT07770828), a global phase 3 registrational trial of OCU410, an investigational one-time subretinal gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD).1 The trial is the first pivotal gene therapy study conducted in GA, and dosing began just weeks after the FDA granted OCU410 regenerative medicine advanced therapy (RMAT) designation on July 29, 2026.1 OCU410 (AAV5-hRORA) delivers the human...
Novartis, BMS Pause Autoimmune CAR-T Trials After Safety Events
Novartis has suspended several autoimmune and neurology trials of its investigational CD19 CAR-T therapy rapcabtagene autoleucel (rap-cel) after 3 patients died following serious cases of immune effector cell-associated hemophagocytic syndrome (IEC-HS).1,2 Bristol Myers Squibb has separately paused at least some studies of its own CAR-T candidate, zolacabtagene autoleucel (zola-cel), after observing transient and reversible inflammatory events in its autoimmune development program.1,2 A...
FDA 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.