CGTLive

CGTLive™ aims to provide healthcare professionals with essential, high-quality information about cell, gene, regenerative, and engineered medicines to ensure optimal patient care. This independent digital platform offers a comprehensive resource that highlights groundbreaking news and insights directly from leading medical conferences and researchers. It is specifically designed for busy healthcare professionals who are either considering or currently managing patients receiving these advanced therapies. The news platform features detailed coverage of emerging products and procedures, addressing the growing need for expertise in an ever-evolving field. CGTLive™ connects users with the latest clinical trial results, FDA updates and approvals, transformative research findings, and expert opinions. Additionally, through engaging video content, CGTLive™ presents lively discussions with knowledgeable experts on important topics relevant to today’s healthcare practitioners. CGTLive™ provides direct access to expert knowledge aimed at enhancing the lives of patients affected by genetic disorders and specific cancers, including lymphoma, Duchenne muscular dystrophy, spinal muscular atrophy, lysosomal storage disorders, retinitis pigmentosa, GM1 and GM2 gangliosidosis, mucopolysaccharidosis, sickle cell anemia, hemophilia, and more.

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Fate Therapeutics’ iPSC-derived CAR-T FT819 Nabs FDA RMAT Designation for Lupus

1 week ago | cgtlive.com | Noah Stansfield

Fate Therapeutics’ FT819, an investigational allogeneic induced pluripotent stem cell (iPSC)-derived chimeric antigen receptor T-cell (CAR-T) therapy, has garnered regenerative medicine advanced therapy (RMAT) designation from the FDA for active moderate to severe systemic lupus erythematosus (SLE), including lupus nephritis (LN).1 FT819, the development of which is supported by a California Institute of Regenerative Medicine grant that provides $7.9 million in funding, is currently being...
Using Armored CAR T-Cells to Tackle Solid Tumors

1 week ago | cgtlive.com | Renier J Brentjens |Noah Stansfield

April 14, 2025By Renier Brentjens, MD, PhD, the chair of the department of medicine at Roswell Park Comprehensive Cancer Center, discussed the innovations necessary to make CAR-T therapy effective in solid tumor indications. One of the ongoing challenges in oncology cell therapy is applying chimeric antigen receptor T-cell (CAR-T) therapy, which has proven efficacious in blood cancers, to solid tumors. One of the methods currently being explored for this purpose is armored CAR T-cells.
CGTLive®’s Weekly Rewind - April 11, 2025

1 week ago | cgtlive.com | Noah Stansfield

Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field. The designations apply to lupus, myositis, and scleroderma indications. The chair of the department of medicine at Roswell Park Comprehensive Cancer Center discussed the innovations necessary to make CAR-T therapy effective in solid tumor indications.
World Parkinson's Day 2025: Looking Back at Progress for Cell and Gene Therapy

1 week ago | cgtlive.com | Noah Stansfield

April 11, 2025In observance of World Parkinson's Day, held annually on April 11, we took a look back at the past year's news in cell and gene therapy for PD. According to the Michael J. Fox Foundation, almost 1 million people in United States live with Parkinson disease (PD) and more than 6 million people in the world live with PD. Notably, the symptoms of PD and their severity vary widely, and can be different for every person.
Lexeo’s Gene Therapy LX2006 Reduces Abnormal LVMI in Patients With Friedreich Ataxia Cardiomyopathy

2 weeks ago | cgtlive.com | Noah Stansfield

Lexeo Therapeutics’ LX2006, an adeno-associated virus (AAV) vector-based gene therapy, has demonstrated the ability to reduce left ventricular mass index (LVMI) in patients with Friedreich ataxia (FA) cardiomyopathy who had a normal LVMI at baseline.1The data come from patients treated across 2 studies: the Lexeo phase 1/2 SUNRISE-FA clinical trial (NCT05445323) and the phase 1a Weill Cornell Medicine investigator-initiated trial (NCT05302271).
Renier Brentjens, MD, PhD, on Supporting Translational Cell Therapy Research

2 weeks ago | cgtlive.com | Renier J Brentjens |Noah Stansfield

This is the fourth part of an interview with Renier Brentjens, MD, PhD. For the first part, click here. For the second part, click here. For the third part, click here. “...[W]e kept publishing more ways of curing mice, and it looked better, the doses were lower, and all of this—and of all that technology that was described, much of it remarkably elegant, very little of it has seeped into the realm of translational research, into clinical trials, and for better outcomes.
Renier Brentjens, MD, PhD, on Barriers to Accessibility for CAR-T

2 weeks ago | cgtlive.com | Renier J Brentjens |Noah Stansfield

This is the third part of an interview with Renier Brentjens, MD, PhD. For the first part, click here. For the second part, click here. “Look, I'm a leukemia doc. I spent years giving high dose chemotherapy to our leukemia patients...
Around the Helix: Cell and Gene Therapy Company Updates - April 9, 2025

2 weeks ago | cgtlive.com | Noah Stansfield

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more. Have a cell and gene therapy news update you’d like to share with our editorial team?
Independent DMC Concludes that Risk-Benefit Ratio for Sarepta’s DMD Gene Therapy Elevidys Remains Favorable

2 weeks ago | cgtlive.com | Noah Stansfield

An independent data monitoring committee (DMC) concluded based on a “totality of evidence” that the risk-benefit-ratio for Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), a marketed adeno-associated virus (AAV) vector-based gene therapy intended to treat Duchenne muscular dystrophy (DMD), remains favorable for continued dosing in 3 clinical trials without changes to the protocols.1 The finding, shared by Sarepta and its partner Roche, relates to 3 clinical...
Renier Brentjens, MD, PhD, on Autologous Versus Allogeneic CAR-T for the Near-Term Future

2 weeks ago | cgtlive.com | Renier J Brentjens |Noah Stansfield

This is the second part of an interview with Renier Brentjens, MD, PhD. For the first part, click here. “...[T]here's certainly virtue to having off-the-shelf products.