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Uttam Rao, MD, MBA, a transplant physician at St. David's South Austin Medical Center of Sarah Cannon, discussed research comparing patient outcomes on different conditioning regimens for CAR-T.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

The professor of medicine at Duke Cancer Institute discussed uptake and research with the cord blood therapy since its approval.

KYV-101 is also being evaluated in lupus nephritis, myasthenia gravis, and systemic sclerosis.

The Chief Medical Officer of Addimmune discussed past and future research with the AGT103-T gene therapy.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Atul Malhotra, MD, PhD, the head of the early neurodevelopment clinic at Monash Children's Hospital, discussed the importance of preclinical guidelines in the context of neonatal cell therapy.

The director of the Stem Cell Transplant & Cellular Therapy Program at St. Louis Children’s Hospital discussed when the choice should be made to transition from symptom management to curative therapies.

Val-rox was approved as Roctavian in June 2023.

The assistant professor at Mayo Clinic School of Medicine discussed plans for further research and a phase 2/3 study.

Judy Lieberman, MD, PhD, the endowed chair in cellular and molecular medicine at Boston Children’s Hospital, discussed how there is still much room for growth for RNA therapeutics despite progress so far.

In observance of Parkinson's Awareness Month, held annually in April, CGTLive® took a closer look at REGENERATE-PD, a new trial for PD gene therapy AB-1005.

The associate professor of otolaryngology from Harvard Medical School discussed further research being pursued with AAV1-hOTOF gene therapy.

Zever-cel is being assessed in the phase 2 LUMMICAR-2 trial (NCT03915184) in North America, previously reporting results of an ORR of 100% among 11 patients.

Vivien Sheehan, MD, PhD, an associate professor of pediatrics at Emory University, discussed how patients should be able to choose to try the recently FDA-approved cell-based therapies for SCD when they are ready.

Review top news and interview highlights from the week ending March 29, 2024.

In light of the CTA clearance, Atamyo plans to carry out a multicenter, open-label phase 1b clinical trial (NCT05973630).

The clinical investigator in the Department of Bone Marrow Transplantation & Cellular Therapy at St. Jude Children’s Research Hospital discussed several areas of interest for research now that CAR-T is here to stay.

A 2-year evaluation of a small cohort of patients with systemic lupus erythematosus, idiopathic inflammatory myositis, and systemic sclerosis showed safety and efficacy which were supportive of additional clinical trials.

The head of the early neurodevelopment clinic at Monash Children's Hospital discussed clinical trials in the field of neonatal cell therapy.

Judy Lieberman, MD, PhD, the endowed chair in cellular and molecular medicine at Boston Children’s Hospital, discussed her research on siRNA technology.

The transplant physician at St. David's South Austin Medical Center of the Sarah Cannon Transplant and Cell Therapy Network discussed a real-world study comparing bendamustine against fludarabine and cyclophosphamide.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Notably, the combination therapy, referred to as SYNCAR-001 + STK-009, will be administered in the trial without the use of lymphodepletion.

In the phase 3 EMBARK trial, treatment with SRP-9001 improved secondary outcomes of time to rise, microdystrophin expression, and 10-meter walk/run.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed the importance of further research into the root cause of MS and other autoimmune diseases.

The company noted that it plans to submit the BLA in the second quarter of 2024.

The associate professor of pediatrics at Emory University also discussed the need to empower patients and families to make their own treatment decisions.