News

For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.

In observance of Crohn and Colitis Awareness Week, observed from December 1 to 7, CGTLive™ took a closer look at the clinical evaluation of the MSC-derived therapy, ExoFlo.

The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham discussed the anticipated approval of the first gene therapy for SCD.

Health Canada has greenlit a protocol amendment for Taysha Gene Therapies’ trial to include patients aged 12 years and older, rather than only adult patients.

The professor in residence of pediatrics at University of California San Francisco discussed factors that will limit the potential first approval, including access and policy.

ASC618 is designed to minimize cellular stress and may increase durability of gene therapy for hemophilia.

uniQure’s AAV9-vector therapy carrying 2 small interfering RNAs targeting GRIK2 was well-tolerated, with a hopeful risk-benefit ratio. A phase 1/2 is set to begin recruitment in late 2023.

The associate professor of ophthalmology at Harvard Medical School discussed early results from a phase 1/2 trial from Mass Eye and Ear and partners.

Review top news and interview highlights from the week ending December 1, 2023.

The senior vice president and head of research at Kyverna Therapeutics discussed the company’s investigational CAR-T therapies, KYV-101 and KYV-201.

The IND clearance sets AMT-191 to be evaluated in a clinical trial alongside the company's gene therapies for ALS and Huntington disease.

The Associate Professor of Medicine at Harvard Medical School discussed upcoming research and strategies to combat present hurdles in the treatment field.

The vice president of discovery at GentiBio discussed the panel she participated in at the inaugural Cell Therapy for Autoimmune Disease Summit.

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

The company also plans to submit an MAA to the EMA in the first half of 2024.

The life science research professional at Stanford University discussed preclinical research she presented at the American Heart Association’s Scientific Sessions 2023.

The FDA has received reports of multiple T-cell malignancies in patients after approved CAR-T treatment.

Pz-cel has a PDUFA date of May 25, 2024.

The CAR-T was already being assessed in the ongoing phase 1a/1b NEXICART-1 clinical trial in Israel ahead of this IND clearance by the FDA.

GC012F recently yielded a 100% ORR in an investigator-initiated trial in patients with newly diagnosed multiple myeloma.

CHMP is expected to give an opinion on the MAA in the second half of next year.

The associate professor of clinical ophthalmology at University of Miami discussed potential future areas of investigation for gene therapy in ocular diseases.

The phase 1 VAN-2201 clinical trial (NCT05657301) is assessing the AAV-delivered gene therapy from Chengdu Origen Biotechnology and Vanotech in 5 dose cohorts of 25 total patients.

Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, spoke about his presentation on gene therapy for cardiologists at AHA’s 2023 Scientific Sessions.

The research associate at the The Texas Heart Institute discussed preclinical research she presented at AHA’s 2023 Scientific Sessions on MSC-derived exosomes.

The chief research officer of the Muscular Dystrophy Association discussed the need for disease-modifying therapies in other muscular dystrophies and programs the association has announced to this end.

Maria Pia Morelli, MD, PhD, assistant professor, MD Anderson Cancer Center, discussed her work on the EVEREST-1 trial of logic-gated CAR T-cell therapy currently being evaluated in colorectal and pancreatic cancers.

Review top news and interview highlights from the week ending November 24, 2023, including coverage from AHA 2023.