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Jennifer Buell, PhD, president and chief executive officer, MiNK Therapeutics, discussed the company’s iNKT cell platform.

Both patients dosed experienced a stable increase in cerebrospinal fluid HexA activity.

The chief scientific officer of the CMT Research Foundation discussed the foundation’s mission.

Oliver Van Oekelen, MD, MSc, resident and PhD student, Icahn School of Medicine, Mount Sinai, discussed findings from a retrospective study.

Review top news and interview highlights from the week ending March 4, 2022.

David Taylor, PhD, an assistant professor in the department of molecular biosciences at UT Austin, discussed the Cas9 research conducted between 2 labs at UT Austin.

The senior vice president and head of clinical development at Mustang Bio discussed the importance of Rare Disease Day.

The decision is the latest action in a line of patent interferences over the revolutionary gene-editing technology.

The oncologists from MD Anderson and Memorial Sloan Kettering Cancer Centers discuss sequencing CAR T-cell therapies and other key therapies in patients with B-cell acute lymphoblastic leukemia.

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

The chief scientific officer of FibroBiologics discussed efficacy findings from a phase 1/2 trial.

The cofounder and chief scientific officer of Cartesian Therapeutics discussed the importance of Rare Disease Day.

The therapy was well-tolerated in 15 patients and no clinically relevant liver findings were observed.

The CAR T-cell therapy from Janssen and Legend Biotech is the second to receive approval with an indication for treatment of multiple myeloma.

The professor from National Taiwan University Hospital discussed the importance of Rare Disease Day for raising awareness.

Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.

The president and chief operating officer of Rocket Pharmaceuticals discussed the importance of Rare Disease Day.

The phase 1b KEYNOTE-B79 trial is investigating the allogeneic cell therapy in participants with metastatic colorectal cancer.

The director of the Powell Gene Therapy Center at the University of Florida discussed the importance of Rare Disease Day.

Crystal Proud, MD, outlines advances in technology and research in neuromuscular diseases.

The professor of ophthalmology from Radboud University, The Netherlands, discussed ongoing gene therapy trials in Stargardt disease.

Review top news and interview highlights from the week ending February 25, 2022.

An interim safety review of 81 patients informed the DSMB's recommendation for the trial to continue.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the organization’s initiative to advance genetic testing.

Cory Nicholas, PhD, cofounder and chief executive officer, Neurona Therapeutics, discussed the company’s lead program in mesial temporal lobe epilepsy.

CT103A is co-developed by IASO Biotherapeutics and Innovent Biologics.

The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed new efficacy findings from the HOPE-B study.

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