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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.

The SARM1 gene is a key driver in the damage that ultimately leads to impaired vision.

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 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.

The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed mitigation strategies in trials and clinic.

The trial design was informed by both patient and physician perspectives.

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed the advantages of AAV gene therapies.

The pause follows a fatal serious adverse event in the first patient treated in the highest dose cohort.

The trial, sponsored by Mustang Bio, will continue to enroll patients and doses will be escalated to 300 million cells.