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The associate professor of otolaryngology from Harvard Medical School discussed investigating gene therapy for DFNB9 deafness.

An investigator-initiated trial presented positive data at the ESGCT annual meeting in October 2023.

Review top news and interview highlights from the week ending January 26, 2024.

Robert M Califf, MD, MACC, the commissioner of food and drugs at the FDA, discussed how 2023 was an exemplary year for how far the field has come over the past few decades.

The internal medicine resident physician at University of Kansas Medical Center also discussed highlights from the ASH 2023 meeting.

The chief medical advisor of the Muscular Dystrophy Association discussed research he is working on with the goal of increasing eligibility for gene therapies and opening the door to redosing.

BriaCell also shared specific positive data from one patient with proptosis and ocular pain.

The European Medicines Agency Committee for Advanced Therapies will likewise hold a SAG-O meeting regarding the Type II variation application for the CAR-T in the EU.

Speech perception was also greatly improved in the children with hearing recovery.

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

The chief medical advisor of the Muscular Dystrophy Association discussed what seasoned clinicians and newcomers to the field alike can look forward to at the 2024 MDA Meeting.

The immune therapy is a replication-incompetent human tumor cell line designed to conjugate to and prime resting natural killer cells.

Aruna Bio stated that it expects to begin a phase 1b/2a clinical trial within the first half of this year.

The associate professor at Fred Hutch Cancer Center discussed trends he observed in the field in 2023 and at ASH 2023.

The postdoctoral researcher at Laboratory for Translational Cancer Immunology, Ludwig-Maximilians-Universität München, discussed research he was excited to see at ASH 2023 and in the field in general.

The agency does not have plans for an advisory committee meeting to discuss obe-cel, which is an investigational autologous CD19-directed CAR-T therapy.

The trial is assessing satri-cel CAR T-cell therapy, which is currently on clinical hold due to CMC concerns.

Sharon Hesterlee, PhD, the chief research officer of MDA, discussed highlights of 2023’s meeting and exciting new things to look forward to at the upcoming 2024 meeting.

In light of this major decision by the agency, CGTLive™ decided to reach out to several experts to get their thoughts on how exa-cel could impact the landscape of care for TDT.

PepGen’s ongoing multiple ascending dose trial is expected to read out initial data in mid-2024.

The president and head of R&D at Taysha Gene Therapies discussed his expectation that younger patients my see even better results with the company’s gene therapy than the adult patients treated so far.

Kiromic announced that the first patient had been dosed with the gamma delta cell therapy in December 2023.

The Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute discussed unmet needs that remain after gene therapy approval for SCD.

TAC01-CLDN18.2 integrates TAC, Triumvira’s proprietary chimeric receptor that is intended to activate and direct T-cells against tumor cells.

Review top news and interview highlights from the week ending January 19, 2024.

Tami John, MD, a clinical associate professor at Stanford Medicine, also discussed ongoing trends in sickle cell disease research.

Dose level 3 showed the most efficacy and has been selected for the phase 2 portion of the trial.

The chief hematology/oncology fellow at University of Chicago discussed further research his center is working on with cell therapy in B-cell acute lymphoblastic leukemia.

Hemogenyx originally submitted the IND to the FDA in May 2023 with the intention of gaining clearance for a phase 1 clinical trial in acute myeloid leukemia.

The cofounder and CEO of Neurona Therapeutics discussed the company’s goals to evaluate the cell therapy in more types of epilepsy and potentially even in Alzheimer disease.