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The company also shared that it has manufactured sufficient IDP-023 to supply the Phase 1 clinical trial through the second half of 2024.

The clinical professor in the Department of Human Genetics at University of Texas Rio Grande Valley discussed how a personalized gene editing approach may help patients avoid development of FVIII inhibitors.

FT825 is an iPSC-derived multiplexed-engineered, CAR T-cell therapy targeting HER2 with a novel antigen binding domain.

The chairman, chief executive officer, and cofounder of Ocugen discussed programs including OCU400 and OCU410.

SRP-5051 is an investigational peptide-conjugated phosphorodiamidate morpholino oligomer.

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed the organization’s research imperatives of 2024.

The multinational, open-label EMERGENE study will seek to enroll 15 patients with LGMD2E/R4 in total.

The chief medical advisor of the Muscular Dystrophy Association discussed how directed evolution may help develop AAV capsids better suited for treating neuromuscular diseases.

Sana also recently announced data from the first patient treated with its CD19-targeted CAR.

Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed NRTX-1001, the company’s allogeneic regenerative neural cell therapy currently being evaluated in MTLE.

The trial will recruit patients aged 6 to 31 months with the intent of treating them while auditory system plasticity is optimal.

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.