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Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed future plans for the expanded applications of the allogeneic regenerative neural cell therapy.

The Prescription Drug User Fee Act (PDUFA) target action date has been set for August 4, 2024.

Review top news and interview highlights from the week ending February 2, 2024.

The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham discussed work the National Alliance of Sickle Cell Centers is conducting.

The ongoing, multistage phase 1/2/3 Cyprus2+ study (NCT04884815) expects to read out initial safety and efficacy data in the first half of 2024.

Vor Bio recently also announced data from the phase 1/2 trial of trem-cel.

The Associate Professor of Medicine at Harvard Medical School discussed CAR T’s journey to treating solid tumors.

Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed the ongoing first-in-human clinical trial evaluating the cell therapy for drug-resistant mesial temporal lobe epilepsy.

The associate professor of clinical ophthalmology at University of Miami discussed the first topical application of gene therapy to the eye.

Kyverna has also announced data on its proprietary CAR-T manufacturing process, Ingenui-T.

Efficacy and safety results from the RESKUE clinical trial will be presented at at the WORLDSymposium 2024 on February 9.

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.