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This is the second IND clearance for CABA-201, which previously received clearance for evaluation in systemic lupus erythematosus, and now for active idiopathic inflammatory myopathy—also known as myositis.

The scientist at Senti Biosciences discussed developing small molecule-regulated gene circuits.

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

The promising preclinical data, observed in an accurate model of brain metastases, support initiation of a phase 1 trial.

The chief scientific officer of LocanaBio discussed preclinical data with an AAV-delivered, snRN, exon 51 skipping approach presented at ASGCT 2023.

The BLA has a PDUFA action date of November 25, 2023.

The assistant professor of microbiology at Penn Medicine discussed research into characterizing type 1 interferon and EGR2 signaling and their effects on CAR T-cell activation.

Florian Eichler, MD, a neurologist at Massachusetts General Hospital, discussed efficacy data from the CANaspire clinical trial that he presented at ASGCT 2023 showing reductions in urine NAA levels.

The Type II variation application for ciltacabtagene autoleucel in adult patients with relapsed and lenalidomide-refractory multiple myeloma is supported by data from the phase 3 CARTITUDE-4 trial.

Alliance for Cancer Gene Therapy (ACGT) awarded The Edward Netter Leadership Award to Crystal Mackall, MD, of Stanford University.

There were no dose-limiting toxicities, serious adverse events, nor infusion-related reactions among 6 patients treated with ENCell’s investigational EN001 therapy.

The principal investigator at Seattle Children’s Research Institute discussed her lab’s preclinical research on nonviral delivery methods for gene editing tools in the context of treating hemophilia A.

Lena Winstedt, PhD, Hansa Biopharma, discussed imlifidase, which is being investigated in combination with gene therapies from Sarepta and Genethon.

The chief scientific officer at REGENXBIO discussed multiple presentations given by the company at the 2023 ASGCT meeting.

Review top news and interview highlights from the week ending May 26, 2023.

All evaluable patients had improvements in natriuretic peptides, left ventricular volume, and NYHA class.

The principal investigator at Seattle Children’s Research Institute discussed the preclinical research on delivery methods for genomic medicines that her lab presented at ASGCT’s 2023 meeting.

There were no serious adverse events reported and no patients discontinued from the study.

All evaluable patients had significant reductions in daily cornstarch intake at 1 year and at last visit during long-term follow-up.

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

The CEO, founder, and president of Siren Biotechnology discussed the preclinical data she presented at ASGCT’s 2023 meeting on the use of AAV immuno-gene therapy for high-grade gliomas.

The investigational gene therapy, also known as SRP-9001, was recently backed by the agency’s advisory committee in a tight decision. Its new deadline for review is June 22, 2023.

The chief scientific officer at Candel Therapeutics discussed late-breaking data presented at ASGCT 2023.

The trial compared data from external controls including a recent domagrozumab null study and a propensity score weighted comparison.

The senior investigator at the National Cancer Institute Center for Cancer Research discussed updated data from a phase 1/2 trial presented at ASGCT 2023.

Alliance for Cancer Gene Therapy (ACGT) recently hosted their 2023 Summit, a scientific gathering of cancer experts to discuss challenges and opportunities with cell and gene therapy.

The approval was based on data from the phase 1/2 EPCORE NHL-1 clinical trial.

The professor of internal medicine at UT Southwestern Medical Center shared his perspective on the current landscape of CAR-T cell therapy in the care of patients with myeloma.