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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.

M. Peter Marinkovich, MD, and Alfonso L. Sabater, MD, PhD, offered their respective perspectives on the recent FDA approval of the topical and redosable gene therapy for DEB.

Diane M. Simeone, MD, discussed the phase 1/2 EVEREST-1 trial, which seeks to assess the novel CAR T-cell therapy A2B530 in patients with solid tumors—hopefully progressing one-and-done engineered therapies in this population.

The chief scientific officer of Sangamo Therapeutics discussed the company’s AAV capsid evolution research.

May is Brain Cancer Awareness Month, and the ACGT is raising awareness of the potential of the human immune system when combined with cell and gene therapy to defeat brain tumors.

The findings, presented at ASGCT’s 2023 conference, included an analysis of biomarker data in relation to disease severity.

The neurologist at Massachusetts General Hospital discussed BBP-812, an investigational AAV vector-based gene therapy being evaluated for Canavan disease in a phase 1/2 trial.

The senior research fellow at Fred Hutch Cancer Center discussed findings from the PLAT-08 trial presented at ASGCT 2023.

All 8 patients with light chain amyloidosis who received HBI0101 responded.

The most robust transduction in nonhuman primates was seen with ICV delivery of pooled PM4.AAV.mRFP and AAV2.mNG gene therapy,

Review top news and interview highlights from the week ending May 12, 2023.

The Krystal Biotech treatment, marketed as Vyjuveck, is now the first topical gene therapy for patients with DEB—marking a milestone in the paradigm of care.

The professor of medicine at University of California San Diego discussed new research from her lab presented at the 2023 ASGCT meeting.

The chief scientific officer of Sangamo Therapeutics discussed data from 2 of the company’s programs that were presented at ASGCT’s 2023 conference.

The senior scientist at Eterna Therapeutics discussed the company’s numerous presentations at ASGCT’s 2023 conference.

Data trends suggesting stabilization of disease were seen in patients with Tay-Sachs, but more research is needed, especially for those with Sandoff disease.

Updated data from the RESTORE study on MCO-010, Nanoscope Therapeutics' investigational gene therapy, were presented at the 2023 ASGCT meeting.

The vice president of research at Prime Medicine discussed a preclinical study on a novel autologous HSCT treatment that was presented at ASGCT’s 2023 annual meeting.

Following implantation with CellGenTech’s LCAT-GMAC, the patient’s serum LCAT activity increased by approximately 50% of baseline.

ImmTOR induces immune tolerance and may reduce or eliminate Nab formation, allowing potential redosing of gene therapy.