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.
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The findings, presented at ASGCT’s 2023 conference, included an analysis of biomarker data in relation to disease severity.
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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,
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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.
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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.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Among 27 evaluated patients with relapsed/refractory B-cell acute lymphoblastic leukemia, 18 achieved a complete response.
Krystof Bankiewicz, MD, PhD, on Dialing Back Progression of Parkinson Disease With GDNF Gene Therapy
The professor at Ohio State University and University of California – San Francisco discussed updated data from a phase 1 study of GDNF gene therapy in patients with PD.
Updated data from a phase 1 study of PCXR201, formerly known as Flexion’s FX201, were presented at the 2023 ASCGT Annual Meeting.
Vitalgen’s gene therapy was generally well-tolerated with no serious adverse events reported.
Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, discussed the need to reevaluate the goal posts used to determine whether gene therapies for eye indications come to market.
The clearance of the CTA is the latest in a series regulatory decisions enabling Decibel Therapeutics’ international clinical development plans.
The cofounder and chief scientific officer of Xcell Biosciences discussed the company’s efforts to introduce new technologies for improving cell therapy development and manufacturing.
CTO1681, which is designed to lower NF-kB signaling without eliminating it altogether, was previously evaluated for safety in a phase 1 clinical trial in healthy adults.
The assistant professor of stem cell transplantation at MD Anderson Cancer Center discussed positive data in ccRCC that he believes should excite the solid tumor field as a whole.
Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, discussed new real world data regarding Spark Therapeutics’ Luxturna that was presented at ARVO’s 2023 conference.
Committee members were mostly split, ultimately voting 8–6 in favor of the benefit-risk profile of SRP-9001 supporting accelerated approval.
Review top news and interview highlights from the week ending May 12, 2023.
The director of therapeutic genome engineering, St. Jude Children’s Research Hospital discussed recent research published in Nature Biomedical Engineering.
