Noah Stansfield

Lisa Nieland, a PhD candidate from Lieden University Medical Center and Breakefield Lab at Mass Gen, discussed her work presented at the ASGCT 2024 meeting.

In honor of World Duchenne Awareness Day, held September 7, several experts have provided their insights on the real-world experience of gene therapy’s use in Duchenne muscular dystrophy.

Miloš Miljković, MD, the chief medical officer of Cartesian Therapeutics, discussed data presented at ASGCT 2024 from a phase 2a study.

Pending the results of the trial, Cartesian plans to eventually evaluate Descartes-15 for the treatment of autoimmune diseases.

The data come from the pivotal part B cohort of the phase 2/3 DEVOTE clinical trial.

Notably, the therapy previously received rare pediatric disease designation from the FDA in July 2024.

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

Among patients who received the pivotal dose level, a median reduction of 85% in HS D2S6 levels was seen in the CSF.

GT201 previously received IND clearance from China’s Center for Drug Evaluation in July 2023.

David Dimmock, MBBS, the chief medical officer at Creyon Bio, discussed future applications for the company’s AI-guided discovery platform.

Scott Jeffers, PhD, the chief technology officer at GenSight Biologics, discussed using Verdot’s FlexiPro system to produce Lumevoq gene therapy.

Among the treated patients, the complete response (CR) rate in adults was 82% and the CR rate in children was 93%.

David Dimmock, MBBS, the chief medical officer at Creyon Bio, discussed findings from a patient treated for a TNP02 missense mutation.

Amarna noted that the meeting, which took place on July 31, 2024, provided the company with feedback on the overall development plan for AM510.

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

David Dimmock, MBBS, the chief medical officer at Creyon Bio, discussed the development of an allele-selective ASO for a single patient with a de novo pathology.

The NMPA’s decision was made with reference to results from the phase 2 CARTIFAN-1 clinical trial (NCT03758417), which took place at multiple sites in China.

The approved indication specifically covers adults with r/r MCL who have previously been treated with at least 2 lines of systemic therapy including BTKis.

Mark Walters, MD, a professor in residence for pediatrics at the Sickle Cell Center of Excellence at the University of California discussed gene therapy’s ongoing transformation of the treatment landscape.

The research scientist at Seattle Children’s Research Institute discussed findings from mouse research he presented at ASGCT’s 2024 conference.

In light of the IND application, Galpagos intends to bring its phase 1/2 ATALANTA-1 clinical trial (NCT06561425), which is currently ongoing in Europe, to the United States.

Jennifer L. Taylor-Cousar, MD, MSCS, a professor of internal medicine and pediatrics at National Jewish Health discussed updated data from the phase 1/2 AEROW trial of 4D-710.

Ignacio Mata, PhD, an associate professor of neurology at the Cleveland Clinic Lerner Institute, discussed the challenges that will need to be overcome to apply gene therapy to a complex neurological disorder like PD.

The study was open to patients with advanced GC/GEJ whose disease was not successfully treated with 2 or more previous lines of therapy.

Brian Kim, MBA, the chief executive officer of Mission Bio, discussed the company’s Tapestri platform for single cell sequencing.

The company expects to launch a study during the first half of next year.

Travis Drow, BS, a research scientist at Seattle Children's Research Institute, discussed mouse model research he presented at ASGCT’s 2024 Meeting.

BMS is seeking to expand liso-cel's indication in the European Union to include adult patients with r/r FL who have previously received at least 2 prior lines of systemic therapy.

Anjali Pradhan, MS, the chief product officer at Mission Bio discussed the company’s Genome Editing Solution.

The company noted that the first 3 patients with enhanced host conditioning have been dosed in the CHIRON and THETIS trials evaluating ATL001.