Real-World Experience With Duchenne Muscular Dystrophy Gene Therapy
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.
CGTLive®’s Weekly Rewind – September 6, 2024
Review top news and interview highlights from the week ending September 6, 2024.
mRNA-CAR-T Descartes-08's Potential for Treating Myasthenia Gravis
Miloš Miljković, MD, the chief medical officer of Cartesian Therapeutics, discussed data presented at ASGCT 2024 from a phase 2a study.
Cartesian Therapeutics Doses First Patient With mRNA CAR-T Descartes-15 in Trial for R/R Multiple Myeloma
Pending the results of the trial, Cartesian plans to eventually evaluate Descartes-15 for the treatment of autoimmune diseases.
Biogen’s Nusinersen Shows Efficacy in SMA Compared With Sham Treatment at Experimental Higher Dose
The data come from the pivotal part B cohort of the phase 2/3 DEVOTE clinical trial.
Ractigen Garners Orphan Drug Designation for Muscular Dystrophy saRNA Therapy RAG-18
Notably, the therapy previously received rare pediatric disease designation from the FDA in July 2024.
Around the Helix: Cell and Gene Therapy Company Updates – September 4, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
REGENXBIO’s MPSII Gene Therapy RGX-121 Continues to Show Efficacy in Long-Term Trial Data
Among patients who received the pivotal dose level, a median reduction of 85% in HS D2S6 levels was seen in the CSF.
FDA Activity Recap: August 2024 Features TCR T-Cell Therapy Approval, REMS Modification, the Lifting of a Clinical Hold, and More
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
Grit Biotechnology’s TIL Therapy GT201 Cleared for US Trial in Advanced Solid Tumors
GT201 previously received IND clearance from China’s Center for Drug Evaluation in July 2023.
Crystal Proud, MD, on Improving Newborn Screening for Spinal Muscular Atrophy
The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, spoke about the enhancements to newborn screening for the disease.
Accelerating Therapy Discovery and Approval With AI
David Dimmock, MBBS, the chief medical officer at Creyon Bio, discussed future applications for the company’s AI-guided discovery platform.
Abata Therapeutics' Treg Therapy ABA-101 Garners FDA Fast Track Designation
The therapy is being developed for the treatment of patients with progressive MS who have imaging evidence of ongoing inflammatory tissue injury and who are HLA-DRB1*15:01 positive.
CGTLive®’s Weekly Rewind – August 30, 2024
Review top news and interview highlights from the week ending August 30, 2024.
The Importance of Precise Reproducibility of AAVs
Scott Jeffers, PhD, the chief technology officer at GenSight Biologics, discussed using Verdot’s FlexiPro system to produce Lumevoq gene therapy.
Orgenesis’ CAR-T ORG-101 Demonstrates Efficacy and Safety in Acute Lymphoblastic Leukemia in Real-World Study in China
Among the treated patients, the complete response (CR) rate in adults was 82% and the CR rate in children was 93%.
A Promising Case Study of Ultra-Rare, AI-Guided, ASO Development
David Dimmock, MBBS, the chief medical officer at Creyon Bio, discussed findings from a patient treated for a TNP02 missense mutation.
Amarna Therapeutics Secures Regulatory Guidance From FDA for Diabetes Gene Therapy AM510
Amarna noted that the meeting, which took place on July 31, 2024, provided the company with feedback on the overall development plan for AM510.
Around the Helix: Cell and Gene Therapy Company Updates – August 28, 2024
AI-Guided ASO Development for Ultra-Rare Diseases
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.
Legend Biotech’s CAR-T Carvykti Approved for R/R MM Indication in China
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.
JW Therapeutics’ Relma-Cell Approved in China for R/R Mantle Cell Lymphoma
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.
The Future of Sickle Cell Disease’s Landscape of Care
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.
Chun-Yu Chen, PhD, on Addressing Hemophilia A With CRISPR/Cas9 mRNA LNP Gene Editing
The research scientist at Seattle Children’s Research Institute discussed findings from mouse research he presented at ASGCT’s 2024 conference.
Galapagos Cleared by FDA to Bring Trial for CAR-T Therapy GLPG5101 in R/R NHL to US
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.
Early Signals of Efficacy With Cystic Fibrosis Gene Therapy
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.
The Research Needed to Bring Gene Therapy to PD
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.
Immunogenicity Outcomes for Val-Rox Reported From Phase 3 GENEr8-1 Study
Among more than 100 patients with hemophilia A, no FVIII-specific responses showed associations with the assessed safety or efficacy parameters.
CGTLive®’s Weekly Rewind – August 23, 2024
Review top news and interview highlights from the week ending August 23, 2024.
CARsgen Finishes Enrolling Patients in Pivotal Trial in China for Gastric/Gastroesophageal CAR-T Satri-Cel
The study was open to patients with advanced GC/GEJ whose disease was not successfully treated with 2 or more previous lines of therapy.
