Barry J Byrne, MD, PhD, on Making Decisions in the Face of Rapidly Expanding Treatment Options
The chief medical advisor of Muscular Dystrophy Association (MDA) gave his view on treatment decision-making in the world of new options for DMD.
ProKidney Puts Focus on US Trial for T2D and CKD Cell Therapy Rilparencel
The company also reported that it is discontinuing the phase 3 REGEN-016 (PROACT 2) clinical trial evaluating rilparencel in Spain.
BridgeBio Pharma Garners FDA RMAT Designation for BBP-812
The gene therapy is currently being evaluated in the phase 1/2 CANaspire clinical trial.
Around the Helix: Cell and Gene Therapy Company Updates – September 11, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Trying New Approaches to AAV Therapy for Glioblastoma
Xandra Breakefield, PhD, an investigator of neurology at Mass General Research Institute, discussed research from her grad student, Lisa Nieland, presented at the ASGCT 2024 meeting.
John Brandsema, MD, on Balancing Risks and Rewards With Muscular Dystrophy Gene Therapy
The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed how Sarepta’s Elevidys has affected the landscape of care for DMD.
Allogeneic CAR-T Azer-Cel Achieves Complete Responses in Some Patients With Diffuse Large B-Cell Lymphoma
The cell therapy, originally developed by Precision BioSciences, is being evaluated for LBCL by Imugene.
Slowing Tumor Growth in Glioblastoma With Novel AAV Therapy
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.
Higher-Dose Nusinersen Boasts 6-Month Motor Improvement in Spinal Muscular Atrophy
Biogen plans to submit for regulatory approval for a higher dose based on the positive topline data from the phase 2/3 DEVOTE study (NCT04089566).
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
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.
