Myasthenia Gravis Awareness Month 2024: Looking Back at a Year of Progress for CAR-T
In observance of Myasthenia Gravis Awareness Month, held annually in June, we took a look back at a year of progress in bringing CAR-T to this autoimmune disease.
Around the Helix: Cell and Gene Therapy Company Updates – June 12, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
George Tachas, PhD, on Tackling DMD Treatment From Multiple Angles
The lead scientist at Percheron Therapeutics discussed research on antisense oligonucleotide therapies in mouse models of DMD.
OAC Therapy Granted Breakthrough Designation for Myotonic Dystrophy Type 1
AOC 001 previously demonstrated benefit in patients in the phase 2 MARINA trial.
David Suhy, PhD, on Using DNA Nanoplasmids to Detect Cancer
The cofounder and chief scientific officer at Earli discussed the company’s unique approach to cancer diagnosis.
Taysha’s Rett Syndrome Gene Therapy Gets RMAT Designation
The FDA granted the designation after reviewing safety and efficacy data from phase 1 of the trial.
Batten Disease Gene Therapy Continues to Make Progress Despite Setbacks
Ineka Whiteman, PhD, head of research and medical affairs, BDSRA Australia, discussed what has occurred in the field of Batten disease gene therapy in the past year.
Deepak L. Bhatt, MD, MPH, MBA, on Incorporating AI into Genetic Research for Cardiovascular Disease
The director of the Mount Sinai Fuster Heart Hospital discussed how artificial intelligence complements data-driven research.
Jeffrey Chamberlain, PhD, on Helping Progress Cell and Gene Therapy Development
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed the role the ASGCT plays in the field.
NKGen Continues Progress Evaluating SNK01 NK Cell Therapy for Alzheimer Disease
The phase 1/2 trial is proceeding to its phase 2 trial after safety was demonstrated in its first phase.
Jonathan W. Weinsaft, MD, on Integrating Genetic Research into Cardiovascular Medicine
The chief of cardiology at Weill Cornell Medical College discussed the center’s increased focus on genetic research.
FDA Activity Recap: May 2024 Features 2 Expanded Indication Approvals, 2 RMAT Designations, 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.
Verdot's FlexiPro Chrom Platform Helps Address Bioprocessing Needs for Gene Therapy Manufacturing
Sebastien Lefebvre, MSc, the vice president of R&D at Verdot, discussed the company's unique platform.
Viral Immunotherapy Combo Improves Survival in Patients With NSCLC
Candel also recently received orphan drug designation for CAN-3110 for treating recurrent, high-grade glioma.
Jacques Galipeau, MD, on Highlights from ISCT 2024’s Presidential Plenary
The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison shared highlights from the discussion.
Data Roundup: May 2024 Features Updates from ASGCT in Rare Neurological, Retinal Diseases, and more.
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
Vanee Pho, PhD, on Detecting Translocations in Gene Edited Cells
The chief product officer at Mission Bio discussed the company’s Genome Editing Solution.
CGTLive®’s Weekly Rewind – June 7, 2024
Review top news and interview highlights from the week ending June 7, 2024.
CRISPR Therapy Warrants Further Study for Inherited Retinal Diseases
Nine of 14 participants receiving EDIT-101 had meaningful improvements in at least 1 key functional outcome.
Adicet Bio Seeks to Join Clinical Trial Stage for Lupus Therapies
ADI-001 is also being evaluated in a phase 1 study in patients with B-cell non-Hodgkin lymphoma.
Michael Wang, MD, on Liso-Cel's Approval in Mantle Cell Lymphoma
The professor in the Department of Lymphoma/Myeloma at MD Anderson Cancer Center discussed the advantages the newly approved CAR-T has for older, frailer patients.
TCR-T Therapy Cleared for AML Trial in Combination With AlloHSCT
BSB-1001 targets HA-1 miHA and will be evaluated in the 1 phase 1/2a TCX-101 trial.
Robert J. Hopkin, MD, on Looking Deeper into Fabry Disease Biology
The associate professor of clinical pediatrics at Cincinnati Children's discussed further investigations into novel therapies and new research.
RNAi Therapy Reduces Acute Pancreatitis in Chylomicronemia, Meets Phase 3 Primary Endpoint
Other of the company’s RNAi therapies have recently shown promising data in hyperlipidemia and asthma.
Where Are We: Development of Gene and Cell Approaches for Parkinson Disease
According to recent studies and the latest insights provided by experts, early developments in gene and cell therapies show promise for patients living with Parkinson disease, but challenges remain.
Around the Helix: Cell and Gene Therapy Company Updates – June 5, 2024
Steven W. Pipe, MD, on Confirming Efficacy, Safety of Hemgenix Gene Therapy in Hemophilia B Populations
The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed a sub analysis of the HOPE-B trial.
Lete-cel Study in Synovial Sarcoma, MRCLS Meets Primary Endpoint With 40% ORR
Nine responses were ongoing as of the March 2023 cutoff date.
Rawan Faramand, MD, on Evaluating Orca-T in Patients With Acute Myeloid Leukemia
The assistant professor at Moffit Cancer Center discussed the subgroup analysis she presented at ASCO’s 2024 Annual Meeting.
Metastatic Colorectal Cancer CAR-T Therapy GCC19CART Demonstrates Safety and Clinical Activity in US Patients
GCC19CART targets both guanylate cyclase 2C and CD19.
