Cartesian Therapeutics’ mRNA CAR-T Descartes-08 Continues to Show Efficacy in Phase 2b Myasthenia Gravis Trial
Cartesian also announced that the first patient has been dosed in a separate clinical trial evaluating the CAR-T therapy in systematic lupus erythematosus.
Eque-cel Demonstrates High PFS in Newly Diagnosed, High-Risk Multiple Myeloma
Patients in FUMANBA-2 received eque-cel after being ineligible for ASCT after 4 cycles of induction therapy.
Ignacio Mata, PhD, on the Challenges of Developing New Therapies in Parkinson Disease
The associate professor of neurology at the Cleveland Clinic Lerner Institute pointed out that genetic forms of PD may be the best place to start for new therapeutic research.
Navigating a Novel Therapy Frontier: Gene Therapy's Stunted Impact on Leukodystrophies
CGTLive takes a look at the path gene therapies have blazed in leukodystrophies, the challenges they’ve faced, and the road they’ve paved.
Wave Life Sciences' Antisense Oligonucleotide WVE-003 Demonstrates Ability to Lower Mutant Huntingtin in Phase 1b/2a Trial
The placebo-controlled multidose portion of the SELECT-HD trial included 23 patients with Huntington Disease in total.
Mark Walters, MD, on the Future of Sickle Cell Disease’s Landscape of Care
The 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.
TScan’s TCR-Ts Get RMAT Designation for Hematologic Malignancies
Eight patients receiving either TSC-100 or TSC-101 have been disease-free for a median of 10 months of follow-up.
Taysha’s Rett Syndrome Gene Therapy TSHA-102 Continues to Show Promise in Adult and Pediatric Patients
Various improvements were seen in 2 adult patients and 2 pediatric patients treated in separate clinical trials.
Jacques Galipeau, MD, on International Insights Into Cell and Gene Therapy Development
The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison emphasized similar variables in the way of therapy uptake regardless of country.
CGTLive®’s Weekly Rewind – June 28, 2024
Review top news and interview highlights from the week ending June 28, 2024.
FDA Issues CRL for Rocket’s LAD-I Gene Therapy
The FDA has requested limited additional CMC information.
Gene Transfer Versus Gene Editing Approaches in Cardiology
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed the company’s decision to supplement the gene transfer programs in its pipeline with gene editing programs.
Sharif Tabebordbar, PhD, on Improving In Vivo Gene Editing for DMD
The cofounder and chief executive officer of Kate Therapeutics discussed research that awarded him an Outstanding New Investigator at the ASGCT 2024 meeting.
REGENXBIO Set to Evaluate Duchenne Muscular Dystrophy Gene Therapy RGX-202 in Patients Aged 1 to 3 Years Old
The company has begun enrolling participants in a new cohort for younger patients in the phase 1/2 AFFINITY DUCHENNE clinical trial.
Intellia Successfully Redoses CRISPR Gene Editing Therapy
Three participants with ATTR amyloidosis have received follow-on doses of NTLA-2001 with no serious complications for up to 3 years of follow-up.
Around the Helix: Cell and Gene Therapy Company Updates – June 26, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Deborah Phippard, PhD, on the Challenges of Evaluating Gene Therapy for Parkinson Disease
The chief scientific officer of Precision for Medicine discussed unique difficulties of designing clinical trials for PD gene therapy candidates.
Vertex’s Diabetes Cell Therapy on Track to Meet Endpoint of Eliminating Severe Hypoglycemic Events
All 3 participants who have follow-up of at least 1 year have met the endpoint so far.
Anixa Biosciences Wraps Up Dosing of Second Cohort in Ovarian Cancer CER-T Trial
The company has dosed the sixth patient in the study, and expects to move onto the third cohort pending continued safety at 1 month posttreatment.
Kyverna’s CAR-T KYV-101 Shows Some Durable Responses in Autoimmune Disease, With One Relapse Reported
One patient is considered disease-free at 1 year posttreatment, but another patient’s disease relapsed at 5 months.
Omid Hamid, MD, on Assessing TIL Combination Therapies, Expanding Past Melanoma
The professor from Cedars Sinai discussed further research being conducted in the field after lifileucel’s approval.
Aurion’s Cell Therapy Wins RMAT, Breakthrough Therapy Designation for Corneal Edema
The CLARA trial has also completed enrollment of participants with corneal edema secondary to corneal endothelial disease.
Nathan Yozwiak, PhD, on Collaboration for Cell and Gene Therapy Development
The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed work the center is engaged in with cell and gene therapy.
enGene Tackles Nonmuscle-Invasive Bladder Cancer With Expansive Phase 1/2 LEGEND Trial for Gene Therapy EG-70
With the company having recently announced that it intends to expand the clinical trial with a new cohort, CGTLive® has decided to take a closer look at this ongoing study.
Huntington Disease Gene Therapy Nets RMAT Designation
AMT-130 has shown some evidence of dose-dependent clinical benefits in treated study participants.
Gene Therapy Improves Body Composition in Mice Models of Obesity Alone and After Semaglutide Withdrawal
Fractyl Health is conducting IND-enabling studies for Rejuva.
Continuing Innovation on Modality-Specific Technology for Cardiovascular-Targeted Genomic Medicines
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed the company’s research on capsids, promoters, and manufacturing improvements.
Genethon’s Duchenne Muscular Dystrophy Gene Therapy GNT0004 Produces Expression of Microdystrophin in Phase 1/2/3 Trial
For the 3 patients who were treated at the higher dose, immunohistochemistry showed that a mean of 54% of muscle fibers were expressing microdystrophin.
Salvador Rico, MD, PhD, on Developing Gene Regulation Therapy for Dravet Syndrome
The chief medical officer of Encoded Therapeutics overviews the company’s timeline and strategy for its gene regulation program for Dravet syndrome.
Neurona’s Focal Epilepsy Cell Therapy NRTX-1001 Garners FDA RMAT Designation
NRTX-1001 consists of human interneurons that provide long-term secretion of gamma-aminobutyric acid, an inhibitory neurotransmitter, which is expected to repair neural networks.
