High-Dose Gene Therapy Slows Huntington Disease Progression
Treated patients had a –0.2 mean change in cUHDRS compared with a –1.0 change in external control data.
Paul Y. Song, MD, on Using Autologous Natural Killer Cells to Treat Parkinson Disease
The chairman and chief executive officer of NKGen discussed the mechanism behind the company’s NK cell therapy SNK01 and promising early results in patients with PD.
Jennifer Taylor-Cousar, MD, MSCS, on Early Signals of Efficacy With Cystic Fibrosis Gene Therapy
The professor of internal medicine and pediatrics at National Jewish Health discussed updated data from the phase 1/2 AEROW trial of 4D-710.
Nkarta Launches Trial for CAR-NK Therapy NKX019 in Lupus and Gains Clearance for Separate Trial in Other Autoimmune Diseases
Ntrust-2 will assess NKX019 in systemic sclerosis, idiopathic inflammatory myopathy, and ANCA-associated vasculitis.
Data Roundup: June 2024 Features Updates from ASCO and in Diabetes, Rare Disease, and more.
Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.
Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC also discussed current strategies with gene therapy administration.
Spur Therapeutics Continues to Make Progress in Evaluation of Gaucher Disease Gene Therapy FLT201
The phase 1/2 GALILEO-1 trial has completed enrollment and a phase 3 trial is planned for 2025.
SPEARHEAD-1 Supports Potential TCR-T Approval for Synovial Sarcoma
The Prescription Drug User Fee Act (PDUFA) target action date is August 4, 2024.
Designing Clinical Trials for Parkinson Disease Gene Therapy Presents Substantial Challenges
Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed unique difficulties of designing clinical trials for PD gene therapy candidates.
Paul Y. Song, MD, on Taking a Holistic Approach to Treating Parkinson Disease
The chairman and chief executive officer of NKGen discussed the potential of SNK01, the company’s autologous natural killer cell therapy, in treating PD.
Interest in Genomic Medicine Research for Heart Disease Continues to Grow
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed trends in precision medicine for cardiovascular indications.
FDA Activity Recap: June 2024 Features Expanded Indication Approval, a CRL, 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.
Ocugen Doses First Patient With Retinitis Pigmentosa Gene Therapy in Phase 3 Trial
The trial's primary endpoint is improvement on Luminance Dependent Navigation Assessment (LDNA).
CGTLive®’s Weekly Rewind – July 5, 2024
Review top news and interview highlights from the week ending July 5, 2024.
Checkpoint Modifier Vaccine Shows Potential to Increase T-cell Response in Patients With HBV
First-in-human data with low dose VRON-0200 were presented at the EASL 2024 congress.
Myrtelle’s Canavan Disease Gene Therapy Selected for FDA START Pilot Program
The therapy, rAAV-Olig001-ASPA, has shown positive clinical benefits in several interim updates from an ongoing, first-in-human phase 1/2 clinical trial (NCT04833907).
Manali Kamdar, MD, on Evaluating Liso-Cel in Mantle Cell Lymphoma by Lines of Therapy, Prior BTKi
The associate professor of medicine at University of Colorado discussed a post hoc analysis study on subgroup analyses of liso-cel response.
Around the Helix: Cell and Gene Therapy Company Updates – July 3, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
IASO Reports Positive First Uses of Eque-Cel for CNS Autoimmunity
The company has reported data in NMOSD, myasthenia gravis, and necrotizing myopathy over the last several months.
Ignacio Mata, PhD, on the Research Needed to Bring Gene Therapy to PD
The 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.
Subhash Tripathi, PhD, on Developing Safe, Specific Engineered Treg Cell Therapy
The senior researcher at Seattle Children’s discussed advantages of engineered Tregs compared with natural Tregs.
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.
