Aligning With Regulatory Expectations as an Important Aspect of Gene Therapy Trial Design
Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed the unique aspects of clinical trials for gene therapies that need to be considered before initiation.
CGTLive’s Weekly Rewind – October 6, 2023
Review top news and interview highlights from the week ending October 6, 2023.
REGENXBIO’s DMD Gene Therapy Well-Tolerated, Efficacy Signals Promising
The AFFINITY trial is set to dose patients at dose level 2 by the end of 2023.
Tenaya’s Trial of Hypertrophic Cardiomyopathy Gene Therapy TN-201 Doses First Patient
The patient received the trial’s low dose of TN-201 at the Cleveland Clinic's Hypertrophic Cardiomyopathy Center, in Cleveland, Ohio.
First Patient With Adrenomyeloneuropathy Treated In Higher Dose Cohort of PROPEL Trial With SBT101 Gene Therapy
The move was carried out in accordance with a recommendation for dose-escalation made by the trial’s independent Data Safety Monitoring Committee.
Milwaukee Teachers Are Ready to Take Biotech Training to the Classroom
The first official participants of the expanded Biotech Discovery Labs program are eager to bring CGT experiments to their students.
Thomas Crawford, MD, on Taking a Closer Look at Nusinersen Data
The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed the updated analyses of the NURTURE study that affected the big picture of the data.
Gene Therapy Increases GCase Activity in Patients With Gaucher
The updated data are from the first 2 patients treated in the phase 1/2 GALILEO-1 trial of FLT201.
Around the Helix: Cell and Gene Therapy Company Updates – October 4, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Capricor Aligns With FDA on DMD Cell Therapy CAP-1002's Trial in Type-B Meeting
The company stated that based on the meeting, the trial’s primary end points will remain the same.
Patients With Type 1 Diabetes Independence Achieve Insulin Independence With Islet Cell Therapy
All patients showed improved glycemic control and a reduction in insulin independence.
Gamida Cell’s Omisirge Administered to First Patient in Commercial Setting
The company also announced it has onboarded 15 transplant centers in the United States for the use of omidubicel, thus exceeding the goal it had set for itself for 2023.
New FDA Pilot Program Will Provide Hands-On Regulatory Guidance for Cell and Gene Therapy Trials in Rare Disease
Sponsors who hope to participate will be able to apply from January 2 to March 1, 2024.
David Boyer, MD, on Promising Efficacy of Suprachoroidal Gene Therapy in nAMD
The senior partner at Retina Vitreous Associates Medical Group discussed efficacy and safety data from the phase 2 AAVIATE trial.
Gracell’s BCMA/CD19 CAR-T GC012F Achieves 100% Stringent CR Rate in Newly Diagnosed Multiple Myeloma
Notably, no patients experienced cases of immune effector cell-associated neurotoxicity syndrome.
2023 AcCELLerate Forum Encourages Discussion on Cell Therapy Landscape, Management, Future
The partnership between ASTCT, CIBMTR, and NMDP is a 2-day forum held October 2-3.
Rocket’s LAD-I Gene Therapy BLA Accepted for Priority Review
The therapy has a PDUFA date of March 31, 2024.
Taysha Doses Second Patient With Rett Syndrome With TSHA-102, per IDMC Recommendation
Both the second and first patient dosed in the study received the lower of the 2 doses of the gene therapy that Taysha Gene Therapies is evaluating in the phase 1/2 REVEAL trial.
Epigenetic Therapy Shows Signs of Efficacy in MYC-Expressing Solid Tumors
The new data are from the phase 1/2 MYCHELANGELO trial of OTX-2002, an mRNA OEC therapy, in patients with hepatocellular carcinoma and other MYC-expressing tumors.
Brian Van Tine, MD, PhD, on Looking Ahead on Cell Therapy for Sarcomas
The professor of medicine and pediatrics at Washington University in St. Louis discussed future challenges and exciting research in the sarcoma field.
News and Expert Insights in Rare Cancer Cell Therapy for Rare Cancer Day
In observance of Rare Cancer Day, held annually on September 30, catch up on the past few months’ news and expert insights related to cell therapies in development for these indications.
CGTLive’s Weekly Rewind – September 29, 2023
Review top news and interview highlights from the week ending September 29, 2023.
Dose-Limiting AAV Toxicity Responsible for Cure Rare Disease’s DMD Trial Death
Unforeseen complications may arise when treating older patients with gene therapy.
Despite the Long History of Gene Therapy Research, Crossing the Blood-Brain Barrier Remains a Challenge
Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed the history and current state of gene therapy research for neurological indications.
Atamyo Therapeutics Submits CTA in Europe for Limb-Girdle Muscular Dystrophy Type 2C/R5 Gene Therapy
Atamyo has received $8.6 million in nondilutive financing from France 2030 for ATA-200's development.
Dilsher Dhoot, MD, on a Promising Start With Diabetic Retinopathy Gene Therapy RGX-314
The adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California discussed new data from the ALTITTUDE trial.
Axi-Cel Shows Efficacy in Patients With R/R LBCL Ineligible for ASCT
Around half of patients with R/R large B-cell lymphoma are considered ineligible for autologous stem cell transplant and are thus ineligible for the approved indication of Yescarta.
FDA AdComm Votes Down BrainStorm Cell Therapeutics’ ALS Cell Therapy NurOwn
Committee members were overwhelmingly against the available data supporting NurOwn as an effective treatment for mild to moderate ALS, with 17 no votes, 1 yes vote, and 1 abstention.
Around the Helix: Cell and Gene Therapy Company Updates – September 27, 2023
Deborah Phippard, PhD, on the Future of Gene Therapy in Neurology
The chief scientific officer of Precision for Medicine discussed what lies on the horizon for gene therapies directed at neurological indications, such as Alzheimer disease and Parkinson disease.
