Kyverna’s CAR-T KYV-101 Gets FDA IND Clearance for Trial in Scleroderma
Kyverna is planning to conduct a phase 1/2 clinical trial (KYSA-5; NCT identifier pending) in patients with scleroderma.
BioCardia Targets Smaller Population of Heart Failure With Proposed Modified Study Protocol
The company aims to assess CardiAMP in patients with NT-proBNP over 500 pg/ml at baseline with a modified composite efficacy endpoint.
Around the Helix: Cell and Gene Therapy Company Updates – October 11, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
CGT Therapies More Likely to Gain Approval for Hematological Malignancies, Orphan Diseases
A NEWDIGS analysis compared clinical trial success rates from 1988 to 2020.
Alfonso Sabater, MD, PhD, on Treating Ocular DEB With B-VEC
The associate professor of clinical ophthalmology at University of Miami discussed his experience using the gene therapy in a compassionate use program.
AviadoBio Begins Trial for Frontotemporal Dementia Gene Therapy AVB-101
The open-label, multicenter, ascending dose trial is expected to treat approximately 9 patients in total across 2 dose cohorts.
FDA Fast Tracks Avenge Bio’s Ovarian Cancer Cell Therapy AVB-001
AVB-001 is currently being evaluated in a phase 1/2 clinical trial (NCT05538624) that was initiated in January 2023.
Antitumor Activity and Bone Regeneration: Tackling Multiple Angles of Osteosarcoma
Fiona Freeman, PhD, assistant professor, University College Dublin, discussed her research into microRNA-29b in suppressing tumor growth and promoting bone remodeling in mice models.
Complex Neurological Disorders Lie on the Horizon of Gene Therapy Research
Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed what may be next for the field of gene therapy after early successes in single-mutation disorders.
Thomas Povsic, MD, PhD, on Ongoing Research With XC001 Angina Gene Therapy
The interventional cardiologist and professor, Duke University School of Medicine, discussed other populations that may benefit from XC001 and the challenges and opportunities with a surgical delivery of treatment.
Leucid Bio’s NKG2D-directed CAR-T LEU011 Cleared for Solid Tumor Clinical Trial in the UK
In September 2023, LEU011 was granted an Innovation Passport for the treatment of solid tumors expressing NKG2D ligands.
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
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.
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