Armin Ghobadi, MD, on Helping Expand CAR T Cells With NT-I7
The associate professor of medicine at Siteman Cancer Center and WUSTL discussed updated data from a study of NT-I7 and tisa-cel presented at ASH 2022.
CGTLive’s Weekly Rewind – December 23, 2022
Review top news and interview highlights from the week ending December 23, 2022.
Gene Therapies Advance in Danon Disease, CLN2 Batten Disease
Both REGENXBIO and Rocket Pharmaceuticals have announced trial updates in their gene therapy programs for rare diseases.
Around the Helix: Cell and Gene Therapy Company Updates – December 21, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Solid Tumor CAR T Collaboration Cleared for Trials
Invectys and CTMC, a joint venture between MD Anderson Cancer Center and National Resilience, announced the therapy and collaboration in June 2022.
Ide-cel Shows Efficacy in Early Relapsed Multiple Myeloma
Patients had an ORR of 83.8% with a median DOR of 15.7 months in data presented at the ASH 2022 meeting.
Radiation-Induced Xerostomia Gene Therapy Shows Promise in Phase 1 Trial
No dose-limiting toxicities or treatment-related serious adverse events were reported among 24 treated patients.
Running With Scissors: The FDA Moves to Tame Gene Therapy Drug Development
With several newly approved therapies hitting the market and an overflowing pipeline, the FDA has stepped in to help keep the burgeoning world of biotech on track.
Steven Pipe, MD, on Continuing Durability of Etranacogene Dezaparvovec in Hemophilia B
The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed new follow-up data from the HOPE-B study presented at ASH 2022.
FDA Approves Ferring's Gene Therapy for Bladder Cancer
The approval follows a CRL that was issued in 2021 due to problems with chemistry, manufacturing, and controls.
CGTLive’s Weekly Rewind – December 16, 2022
Review top news and interview highlights from the week ending December 16, 2022.
B-VEC Continues to Show Durable Wound Healing in Dystrophic Epidermolysis Bullosa
Half of wounds treated with the gene therapy had complete healing at both months 3 and 6 compared to 7% of placebo-treated wounds.
Sham Mailankody, MBBS, on Updates on ALLO-715 CAR T in R/R Multiple Myeloma
The associate attending physician at Memorial Sloan Kettering Cancer Center discussed updated data presented at the ASH 2022 meeting.
Around the Helix: Cell and Gene Therapy Company Updates – December 15, 2022
GPRC5D-Targeted CAR T-Cell Therapy Shows Promising Efficacy After BCMA Therapy in R/R MM
New data from a phase 1/2 trial of BMS-986393 were presented at the 64th Annual ASH Meeting.
David Sallman, MD, on Advantages of Allogeneic CAR T-Cell Therapy in AML
The assistant member of the department of malignant hematology at Moffitt Cancer Center discussed unmet needs in acute myeloid leukemia and how UCART123v1.2 may address these.
Akshay Sharma, MBBS, on Inducing Fetal Hemoglobin in Sickle Cell With CRISPR/Cas9-Edited Stem Cells
The assistant member of the bone marrow transplant department at St. Jude Children’s Research Hospital discussed new data from a phase 1/2 trial presented at ASH 2022.
SPK-8011 Yields Durable FVIII Expression in Hemophilia A
Annualized bleed rate and FVIII infusion rates significantly decreased after infusion with the gene therapy.
Allogeneic CD123 CAR-T Shows Some Clinical Activity in R/R Acute Myeloid Leukemia
Four of 17 patients who received UCART123v1.2 showed clinical benefit.
Gerhard Ehninger, MD, on Beneficial Safety in AML With Rapidly Switchable CAR Therapy
The professor at University Hospital Dresden discussed data presented at the 2022 ASH meeting.
Axi-cel Shows Efficacy in Patients With R/R Aggressive B-Cell Lymphomas Ineligible for HDCT and ASCT
The complete metabolic response rate was 70% 3 months after infusion.
Matthew Frigault, MD, MS, on CART-ddBCMA's Potential in Multiple Myeloma
The study's primary investigator shared his thoughts on where CART-ddBCMA could fit within the treatment landscape.
Novartis' Rapcabtagene Autoleucel Produces Durable Response in Relapsed/Refractory DLBCL
Rates of complete response to the novel CAR-T varied from 67% to 75% across the 4 dosage levels tested.
Aimee C. Talleur, MD, on Predicting Response and Toxicity to Cell Therapy
The assistant member of the bone marrow transplant department at St. Jude Children’s Research Hospital discussed the session she moderated at the ASH 2022 annual meeting.
DdBCMA-Targeted CAR-T Therapy Continues to Show Promise in Multiple Myeloma
Among the 38 patients now dosed and evaluable, the ORR remains at 100%.
Axi-Cel Demonstrates Promising Efficacy and Acceptable Safety in CNS Lymphoma
A complete response rate of 67% was reported and no treatment-limiting toxicity occurred.
Gene Therapy for Sickle Cell Disease Cost Effective in Reducing Health Disparities
A DCE analysis weighed gender-, age-, and disease-severity-specific annual costs as opposed to traditional cost-effectiveness models.
bluebird bio's Lovo-Cel Has Durable Effect in Sickle Cell Disease
Additional data on 2 patients who developed persistent anemia suggests a genetic cause may be behind the serious adverse event.
Neoantigen Reactive T Cell Therapy Shows Early Signs of Efficacy in NSCLC and Melanoma
Achilles Therapeutics presented updated data from CHIRON and THETIS at the 2022 ESMO immuno-oncology congress.
Stem Cell Gene Therapy Show Clinical Improvements in Gaucher Type 1 and 3
AVROBIO plans to initiate a global phase 2/3 trial of AVR-RD-02 in GD3 in the second half of 2023.
