Around the Helix: Cell and Gene Therapy Company Updates – December 15, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
Around the Helix: Cell and Gene Therapy Company Updates – December 7, 2022
BioCardia Follows up CardiAMP With CardiALLO Clearance for Heart Failure
The allogeneic mesenchymal stem cell therapy trial will enroll patients ineligible for treatment with the autologous CardiAMP.
Genome-Edited Cell Therapy Reduces VOEs in Sickle Cell Disease
The first 2 participants dosed are free of vaso-occlusive events as of 5 and 1.5 months of follow-up after treatment with EDIT-301.
Leveraging Cell Therapy Advances for Treating Hodgkin Lymphoma, Other Cancers
Ivan Horak, MD, discussed Tessa’s cell therapy platforms and the ACTION clinical trial.
Neural Cell Therapy Reduce Seizures in MTLE
Updated data were presented at the AES annual meeting in Nashville.
Nkarta's Allogeneic CAR T Shows Clinical Responses in Non-Hodgkin Lymphoma
Patients with non-Hodgkin lymphoma in Nkarta's phase 1 study had a 75% complete response rate.
Nirav N. Shah, MD, on Unmet Needs With CAR T-Cell Therapies in Hematologic Malignancies
The associate professor from the Medical College of Wisconsin discussed how to address unmet needs with CAR T-cell therapies.
Retinitis Pigmentosa Gene Therapy Cleared for Trials
Initial safety data from the PRODYGY study are expected in 2023.
Multiplex-Base Edited CAR T Therapy Cleared for Trial in T-ALL After Hold
Beam Therapeutics provided data on genomic rearrangement, off-target edits and from a cytokine independent growth assay to the FDA in November 2022.
CGTLive’s Weekly Rewind – December 2, 2022
Review top news and interview highlights from the week ending December 2, 2022.
Allogene’s CAR T Therapies Show Antitumor Activity in Solid and Liquid Tumors
Updated data from 3 of the company’s programs were presented at an R&D showcase.
Leber Congenital Amaurosis 5 Gene Therapy Cleared for Trials
OPGx-001 is the first of Opus Genetics’ gene therapy candidates for treating LCA to enter clinical trials.
OTOF Hearing Loss Gene Therapy Granted Orphan Drug Designation
Sensorion indicated it is still on track to submit a clinical trial application in the first half of next year.
