First Patient with B-cell Lymphoma Dosed With Allogeneic NK CAR T Therapy
The FDA cleared Century’s IND for CNTY-101 in August 2022.
Around the Helix: Cell and Gene Therapy Company Updates – February 8, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Preclinical AMD Gene Therapy Shows Potential for Other Retinal Diseases
The new findings are consistent with previous research, in which ophNdi1 demonstrated benefit in AMD models.
Terese Hammond, MD, on Evolving Treatments for ARDS and More With Immunotherapy
The director of the CCU/ICU at Saint John’s Health Center discussed a possible more collaborative approach to treating patients in the future.
Danon Disease Gene Therapy Granted RMAT Designation by FDA
A pivotal phase 2 clinical trial for RP-A501 is expected to initiate in Q2 2023.
Jakub Svoboda, MD, On Improving CR Rates With Next-generation CAR T Therapies
The associate professor of medicine from University of Pennsylvania discussed advantages of huCART19-IL18 in NHL and CLL.
Allogeneic CAR-T Shows Promise in R/R Multiple Myeloma
Patients treated with ALLO-715 achieved an ORR of 55.8%.
Sham Mailankody, MBBS, on Further Research With ALLO-715
The associate attending physician at Memorial Sloan Kettering Cancer Center discussed further research that remains to be conducted with the allogeneic cell therapy.
Dual-Targeting, FasTCAR-T Therapy Cleared for Phase 1b/2 Trial in R/R Multiple Myeloma
GC012F recently showed efficacy in newly-diagnosed MM in investigator-initiated trials.
IND for Gene Replacement Therapy for Inherited Retinal Dystrophy Cleared by FDA
The company is developing a one-time, non-AAV2 gene replacement therapy to restore, treat, and prevent blindness of patients with RPE65 mutation-associated retinopathies.
David Sallman, MD, on Updates From the AMELI-01 Trial of UCART123v1.2 for AML
The assistant member of the department of malignant hematology at Moffitt Cancer Center discussed updated data on the allogeneic CAR T therapy, UNICART123v1.2.
FDA Blocks 4D’s Gene Therapy Program for Fabry, Clears IND for Diabetic Macular Edema
The clinical hold comes a few weeks after the company announced it was stopping enrollment in the phase 1/2 clinical trial.
Raj Mehra, PhD, and Krishna Subramanian, PhD, on Striving For Disease-Modifying Therapies in Parkinson Disease
Mehra and Subramanian discussed preclinical research with the investigational gene therapy SLS-004.
Gene Therapy Trial Doses First Patient With Wet AMD in China
The phase 1 clinical trial is the second of Frontera’s trials to begin dosing in 2023.
Phase 2b Myasthenia Gravis RNA CAR-T Trial Doses First Patient
Cartesian Therapeutics’ Descartes-08 will be administered as 6 weekly infusions and does not require preconditioning chemotherapy.
Gerhard Ehninger, MD, on Managing Toxicities With Switchable CARs in AML
The professor at University Hospital Dresden discussed the positive safety profile of Unicar-T-CD123.
Otoferlin-Related Hearing Loss Gene Therapy Cleared for UK Clinical Trial
Decibel Therapeutics expects the trial for DB-OTO to begin in the first half of 2023.
Around the Helix: Cell and Gene Therapy Company Updates – February 1, 2023
Giant Axonal Neuropathy Gene Therapy Needs Double-blind Trial Before BLA Submission
The dosing of additional patients in a double-blind, placebo-controlled design will help support a BLA submission for TSHA-120.
Remestemcel-L Cell Therapy Makes Comeback With Resubmitted BLA for Pediatric GvHD
Mesoblast is resubmitting its BLA with new CMC, survival, and mechanism of action data on the therapy and phase 3 trial.
NK Cell Therapy/Rituximab Combination Fast Tracked for B-cell Non-Hodgkin Lymphoma
AB-101 is also being investigated with the innate cell engager AFM13 for CD30 lymphomas.
Working Toward a Single Dose Gene Therapy for Type 1 Gaucher Disease
Olga Uspenskaya, MD, PhD, vice president, clinical development, Prevail Therapeutics, discussed the PROCEED trial of PR001.
Senti Bio Prioritizes Logic Gated Cell Therapies
Positive preclinical data related to SENTI-202 and SENTI-401 were presented at conferences last year.
Multi-characteristic Opsin Gene Therapy Gets Second Fast Track for Retinal Degenerative Diseases
MCO-010 is in phase 2 clinical trials for treating retinitis pigmentosa and Stargardt disease.
Rajni Agarwal-Hashmi, MD, on Safety Advantages of Gene Therapy in Fanconi Anemia Over SOC
The professor of pediatrics at Stanford Cancer Institute discussed recent data from clinical trials of the lentiviral gene therapy RP-L102.
Solid Tumor Cell Therapy Combination Trial Cleared to Continue
There were no deaths or safety signals identified by the DSMB for the 3 treated patients in cohort 1.
Allogeneic CAR T Cleared for B-cell Malignancy Trials
Sana Biotechnology also plans to submit another IND for the same indications for a CD22-targeted CAR T later in 2022.
MSC Therapy Shows Promise in Diabetic Kidney Disease
No adverse events related to the infusions of stromal cells were reported.
Advanced Colorectal Cancer CAR T Cleared for Trial
The phase 1 portion of the trial assessing CNA3103 will start enrollment in Australia in the first half of 2023; the phase 2 portion will expand to the US.
Cilta-cel CARTITUDE-4 Trial to be Unblinded After Meeting Primary End Point
Cilta-cel demonstrated a significant improvement in progression-free survival over standards of care.
