Type 1 Diabetes Cell Therapy Cleared to Begin Clinical Trial
AlloStem will be evaluated in the CELZ-201 clinical trial for patients with newly diagnosed type 1 diabetes.
Around the Helix: Cell and Gene Therapy Company Updates – November 2, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
CAR Monocyte Therapy Fast Tracked for Peripheral T-Cell Lymphoma
MT-101 is designed for quick delivery to patients with a vein-to-vein time of 8 days.
TIL Therapy Cleared to Begin Clinical Trial for Patients with Advanced Solid Tumors
TIDAL-01 will be evaluated in patients with advanced or metastatic breast carcinoma, colorectal adenocarcinoma, or uveal melanoma.
EBV-targeted T-cell Immunotherapy Associated With Improvements in Progressive Multiple Sclerosis
MRI revealed that participants who achieved sustained or confirmed disability improvement showed significantly less ventricular enlargement at 12 months.
Gaucher Disease Gene Therapy Granted FDA Rare Pediatric Disease Designation
AVR-RD-02 also received ILAP designation in the UK earlier this month.
Hepatocellular Carcinoma RNA-Editing Therapy Gets FDA IND Clearance
RZ-001 previously received IND clearance from the South Korean Ministry of Food and Drug Safety in June.
GSK, Lyell Immunopharma Terminate Agreement for Targeted Cell Therapies
The decision comes after strategic uncertainty regarding a review of preliminary clinical data from GSK’s lete-cel product candidate trial.
Around the Helix: Cell and Gene Therapy Company Updates – October 26, 2022
New Process Could Drastically Reduce CAR-T Manufacturing Time
Cells produced by this method were also shown to be more potent in animal models than cells made with typical processes.
TCR T-cell Therapy Trial for MAGE-A1-Positive Solid Tumors Doses First Patient
In preclinical research, T-cells bearing TK-8001's TCR showed better in vivo antitumor activity than T-cells incorporating human donor-derived TCRs.
Engineered TIL Therapy Trial Doses First Patient
ITIL-306 utilizes a CoStAR molecule intended to improve cytokine release, cytolytic activity, and proliferation of TILs.
Around the Helix: Cell and Gene Therapy Company Updates – October 19, 2022
Stem Cell Transplant Linked to Decreases in Multiple Sclerosis Biomarkers
The proportion of patients with concentrations of NfL and MBP above the upper limit of normal decreased from 67% and 63%, respectively, to 12% at 5 years.
Otoferlin-Related Hearing Loss Gene Therapy Gets IND Clearance
In a rodent model, durable auditory brainstem responses to sound were observed after DB-OTO administration.
Retinitis Pigmentosa Gene Therapy Trial Finishes Dosing Second Cohort
OCU400 utilizes Ocugen’s modifier gene therapy platform, which may allow the product to treat multiple retinal diseases.
Gene Therapy Continues to Show Promise in Canavan Disease
Participant 3 showed an 89% decrease in NAA in the CSF at 3 months post-treatment.
Anti-HIV CAR-T Therapy Trial Doses First Patient
Preclinical research showed the therapy was able to reduce cellular HIV infection by up to 99% in an in vitro model and more than 97% in a mouse model.
Around the Helix: Cell and Gene Therapy Company Updates – October 12, 2022
Retinitis Pigmentosa Optogenetic Therapy Gets FDA Fast Track Designation
The therapy was previously granted orphan drug designation for both RP and Stargardt disease.
Multiple Sclerosis Cell Therapy Demonstrates Promising Safety and Efficacy in Phase 2 Trial
Nearly 20% of treated patients achieved at least 25% improvement in timed 25-foot walk speed and/or 9-Hole Peg test at 28 weeks.
Allogeneic CAR-T Begins Phase 2 Clinical Trial
ALLO-501A received RMAT designation from the FDA in June 2022.
Fabry Disease Gene Therapy Clinical Trial Moves Onto Dosing Second Cohort
The FDA recently cleared Freeline Therapeutics to begin treating patients in the United States.
Gene Therapy Shows Promise in Leber Congenital Amaurosis
Significant improvements were reported in full-field stimulus testing.
Around the Helix: Cell and Gene Therapy Company Updates – October 5, 2022
Gene Therapy Trial Makes Headway in Canavan Disease
Data from the first 3 of the 8 patients dosed indicates that the patients improved on the GMFM and the MSEL.
Allogeneic Anti-CD7 CAR-T Demonstrates Promising Results in Hematological Malignancies
Among the 11 patients evaluated for efficacy, 9 patients (81.8%) had objective responses at 28 days.
BioMarin Resubmits BLA for Hemophilia A Gene Therapy Val-Rox
A prior resubmission in June 2022 was delayed with requests for additional durability and safety data.
GPRC5D-Targeted CAR-T Shows Promise in Heavily Pre-treated Multiple Myeloma
Of the 17 patients treated, 12 patients achieved a response, including 6 who achieved a complete response.
Around the Helix: Cell and Gene Therapy Company Updates – September 28, 2022
