American Gene Technology’s Cell Therapy Continues to Show Safety in HIV
An independent Data Safety and Monitoring Board has recommended that the phase 1 RePAIR trial continue at an accelerated pace.
IND Accepted for Sickle Cell Disease Cell Therapy
Beam Therapeutics will assess the safety and efficacy of BEAM-101 for SCD in the phase 1/2 BEACON-101 study.
IND Accepted for HER2+ Gastric and Gastroesophageal Junction Cancer Cell Therapy
CYNK-101 is set to be evaluated in combination with standard chemotherapy, trastuzumab and pembrolizumab in a phase 1/2a study.
CRISPR/Cas9-Edited CAR and NK Cell Therapies Show Preclinical Efficacy
CRISPR Therapeutics presented positive data on 2 preclinical programs at the 2021 SITC meeting.
Hemophilia A Gene Therapy Placed on Clinical Hold
Pfizer and Sangamo voluntarily paused the trial after participants experienced FVIII activity levels of over 150%.
FDA Accepts Beti-Cel BLA for β-thalassemia
The investigational gene therapy, which will receive priority review, has a PDUFA date of May 20, 2022.
Hemophilia A Gene Therapy Shows Sustained Factor VIII Expression
A 1-stage factor VIII assay revealed no apparent decrease in factor VIII activity over time.
Gene-Edited, Autologous HSP Cells Correct Metabolic Activity in Hurler Syndrome
The gene-edited approach hopes to overcome with the shortcomings of autologous hematopoietic stem-cell transplantation.
Danon Disease Gene Therapy Yields Sustained Clinical Benefit
Following updated safety data, Rocket Pharmaceuticals has decided to discontinue the high-dose cohort.
Charcot-Marie-Tooth Gene Therapy Yields Improvements in Disability and Muscle Loss
The phase 1 trial is the first in-human trial to assess a gene therapy for CMT.
OpRegen Shows Durable Efficacy in Dry AMD Geographic Atrophy
The Doheny Image Reading and Research Lab is leading a sub study to analyze OCT images with their proprietary 3D-OCTOR software.
CYNK-101 Demonstrates Anti-Tumor Activity in HNSCC Cells
The NK cell therapy is also being evaluated in a phase 1 study for acute myeloid leukemia.
Dendritic Cell Vaccine Boosts Survival in Glioblastoma
Overall survival was 72% at 12 months but dropped to 54% at 14.6 months.
FDA Manufacturing Data Request Delays Omidubicel BLA Submission
The request aims to ensure the comparability of omidubicel manufactured at different sites.
CAR Macrophages Well-Tolerated, May Reprogram Tumor Microenvironments
The first in-human study of CAR macrophages has dosed 2 participants so far.
Tmod CAR T-Cell Therapy Offers Precise Targeting of Solid Tumors
The BASECAMP-1 study is identifying patients with human leukocyte antigen (HLA) loss of heterozygosity (LOH) for future use of these therapies.
CAR T-Cell Therapy Plus Amplifying Vaccine Shows Initial Safety, Efficacy in Solid Tumors
Three patients dosed had tumors shrinkages of 18%, 21%, and 27%.
TCR T-Cell Therapy Shows Efficacy Across Solid Tumor Types
Data from the IMA203 trial were presented at the SITC 2021 annual meeting.
Advancing Natural Killer Cell Therapies for Hematologic Malignancies
Sonny Hsiao, PhD, chief executive officer, president and cofounder, Acepodia, discussed the company’s future research and plans.
Gavo-Cel to Be Evaluated With Nivolumab, Ipilimumab for Solid Tumors
TCR² Therapeutics is collaborating with Bristol Myers Squibb to evaluate combination therapies in a phase 2 trial.
Gene Therapy Well-Tolerated in Fabry Disease
Primary efficacy data released by Sangamo Therapeutics showed that above normal α-Gal A activity was maintained for up to a year after treatment.
Gene Therapies Show Efficacy in Fabry Disease, Choroideremia
4D Molecular Therapeutics announced positive data from trials assessing 2 of their gene therapies.
Improving Cell Therapy Manufacturing Through Strategic Partnerships
Paul Lammers, MD, MSc, president and chief executive officer, Triumvira Immunologics, discussed the challenges of manufacturing cell therapies.
Cell Therapy Cancer Vaccine Shows Promise for Progression-Free Survival in Metastatic Pancreatic Cancer
The vaccination therapy improved progression-free survival by 3.1 months over standard care in 1 patient treated.
Rare Disease Consortium to Focus on Optimizing AAV Gene Therapies
Partners in the Bespoke Gene Therapy Consortium include the NIH, FDA, private companies such as Pfizer and Biogen, and non-profits.
CRISPR Therapy for ATTR Amyloidosis Granted Orphan Drug Designation
The phase 1 study of NTLA-2001 previously showed positive interim data, including serum TTR reductions, which were reported in June 2021.
MPS 2 Systemic Gene Therapy Evaluated in New Trial
The FDA cleared the IND application for HMI-203 in October 2021.
FDA Grant Supports Recessive Dystrophic Epidermolysis Bullosa Gene Therapy Study
Dabocemagene autoficel has previously received orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy designations.
Gene Editing Clinical Trial for PKU Announced
Homology is also developing a gene therapy, HMI-102, being evaluated in the phase 1/2 pheNIX trial.
Gene Therapy Promotes Wound Closure of Diabetic Foot Ulcers
VM202 was found to be particularly effective in treating neuroischemic ulcers.