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.
Eli-cel Gene Therapy for Early CALD to Be Withdrawn From EU, UK
bluebird bio is also planning to withdraw the marketing authorization for their β-thalassemia therapy, beti-cel, from the EU and UK.
In-Vivo Genome Editing Therapy Shows Efficacy in Children With MMA
The phase 1/2 SUNRISE trial is now enrolling patients as young as 6 months old with methylmalonic acidemia after positive review under a DSMB.
Gene Therapy for LAD-1 Demonstrates Efficacy
Rocket Pharmaceuticals announced positive data on RP-L201 in leukocyte adhesion deficiency-I, as well as RP-A501 for Danon disease.
Fabry and Gaucher Gene Therapies Well-Tolerated
AVROBIO shared interim safety data from ongoing phase 1 and 2 trials.
Diabetes Cell Therapy Improves Insulin Production in First Patient Dosed
The patient’s daily insulin requirement went from 34 units a day at baseline to 2.9 units a day 90 days after treatment.
Solid Tumors Targeted in Next-Generation CAR T Collaboration
The collaboration combines AdAlta’s i-bodies and Carina Biotech’s chemokine receptor platform.
Optogenetic Therapy Shows Efficacy in Autosomal Recessive Retinitis Pigmentosa
Results from a phase 1/2 study from Nanoscope Therapeutics were presented at the 2021 ASRS meeting.
Study Investigates New CAR T Therapy for T-Cell Lymphoma
The study follows Legend Biotech’s initial success with cilta-cel, whose PDUFA date is set for November 2021.
CAR T-Cell Therapy From CRISPR Therapeutics Efficacious in CD19+ B-Cell Malignancies
Enrollment in the CARBON trial has so far focused on aggressive disease such as diffuse B-cell lymphoma.
Ocular Gene Therapy RGX-314 Expands Efficacy to Diabetic Retinopathy
REGENXBIO previously announced positive data in patients with wet AMD who were treated with the same gene therapy.
Sarepta’s Duchenne Gene Therapy Shows Efficacy in Multiple Studies
Sarepta Therapeutics has also initiated the pivotal phase 3 EMBARK study in pediatric patients with DMD.
TCR-T Cell Therapy Cleared for Phase 1/2 Study in Acute Myeloid Leukemia
Intellia Therapeutics is also initiating a phase 1/2 study of a CRISPR therapy for hereditary angioedema in New Zealand.
Posoleucel for Virus-Associated Hemorrhagic Cystitis Receives Orphan Drug Designation
ALVR109, another of AlloVir’s T-cell therapies, also recently showed efficacy in treating COVID-19 in high-risk patients.
AlloCAR T Programs Halted Following Chromosomal Abnormality
Allogene continues to investigate and characterize the chromosomal abnormality and its relationship to the gene editing.
Natural Killer Cell Therapy Well Tolerated in Solid Tumors
Thorsten Graef, MD, PhD, chief medical officer, Acepodia, discussed data on ACE1702 presented at ESMO 2021.
Krabbe Disease Gene Therapy Receives Second Orphan Drug Designation
Forge Biologics and Solid Biosciences have also partnered to help develop a gene therapy for Duchenne muscular dystrophy.
Gavo-Cel Efficacious in Refractory Mesothelin-Expressing Solid Tumors
Investigators have identified the maximum tolerated dose and continue to refine the recommended phase 2 dose in the phase 1 part of the study.
SGT-001 Improves Pulmonary Function in Duchenne Muscular Dystrophy
Improvements in peak expiratory flow, forced expiratory volume, and forced vital capacity were observed in all evaluable treated patients in the IGNITE-DMD trial.