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Eli-cel Gene Therapy for Early CALD to Be Withdrawn From EU, UK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.
Suprachoroidal Delivery of RGX-314 Well-Tolerated in Wet AMD
Patients treated with RGX-314 also had stable BCVA and central retinal thickness.
Angelman Syndrome Gene Therapy Trial Resumes Following Clinical Hold
A clinical hold was previously placed after patients treated with the highest dose developed lower extremity weakness.
Gene Therapy for AADC Deficiency Yields Durable Developmental Improvements
Improvements persisted through up to 10 years of follow-up.
High Dose Achromatopsia Gene Therapy Yields Severe Inflammation
The dose was previously evaluated in adult patients with no comparable inflammation.
Pfizer Restricts Enrollment in Duchenne Gene Therapy Trial Following Adverse Events
The decision comes after 3 participants experienced serious adverse events during the phase 3 trial.
CRISPR Therapy Shows Some Benefit in Leber Congenital Amaurosis
A competing RNA-based therapy for LCA10 is currently being evaluated in a phase 2/3 trial.
Cardiac-Derived Cell Therapy Shows Efficacy in Duchenne Muscular Dystrophy
CAP-1002 met primary and secondary end points in the HOPE-2 trial.
Gene Therapies for Liver Diseases the Focus of New Collaboration
Precision BioSciences is collaborating with iECURE, a new company focused on mutation-agnostic, liver disease gene therapies.
SGT-001 Shows Efficacy in DMD in 1.5-Year Data
Patient-reported outcomes, 6-minute walk test, and NSAA scores all showed improvements over natural history data.
Developing Cell Therapies for Solid Tumors and Hematologic Malignancies
Sonny Hsiao, PhD, chief executive officer, president and cofounder, Acepodia, discussed the company’s cell therapy technologies.
Addressing Adverse Events in Gene Therapy
The recent FDA Advisory Committee meeting follows a turbulent year for gene therapy studies.