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.
OpRegen's Continued Efficacy in Dry AMD Geographic Atrophy
Brian Culley, chief executive officer, Lineage Cell Therapeutics, discussed updated data from the phase 1/2 study of OpRegen.
beti-cel BLA Submitted for β-Thalassemia
The news comes after another of bluebird bio’s programs, eli-cel, was placed on clinical hold.
New Collaboration to Develop ROR1 Targeted Cell Therapies
Oncternal and Celularity hope to produce ROR1-targeted NK, CAR-NK, and CAR T-cell therapies.
HIV Gene Therapy Gets Go-Ahead for Early-Phase Trial
A phase 1 trial of a cell therapy for HIV is also currently underway.
OpRegen Continues to Show Efficacy in Dry AMD Geographic Atrophy
Lineage Cell Therapeutics announced promising data from interim results of the phase 1/2a clinical trial assessing the cell therapy.
Cell Therapy for Critical Limb Ischemia Shows Positive Interim Data
An interim analysis has met prespecified safety and efficacy checkpoints.
Astellas Pauses Gene Therapy Trial for X-Linked Myotubular Myopathy
Following a voluntary pause, a patient experiencing a liver AE has died and the trial is now on clinical hold.
T Cell Therapy Trial for High-Risk Patients With COVID-19 Now Enrolling
TVGN-489 is an investigational allogeneic cytotoxic CD8+ T lymphocyte therapy that may help prevent breakthrough COVID infection.
Bone Therapeutics Shifts Focus to Cell Therapies Following Phase 3 Fail in Knee Osteoarthritis
The company is currently evaluating ALLOB, its investigational cell therapy product for bone regeneration.
TAC T-Cell Therapy vs CAR T-Cell Therapy
Paul Lammers, MD, MSc, president and chief executive officer, Triumvira Immunologics, discussed the company’s T-cell antigen coupler technology.