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.
Gene Therapy for Congenital Hearing Loss Granted Orphan Drug, Rare Pediatric Disease Designations
Decibel Therapeutics previously presented favorable preclinical data at ASGCT 2021.
PKU Gene Therapy Trial on Clinical Hold Following Tumor Development in Mice
Six of 7 mice treated with a new, higher dose of BMN-307 showed tumors in liver necropsy at 52 weeks post-treatment.
New Gene Therapy Collaboration Targets Huntington Disease
NeuExcell and Spark Therapeutics each add to their gene therapy pipelines with the collaborative program.
Recruitment Resumed in Gene Therapy Trial for Ovarian Cancer
The FDA has cleared a batch of VB-111 manufactured in Modiin, Israel, following a technical review.
Utilizing CRISPR Technology to Develop Cell Therapies for Diabetes, Blood Diseases
Both Vertex and Arbor are adding to their list of gene and cell therapy partnerships with the announcement.
Gene Therapies for Neurological Diseases Will Be Focus of New Shape Therapeutics, Roche Collaboration
The 2 companies will focus their strategic partnership on the development of gene therapies for rare diseases, as well.
Gene Therapies for Hemophilia Have Positive Real-World Impact, Studies Show
Presentations at ISTH 2021 revealed attributes most important to patients when considering gene therapies and where further research is needed.
IND Cleared for New CAR T-Cell Therapy in Large B-Cell Lymphoma
KITE-363 is set to be investigated in a phase 1 clinical trial at the end of 2021.
Mustang Bio, Mayo Clinic Collaborate on New CAR T Platform
Mustang Bio plans to file an IND and start a phase 1 clinical trial as soon as a lead construct is identified.
JQ1, a BET Inhibitor, Increases CAR T Efficacy and Durability
Investigators sought to understand the mechanisms of transferred T cell proliferation and expression.
FDA Lifts Hold on Danon Disease Gene Therapy
Rocket Pharmaceuticals plans to quickly resume the phase 1 trial and commence dosing in their new, low-dose, pediatric cohort in Q3 2021.
Gene Therapy for Wilson Disease Receives FDA Fast Track Designation
A phase 1/2 study evaluating Vivet Therapeutic’s VTX-801 is set to initiate in August 2021.
Hemophilia Gene Therapy Registry Launched to Monitor Long-Term Outcomes
The World Federation of Hemophilia’s registry will include long-term safety and efficacy data in people with hemophilia treated with gene therapies.
FDA Places Clinical Hold on Bluebird Bio’s eli-cel for CALD
Bluebird Bio’s latest clinical hold follows another report of MDS in a treated patient.
Gene Therapy for Diabetic Macular Edema Halted Following Serious Adverse Reaction
Despite setbacks in DME, Adverum Biotechnologies said it will continue to develop ADVM-022 for wet AMD.
Fidanacogene Elaparvovec Well-Tolerated in Hemophilia B
No concerning effects to liver health were observed in the phase 1/2a study.
FDA Lifts Clinical Hold on Novartis SMA Gene Therapy
The company announced plans to initiate the STEER study of intrathecal OAV-101 for older patients with SMA type 2.
Hemophilia B Gene Therapy AMT-060 Shows Durable Effect
Phase 3 studies are continuing in the enhanced Padua FIX variant AMT-061 for hemophilia B.
Gene Therapies for Batten Disease, ALS Receive FDA Designations
The designations allow for expedited drug development and review of the investigational gene therapies for CLN2 Batten disease and SOD1 ALS.
MRI Data Supports Gene Therapy for Sanfilippo Syndrome Type A
Abeona Therapeutics is seeking a path towards BLA filing based on the promising clinical data.