Real-World Luxturna Data Shows Good Efficacy, New Adverse Reaction in Retinal Dystrophy
Investigators presented up to 2 years of post-marketing data on the first-ever gene therapy.
FDA Fast Tracks Graphite Bio's Sickle Cell Disease Gene-Edited Cell Therapy
Enrollment is ongoing in the phase 1 CEDAR trial, which plans to dose the first patient later in 2022.
Taysha Granted European Orphan Drug Designation for Giant Axonal Neuropathy Gene Therapy
The AAV9 gene therapy is currently being evaluated in a clinical trial conducted by NINDS.
Treatment With OpRegen Shows Improvement in Visual Acuity, Cell Persistence in Dry AMD
New data from cohort 4 showed outer retinal restoration following treatment with OpRegen.
Neutralizing Antibodies to AAV Vector for AMD Gene Therapy May Boost Therapeutic Effect
The post-hoc analysis of the OPTIC trial explored the effects of neutralizing antibodies on patients treated with ADVM-002.
CAR T-Cell Therapy Is Safe, Effective in Minorities, Study Finds
The real-world analysis addresses a clear gap in data from previous and ongoing cell therapy clinical trials.
Gene Therapy for Frontotemporal Dementia Granted Orphan Drug Status
The one-time therapy is meant to address familial early-onset dementia linked to the GRN gene.
ZUMA-7 Axi-Cel Superiority Calls for Treatment Paradigm Shift in R/R LBLC
The analysis of data from ZUMA-7 demonstrated significant efficacy for axi-cel over second-line standard treatment.
Obe-Cel Granted RMAT Designation in R/R B-Acute Lymphocytic Leukemia
The gene-edited CAR T-cell therapy is currently being investigated in a phase 2 clinical trial.
CD20-Targeted CAR T-Cell Therapy Shows Safety, Good Response Rates in B-Cell Non-Hodgkin Lymphoma
A phase 1/2 multicenter study is underway following MB-106’s IND approval.
Off the Shelf NK Cell Therapy Feasible in R/R Acute Myeloid Leukemia, Myelodysplastic Syndrome
Preliminary findings from the phase 1 clinical trial demonstrate good cell persistence and expansion in vivo.
First Patient Dosed With CRISPR Therapy Targeting E. Coli Colonization
The oral therapy is intended for high-risk hematological cancer patients.
T Cell Therapy for Post-Transplant Virus Prevention Granted RMAT Designation
This is the third regenerative medicine advanced therapy designation granted to AlloVir’s posoleucel therapy.
Combination CAR T-Cell Therapy, RNA Vaccine Shows Efficacy in Solid Tumors
The novel regimen from BioNTech demonstrated encouraging results in patients with testicular or ovarian cancer.
Bluebird Bio Slashes Workforce as It Awaits Key FDA Gene Therapy Decisions
The biotechnology company previously pulled out of Europe after encountering payor challenges for their gene therapies.
CGTLive’s Weekly Rewind – March 25, 2022
Review top news and interview highlights from the week ending March 25, 2022.
Proposed FDA Gene Editing Guidance Emphasizes Long-Term Safety Evaluations
The draft guidance from the Center for Biologics Evaluation and Research outlines safety considerations throughout the clinical development program timeline.
Celyad Pauses CAR T Cell Therapy Trial of CYAD-101 Following Patient Deaths
The phase 1b KEYNOTE-B79 trial is investigating the allogeneic cell therapy in participants with metastatic colorectal cancer.
Gene Therapy for Friedreich Ataxia Cardiomyopathy Set to Move Forward
The FDA has approved LEXEO Therapeutics’ investigational new drug application.
Fabry Disease Gene Therapy Shows Encouraging Effect on Cardiac End Points
The dual-mechanism of the therapy helps address both systemic and organ-specific deficits.
Severe Neurodegenerative, Epileptic Disorders the Target of Taysha Gene Therapies
With numerous targets in CNS diseases and a unique partnership with gene therapy experts at UT Southwestern, Taysha is hoping to fill significant unmet needs.
Poseida’s CAR-T Cell Therapy for Prostate Cancer Shows Promising Durable Response in Phase 1
The early-phase data readout follows news that the FDA recently approved its IND for its CAR-T candidate in multiple myeloma.
Race to Treat GM1 Gangliosidosis With Gene Therapy Continues
The first US-based patient has been dosed in Lysogene’s clinical trial of LYS-GM101, following 2 other competing trials.
2021 CAR-TCR Summit Elevates Industry Expertise in Cell Therapies
The 4-day virtual conference will feature plenary presentations, workshops, and bootcamps led by cell therapy industry leaders with expertise in discovery, development, manufacturing, and more.
CRISPR Gene Editing for Transthyretin Amyloidosis Safe, Effective
Interim data from a phase 1 study from Intellia Therapeutics and Regeneron Pharmaceuticals is the first to support in vivo CRISPR genome editing in humans.
StrataGraft Cellularized Scaffold Approved for Treatment of Deep Partial Thickness Burns
The allogeneic, cellularized scaffold product helps support autologous skin tissue regeneration in patients with thermal burns requiring surgical intervention.
Targeting Dysfunctional RNA for a More Precise Approach to Gene Therapy
Locanabio CEO James Burns, PhD, discusses the firm's targeted approach to treating genetic disorders of the central nervous system and the eye.
Cynata Therapeutics Takes Fresh Approach to Stem Cell Production
Ross Macdonald, PhD, managing director and CEO of Cynata Therapeutics, discusses the biotech company's approach to overcoming reproducibility challenges with mesenchymal stem cells.
Novel Film Matrix May Solve AAV-Based Therapy Production, Storage Challenges
Maria A. Croyle, PhD, discusses the development of a novel film matrix that can safely and effectively store and transport AAV-based gene therapies.
Addressing Challenges in Gene Therapy Vector Manufacturing and Storage
Maria A. Croyle, PhD, discusses the manufacturing challenges facing the gene therapy space and her inspiration for developing a novel preservation method for viral vector-based therapies.