REGENXBIO Set to Evaluate Duchenne Muscular Dystrophy Gene Therapy RGX-202 in Patients Aged 1 to 3 Years Old
The company has begun enrolling participants in a new cohort for younger patients in the phase 1/2 AFFINITY DUCHENNE clinical trial.
Intellia Successfully Redoses CRISPR Gene Editing Therapy
Three participants with ATTR amyloidosis have received follow-on doses of NTLA-2001 with no serious complications for up to 3 years of follow-up.
Around the Helix: Cell and Gene Therapy Company Updates – June 26, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Deborah Phippard, PhD, on the Challenges of Evaluating Gene Therapy for Parkinson Disease
The chief scientific officer of Precision for Medicine discussed unique difficulties of designing clinical trials for PD gene therapy candidates.
Vertex’s Diabetes Cell Therapy on Track to Meet Endpoint of Eliminating Severe Hypoglycemic Events
All 3 participants who have follow-up of at least 1 year have met the endpoint so far.
Anixa Biosciences Wraps Up Dosing of Second Cohort in Ovarian Cancer CER-T Trial
The company has dosed the sixth patient in the study, and expects to move onto the third cohort pending continued safety at 1 month posttreatment.
Kyverna’s CAR-T KYV-101 Shows Some Durable Responses in Autoimmune Disease, With One Relapse Reported
One patient is considered disease-free at 1 year posttreatment, but another patient’s disease relapsed at 5 months.
Omid Hamid, MD, on Assessing TIL Combination Therapies, Expanding Past Melanoma
The professor from Cedars Sinai discussed further research being conducted in the field after lifileucel’s approval.
Aurion’s Cell Therapy Wins RMAT, Breakthrough Therapy Designation for Corneal Edema
The CLARA trial has also completed enrollment of participants with corneal edema secondary to corneal endothelial disease.
Nathan Yozwiak, PhD, on Collaboration for Cell and Gene Therapy Development
The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed work the center is engaged in with cell and gene therapy.
enGene Tackles Nonmuscle-Invasive Bladder Cancer With Expansive Phase 1/2 LEGEND Trial for Gene Therapy EG-70
With the company having recently announced that it intends to expand the clinical trial with a new cohort, CGTLive® has decided to take a closer look at this ongoing study.
Huntington Disease Gene Therapy Nets RMAT Designation
AMT-130 has shown some evidence of dose-dependent clinical benefits in treated study participants.
Gene Therapy Improves Body Composition in Mice Models of Obesity Alone and After Semaglutide Withdrawal
Fractyl Health is conducting IND-enabling studies for Rejuva.
Continuing Innovation on Modality-Specific Technology for Cardiovascular-Targeted Genomic Medicines
Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, discussed the company’s research on capsids, promoters, and manufacturing improvements.
Genethon’s Duchenne Muscular Dystrophy Gene Therapy GNT0004 Produces Expression of Microdystrophin in Phase 1/2/3 Trial
For the 3 patients who were treated at the higher dose, immunohistochemistry showed that a mean of 54% of muscle fibers were expressing microdystrophin.
Salvador Rico, MD, PhD, on Developing Gene Regulation Therapy for Dravet Syndrome
The chief medical officer of Encoded Therapeutics overviews the company’s timeline and strategy for its gene regulation program for Dravet syndrome.
Neurona’s Focal Epilepsy Cell Therapy NRTX-1001 Garners FDA RMAT Designation
NRTX-1001 consists of human interneurons that provide long-term secretion of gamma-aminobutyric acid, an inhibitory neurotransmitter, which is expected to repair neural networks.
CGTLive®’s Weekly Rewind – June 21, 2024
Review top news and interview highlights from the week ending June 21, 2024.
Ultragenyx Intends to Seek Accelerated Approval for MPSIII Gene Therapy BLA
The company anticipates that it will be able to submit a BLA in late 2024 or early 2025.
Kyverna’s CAR-T Cleared for Stiff-Person Syndrome Trial
KYV-101 racks up another potential indication in addition to its primary autoimmune investigations.
FDA Approves Sarepta’s DMD Gene Therapy Elevidys for Expanded Indication
The gene therapy is now indicated for ambulatory patients aged 4 years and older, and has been granted accelerated approval for nonambulatory patients.
Cabaletta Bio’s CAR-T CABA-201 Shows Initial Signs of Efficacy in Myositis and Lupus Trials
The data, from the first patients dosed in each trial, also continues to show safety.
Leigh Ramos-Platt, MD, on Sharing Experience With Gene Therapy Administration
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC discussed her learning experience administering gene therapies for the first time.
Gene-Edited Exa-Cel Continues to Show Long-Term Benefit in Sickle Cell, Thalassemia
Data from up to 5 and 6 years of follow-up were presented at the 2024 EHA Congress.
Choosing the Right Treatment Path for Sickle Cell Disease
In observance of World Sickle Cell Day, CGTLive brought together a variety of expert insights on navigating the rapidly expanding landscape of care for this inherited blood disorder.
Around the Helix: Cell and Gene Therapy Company Updates – June 19, 2024
Daniel Hart, PhD, on CRISPR-Mediated In Vivo Epigenomic Activation
The Senior Director and Head of Technology Development at Epic Bio discussed potential applications of the new technology.
Kyverna’s CAR-T KYV-101 Improves Walking Speed in Patient With Stiff-Person Syndrome
The case study was recently published in the Proceedings of the National Academy of Sciences.
Rett Syndrome Gene Therapy Shows Clinical Benefit, Challenging Adverse Events in Pediatric Patients
An investigator noted challenging side effects related to immunosuppressant treatment.
Mustang Bio’s CAR-T MB-106 Achieves Responses in 9 of 10 Patients With Waldenstrom Macroglobulinemia
The responses seen in the 9 patients included 3 complete responses (CRs), 2 very good partial responses, and 4 partial responses.
