Noah Stansfield

The data, from the CARTITUDE-4 clinical trial, compared outcomes to patients treated with standard of care options.

The chief scientific officer and head of research, development, and Medical at Ocugen, also discussed future plans for the gene therapy, which is now in a phase 3 study.

The company pointed out that today, September 30, is LGMD Awareness Day.

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed several still-investigational drugs and therapies for DMD.

Enrollment difficulties were cited as the reason for discontinuing recruitment activities for KarMMa-9.

The approvals are indicated for patients 12 years and older.

Notably, today, September 26, is annually observed as Mesothelioma Awareness Day by the patient and clinician communities.

The chief scientific officer and head of research, development, and Medical at Ocugen, discussed OCU400, the company’s gene-agnostic gene therapy for IRDs.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed the complicated choice doctors, patients, and families now face with a wide range of treatment options available.

In lymph node biopsies from patients treated in the study, a mean exposure of 236,701 CAR T-cells per million across all dose levels was observed.

There were no SUSARS and no endophthalmitis observed among 29 patients treated in the study.

Kiromic anticipates that Deltacel-01's expansion portion, which is expected to include around 9 new patients, will begin screening patients before the end of September.

John Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed how Sarepta’s Elevidys has affected the landscape of care for DMD.

AAVB-081 is intended to address retinitis pigmentosa that results from Usher syndrome type 1B.

In honor of Usher Syndrome Awareness Day, CGTLive® interviewed Zheng-Yi Chen, DPhil, associate professor, Otolaryngology–Head and Neck Surgery, Harvard Medical School, about the current state of research in this rare disease.

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, shared his outlook on the trajectory of research in the field.

The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed several still-investigational drugs and therapies for DMD.

The product is currently being evaluated in a phase 1/1b clinical trial.

The single-arm trial enrolled 12 male patients in total across the United States and European Union.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia discussed the complicated choice doctors, patients, and families now face with a wide range of treatment options available.

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, highlighted studies presented at MDA's 2024 conference.

VNX-101 is intended to transduce cells of the liver, causing them to express a transgene coding for a bispecific T-cell engager.

The company made the decision to axe the program in light of newly reported topline results from the clinical study.

Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed his research career with muscular dystrophies.

The company also discontinued its other clinical programs as well, which included small molecule and monoclonal antibody modalities.

Kevin Campbell, PhD, a Howard Hughes Investigator at the University of Iowa, discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.

The chief medical advisor of Muscular Dystrophy Association (MDA) gave his view on treatment decision-making in the world of new options for DMD.

The company also reported that it is discontinuing the phase 3 REGEN-016 (PROACT 2) clinical trial evaluating rilparencel in Spain.