Noah Stansfield

Patients in the UK with r/r multiple myeloma may still be able to access the CAR-T through ongoing clinical trials, which are not impacted by the decision.

Intergalactic Therapeutics’ IG-002 utilizes a proprietary non-viral delivery method.

Jeffrey S. Chamberlain, PhD, Professor and McCaw Chair, Muscular Dystrophy, and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, discussed the potential use of microdystrophin as an end point for clinical evaluations of gene therapies for DMD.

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

In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest expert insights in gene therapies for hemophilia, which is among the most common of these disorders.

GenSight Biologics reported that 73% of patients treated bilaterally with Lumevoq improved at least +15 ETDRS letters relative to their worst recorded BCVA.

Matthew B. Harms, MD, associate professor of neurology at Columbia University, and medical consultant and care center director at the MDA, discussed the session he will be chairing at the conference.

The planned clinical trial of EB103 has received clearance to include patients with HIV-associated lymphoma and primary and secondary central nervous system lymphoma.

In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news in gene therapies for hemophilia—one of the most common of these disorders.

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

Samiah Al-Zaidy, MD, vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the latest results from the Imagine-01 clinical trial evaluating the investigational treatment PBGM01.

Three patients treated with Genethon’s GNT-003 were able to cease treatment with standard of care phototherapy for at least 1 year.

The company has halted phase 3 plans for BIVV003 in favor of therapies for Fabry and immune rejection in kidney transplantation.

AskBio’s AB-1003 was previously granted fast track designation by the FDA.

Four of 5 patients showed improvements in ejection fraction following bone marrow transplant.

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

4D Molecular Therapeutics’ 4D-310 effected clinically meaningful improvements in left ventricular function, exercise capacity, and quality of life.

In observance of Rare Disease Day, held annually on February 28, catch up on some of the latest data updates from clinical trials for rare diseases.

Patients who received the high dose of PBGM01 showed an increase in β-Gal activity in the CSF of 4.7 to 5.2 times baseline.

Following treatment with AT845 gene therapy, 3 of 4 patients withdrew from their previous enzyme replacement therapy.

The patient has not re-started any of his previous Gaucher-specific therapy since receiving AVR-RD-02.

Two patients treated with AVR-RD-02 showed clinically meaningful reductions in liver size.

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

By 3 months, 12 of 17 patients treated with a full cycle of EG-70 achieved a complete response.

It was found that ctDNA levels at 28 days post-treatment were significantly higher for patients who ultimately progressed by 90 days post-treatment.

Median PFS was 13.3 months in the ide-cel arm, compared to 4.4 months for the SOC arm.

Patients with large B-cell lymphoma had an ORR of 68% and a CR rate of 53%.

Among the 14 patients with relapsed/refractory mantle cell lymphoma who were treated with LV20.19 CAR in the trial, the overall response at 28 days post-treatment was 100%.

Among 43 patients who had EBV+ PTLD following allogeneic hematopoietic cell transplant or solid organ transplant, the overall response rate was 51.2%.

Neither patient treated with RP-L301 required RBC transfusions at any point post-engraftment.