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The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed the PDC group and collaborating with advocacy groups.

No treatment-emergent adverse events experienced were deemed related to AstroRx, although 9 serious TEAEs did occur.

Atsena Therapeutics’ AAV gene therapy is being evaluated in 15 patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D across 5 cohorts of various doses. Additional data are expected to be presented later this year.

The neurology resident at Penn Medicine spoke about the preparations taken in the lead up to tofersen’s PDUFA date, which is set for April 25, 2023.

The clinical trial of HG004 for RPE65-LCA2 dosed its first patient in February 2023.

The rolling BLAs also included requests for priority review of the CRISPR/Cas9 gene-edited therapy, and is supported by data from the phase 3 CLIMB-111 and CLIMB-121 studies, as well as the long-term follow-up study CLIMB-131.

The co-CEO of BrainStorm Cell Therapeutics spoke about the current standards of care in ALS and the potential of NurOwn to address unmet needs.

The company also recently announced updates in the MATRICS-1 clinical trial for hemorrhagic trauma.

Precigen’s investigational UltraCAR-T therapy is being assessed in a dose-escalation and -expansion study that will include patients with solid tumors, chronic lymphocytic leukemia, mantle cell lymphoma, acute lymphoblastic leukemia, and diffuse large B-cell lymphoma.

Review top news and interview highlights from the week ending March 31, 2023.

CB-011 and CB-010 are being evaluated for the treatment of r/r multiple myeloma and r/r B-cell non-Hodgkin lymphoma, respectively.

The associate professor of neurology at Columbia University spoke about the importance of identifying patients with ALS-associated gene mutations as early as possible.

Most patients with RP treated with MCO-010 had clinically meaningful improvements in vision.

The Study Safety Committee has recommended that the trial proceed to enroll patients in the highest-dose cohort.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about preclinical research with an iPS cell-derived product.

The gene-edited cell therapy had previously been under a partial clinical hold restricting pediatric enrollment to the clinical trial.

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

Intellia Therapeutics received clearance to enroll patients from the US in the global phase 2 portion of its clinical trial earlier this month.

The associate professor of neurology at Columbia University spoke about the current and future treatment landscape in ALS.

OCU400 is also being evaluated in other retinal degeneration indications.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about the session she chaired at the 2023 MDA Clinical and Scientific Conference.

In November 2022, BrainStorm received a refusal to file letter from the FDA for the biologics license application of NurOwn.

The FDA will determine the acceptability of the BLA within the next 60 days.

Krystal Biotech's investigational topical gene therapy has shown promise in DEB. It is designed to deliver COL7A1 and restore C7 protein in patients with the rare disorder.

The co-CEO of BrainStorm Cell Therapeutics spoke on the supporting data for the investigational ALS cell therapy that will be discussed at the meeting, and expectations for it.

The company's persistence in bringing NurOwn to the market amidst negative regulatory feedback has garnered an AdComm meeting.

Review top news and interview highlights from the week ending March 24, 2023.

The Delphi panel considerations were informed by safety data from 3 clinical trials of the therapy, and included vomiting, myocarditis, acute liver injury, and immune-mediated myositis.

Direct Biologics’ ExoFlo is also being evaluated for the treatment of COVID-19 acute respiratory distress syndrome.

The patient was treated at the new fifth dose level and had concomitant metapneumovirus infection.