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CGTLive®’s Weekly Rewind – September 5, 2025

Robert Alexander Wesselhoeft, PhD, on the Therapeutic Potential of Circular RNA

Nanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy MCO-010

Preclinical Evidence for PH1 Gene Therapy

Around the Helix: Cell and Gene Therapy Company Updates – September 3, 2025

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The chief scientific officer and senior director of preclinical pharmacology Arbor Biotechnologies discussed ABO-101, the company’s gene therapy for primary hyperoxaluria type 1.

The rAAVrh8 gene therapy delivers HEXA and HEXB genes and has posted positive phase 1/2 results in infantile GM2 gangliosidosis, with enhancements in enzymatic activity and neurological outcomes.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

Review top news and interview highlights from the week ending August 29, 2025.

Annaiz Grimm, BS, a research scientist at Seattle Children's Research Institute, discussed the potential use of EngTregs to treat autoimmune disease.

The research scientist at Seattle Children's Research Institute discussed a potential alternative to standard of care immunosuppressive therapy.

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

Prerna Mewawalla, MD, the medical director of Apheresis and a hematologist-oncologist in the Division of Hematology and Cellular Therapy at Allegheny Health Network, spoke on unmet needs in patients who receive early-line CAR-T for R/R MM.

Notably, the FDA granted Papzimeos a full approval, without a requirement for a confirmatory trial.

Ultragenyx anticipates that the full BLA will have been submitted in the fourth quarter of this year.

The medical director of Apheresis and a hematologist-oncologist in the Division of Hematology and Cellular Therapy at Allegheny Health Network spoke about unmet needs in patients who receive early-line CAR T for R/R MM.

The new PDUFA date is set for February 8, 2026.

Surbhi Sidana, MD, the assistant professor of medicine, bone marrow transplantation, and cellular therapy at Stanford discussed the benefit of second-line cilta-cel in PFS and OS.

Review top news and interview highlights from the week ending August 22, 2025.

The hold was originally placed on the trial by the FDA on May 23, 2025, following the death of a patient treated in the study.