Following Up on Research from the 25th Annual Meeting

If you’ve attended the ASGCT Annual Meeting before, you know it’s the best place to hear about updates in the gene and cell therapy field. We looked back at the abstracts we received from the 2022 meeting, and we want to share a few updates about where the research currently stands. Read on to learn more from previous presenters and make sure you’re registered to attend the 26th Annual Meeting in May—we’ll have some exciting research to share.

In Longest XSCID Follow Up, GT Is Well Tolerated

During the 25th Annual Meeting’s Clinical Trials Spotlight Symposium, researchers from St. Jude Children’s Research Hospital reported what they believed to be the longest follow-up data (a median of 2.4 years) on the largest cohort of infants with X-linked severe combined immunodeficiency (XSCID) treated with a lentiviral vector gene therapy. The team reported that the gene therapy with low-dose busulfan conditional was well tolerated and resulted in the development of a durable and functional immune system with no evidence of malignant transformation.

In December, ASGCT Member Stephen Gottschalk, MD, a co-author on the St. Jude-led 2019 study that produced those results, said the team hads so far treated 24 children who have been tracked for five years and their outcomes remain excellent.

That same month, ASGCT Member Morton Cowan, MD, also part of the 2019 research team, co-authored a study published in the New England Journal of Medicine reporting initial success in using a gene therapy to treat 10 children with another form of the disease, Artemis-deficient (ART)-SCID.

Phase 1 Trial for Heart Failure GT Ongoing

Three patients treated with NAN-101, a gene therapy for congestive heart failure (CHF), saw consistent improvements in heart function after one year, according to clinical trial results reported last fall.

Compared to baseline, all three patients in the phase 1 trial’s first cohort tolerated the treatment well and showed clinically meaningful improvements in endpoints such as left ventricular ejection fraction (LVEF) and New York Heart Association (NYHA) Class III symptoms at month 12. The therapy has an excellent safety profile and no adverse events were reported in cohort one. The 12-month follow-up of cohort two is ongoing.

Last May, researchers from Asklepios BioPharmaceutical, Inc. (AskBio) submitted an abstract (#28) to the 2022 Annual Meeting reporting on a first-in-human dose escalation clinical trial testing the therapy, which is delivered via a one-time injection into the heart’s coronary arteries. In their 2022 abstract, the team reports developing a novel adeno-associated viral vector, AAV2i8, that is cardiotropic and de-targets the liver to deliver a constitutively active protein phosphatase 1 inhibitor 1 (I-1c) - AAV2i8.I-1c.

Gene Therapy Improves Heart Function in Mice with ARVC

A research team from Tenaya Therapeutics submitted an abstract for presentation at the 25th Annual Meeting about an AAV-based, PKP2 gene therapy improving heart function in a mouse model of Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC). They concluded that their first-in-class gene therapy, TN-401, has the potential to address key aspects of ARVC, including arrythmias, progression of adverse heart remodeling, and overall survival.

In November 2022, TN-401 received orphan drug status from FDA. Tenaya is currently conducting IND-enabling studies for the program and expects to submit an IND this year to eventually conduct a clinical trial.

The Annual Meeting program is updated twice per week, so please check back for more information to be added as it’s confirmed. Thank you to everyone who submitted their research! We look forward to learning from it in less than two months in Los Angeles and virtually.

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