CGTLive's 2024 Pillars of Progress: Most-Read Conference Coverage Stories

For all of 2024, our team was following the clinical development of targeted and novel engineered approaches to the treatment of patients with various medical disorders. These efforts included holding in-depth conversations with experts in the clinical care of these individuals, as well as in cell and gene therapy development, culminating in our coverage of each step of progress that the most exciting cellular and genetic treatments have made along the pipeline.

Many of those conversations happened on-site (or virtually) at meetings of various medical organizations around the world. A key locale for identifying the important updates around ongoing clinical trials and the latest new research that's being done, conferences are part of the lifeblood of CGTLive's efforts to keep our audience in the know about cell and gene science.

Here, we'll offer previews of some of the most-read stories from CGTLive's conference coverage in 2024. Click the buttons to read further into these stories.

CRISPR-Editing EBT-101 Therapy Safe, Temporarily Suppresses HIV Infection

“Initial data from the EBT-101-001 trial provides important clinical evidence that a gene editing treatment modality can be safely delivered for targeting the HIV DNA reservoirs in human cells. This study provides researchers with invaluable insights for how CRISPR technology can be applied for addressing infectious disease and was an important first step towards additional programs designed to optimize this treatment modality for treating the millions of individuals who are impacted by HIV and other infectious disease."
— Rachel M. Presti, MD, PhD

Excision BioTherapeutics’ EBT-101-001 phase 1/2 trial (NCT05144386) of EBT-101 CRISPR-based gene editing therapy has met its primary safety endpoint and its secondary biodistribution/immunogenicity endpoint. Data from the trial were presented at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, Maryland by principal investigator Rachel M. Presti, MD, PhD, a professor of medicine at Washington University School of Medicine, St. Louis.

Vertex’s Diabetes Cell Therapy on Track to Meet End Point of Eliminating Severe Hypoglycemic Events

“This positive data adds to the growing body of evidence for VX-880’s potential to revolutionize the treatment of type 1 diabetes that would give patients an alternative solution other than exogenously administered insulin. These findings will also support further evaluation of VX-880, and we hope to see this treatment become a pivotal development in type 1 diabetes care.”
— Piotr Witkowski, MD, PhD

Data continue to support VX-880 (Vertex Pharmaceuticals)’s potential to eliminate or reduce the need for insulin use in patients with type 1 diabetes (T1D). Updated data from the phase 1/2 FORWARD trial (NCT04786262) were presented at the American Diabetes Association 84th Scientific Sessions, held June 21-24 in Orlando, Florida, and virtually, by investigator Piotr Witkowski, MD, PhD, a professor of surgery and the director of the Pancreatic and Islet Transplant Program at University of Chicago, Illinois.

Nanoscope Therapeutics’ Retinitis Pigmentosa Gene Therapy MCO-010 Improves Visual Acuity in Randomized Controlled Clinical Trial

“The durable statistically significant vision improvement achieved at multiple time points during the 2-year study is highly noteworthy. This degree of improvement has never been observed in a randomized, controlled trial of a highly heterogeneous severe vision-loss patient population. These findings deliver hope to patients and ophthalmologists that a solution is close to being found. We are finally on the brink of an impactful in-office, mutation-agnostic gene therapy for people with severe vision loss.”
— Allen C. Ho, MD, FACS, FASRS

Nanoscope Therapeutics’ MCO-010 (sonpiretigene isteparvovec), an investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy, has improved visual acuity in patients with retinitis pigmentosa (RP) in comparison to patients who received a placebo in data from the phase 2b RESTORE clinical trial (NCT04945772). The data were presented at the American Academy of Ophthalmology 2024 Annual Meeting, held October 18 to 21 in Chicago, Illinois. The randomized, controlled clinical trial treated patients with either a high dose of MCO-010, a low dose of MCO-010, or a sham–control.

Genethon’s Duchenne Muscular Dystrophy Gene Therapy GNT0004 Produces Expression of Microdystrophin in Phase 1/2/3 Trial

“DMD is a rare, X-linked progressive disease caused by mutations in the dystrophin gene, leading to dystrophin deficiency in muscles. The lack of dystrophin in myocytes results in progressive muscle degeneration that manifests primarily as muscle weakness and early death during the second or third decade of life, with the most common cause of death being cardiorespiratory failure. GNT0004 is a recombinant serotype 8 AAV vector-based gene therapy containing a shortened, but functional version of DMD gene (hMD1) optimized with key functional domains of full-length dystrophin. The hMD1 transgene is driven by aSpc5.11 promotor, which leads to expression in skeletal and cardiac muscle target tissues.”
— Francesco Muntoni, MD, et al

Genethon’s GNT0004, an investigational recombinant adeno-associated virus (AAV) vector-based gene therapy intended to treated Duchenne muscular dystrophy (DMD), has demonstrated the ability to produce expression of microdystrophin, among other signs of efficacy, in a phase 1/2/3 clinical trial. The data were presented by Francesco Muntoni, MD, the principal investigator of the trial and the chair of paediatric neurology at University College London Great Ormond Street Institute of Child Health, at the Myology 2024 International Scientific Congress, held April 22 to 25, in Paris, France.

Kyverna’s CAR-T KYV-101 Shows Some Durable Responses in Autoimmune Disease, With One Relapse Reported

"CAR T-cell therapies are rapidly establishing themselves as the new North star in the autoimmune disease treatment universe. We are seeing rising interests in unlocking the full potential of cell therapies in rheumatological and neurological B-cell driven autoimmune diseases."
— Peter Maag, PhD

Kyverna Therapeutics’ KYV-101, an investigational CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy being evaluated for various autoimmune indications, has shown mixed efficacy results in newly reported data from the first set of patients treated. The data were presented at the European Alliance of Associations for Rheumatology (EULAR) European Congress of Rheumatology 2024, held June 12-15, in Vienna, Austria.