Highlights: American Society of Gene & Cell Therapy Meeting

illustration of gene therapy using light points

The annual meeting of the American Society of Gene & Cell Therapy (ASGCT) was held in Washington, DC this week, with literally hundreds of abstracts and presentations. Here’s a look at some of the highlights.

Children with X-linked MTM Breathe on Their Own after Gene Therapy

Researchers with Audentes Therapeutics, Hopital Armand Trousseau in Paris, UCLA Medical Center, Children’s Hospital of Chicago and several other institutions, presented data from a Phase I/II clinical trial of gene therapy in X-linked Myotubular Myopathy (XLMTM). XLMTM is a rare monogenic disease caused by mutations in the MTM1 gene, which codes for myotubularin, which is a protein needed for development and function of skeletal muscle. The disease results in extreme muscle weakness, respiratory failure and early death. Of the nine boys who received the gene therapy, all have improved neuromuscular function, most can sit on their own and four can now breathe on their own without ventilators. Principal investigator Perry Shieh, a neurologist at UCLA, said the boys “have gone from nothing to something. Time will tell how much that something will be.”

The gene therapy is AT132, which delivers functional copies of the MTM1 gene to skeletal muscle cells.

MaxCyte Presented Data on CARMA Trial in Cancer

Gaithersburg, Md.-based MaxCyte presented data on its CARMA drug candidate, MCY-M11, a mesothelin-targeting chimeric antigen receptor (CAR), which is in a Phase I clinical trial in mesothelin-expression solid tumors at the National Cancer Institute (NCI) and Washington University in St. Louis.

The CARMA platform is noted for its rapid manufacturing and delivery capabilities without a viral component, which was also a significant aspect of the presentation. “The advancement of our first CARMA clinical trial, which is consistently showing the feasibility of our rapid manufacturing process, is significant for MaxCyte and the application of our technology,” stated Claudio Dansky Ullmann, MaxCyte’s chief medical officer. “Development of a cell therapy with application in solid tumors is impactful for patients with unmet needs in a variety of cancers and we look forward to further advancing this program.”

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Moderna Presented Research from 7 mRNA-based Therapies, and 5 Oral Presentations

Moderna always has a lot of irons in the fire and they presented research on many of them at the meeting. The data included preclinical research in ornithine transcarbamylase deficiency (OTC) in collaboration with the Perelman School of Medicine at the University of Pennsylvania; maple syrup urine disease (MSUD), also in collaboration with Penn; arginase-1 (ARG1) deficiency in collaboration with the University of California, Los Angeles Department of Surgery and Department of Molecular and Medical Pharmacology; Factor VIII deficiency (hemophilia A), in collaboration with Seattle Children’s Research Institute; and several other programs.

“mRNA medicines have the potential to treat the underlying cause of many metabolic diseases, and may offer important advantages over conventional gene and enzyme replacement therapies for eligible patients,” stated James M. Wilson, director of the Gene Therapy Program at Penn. “This includes the potential to develop controlled, dose-dependent and transient treatments that may benefit infants and children with these disorders and patients with diseases that are not addressable with current viral-based approaches.”

Translate Bio Presented mRNA Therapies in Metabolic Disorders

Translate Bio presented “Treatment of Metabolic Disorders Using Lipid Nanoparticle (LNP)-Encapsulated Messenger RNA Therapeutics (MRT).” The presentation summarized data from research in three mouse models of metabolic disorders including ornithine transcarbamylase (OTC) deficiency, arginosuccinate synthetase (ASS1) deficiency (citrullinemia) and methylmalonic acidemia (MMA).

“We have engineered our mRNA therapeutic (MRT) platform to deliver mRNA that codes for the missing enzymes in several metabolic disorders,” stated Frank DeRosa, Translate Bio’s senior vice president of research and process development. “These data demonstrate the potential broad applicability of the MRT platform to treat various genetic disorders using our proprietary liver delivery technology.”

Homology Medicines Discusses Gene Therapy Platform

Homology Medicines discussed its manufacturing platform, including serum-free suspension transfection, which is both scalable and optimized to manufacture its adeno-associated virus vectors (AAVHSCs). Specifically, its AAVHSC15 vector showed an 18-fold decrease in phenylalanine compared to an AAV5 vector carrying the identical DNA construct in the phenylketonuria (PKU) murine model.

“We have a great sense of urgency to advance our gene therapy and gene editing programs with the goal of bringing transformative treatments and potential cures to patients with rare genetic diseases,” stated Albert Seymour, Homology’s chief scientific officer. “We are pleased to share the progress that we have made across our PKU and MLD development programs, as well as demonstrate the scalability, quality and superiority of our manufacturing process for our human-derived AAVHSCs. We are carrying this momentum forward in our IND-enabling studies in MLD and in our gene editing program for PKU and look forward to starting our Phase I/II pheNIX trial in adults with PKU and reporting initial clinical data this year.”

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