MacDougall Musings

ASGCT Recap: A Wave of Cell & Gene Therapy Innovation

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Last month’s American Society of Gene & Cell Therapy (ASGCT) Annual Meeting showcased the field’s growing momentum in biotech. From perennially covered companies, such as Moderna and Pfizer, to young startups – more companies than ever are advancing cell & gene therapy (CGT) programs. Read on to learn about the biggest stories from the conference and current trends in CGT.

Innovation Across the Board

Given that more than 1,600 abstracts were presented at this year’s conference, companies needed a big name, a strong story or compelling data to grab the media’s attention. As has been the case since the field’s genesis, most of the programs featured focus on the treatment of either rare genetic conditions or cancer. However, early preclinical validation for numerous novel platforms is emerging – while the first gene therapy programs to receive FDA approval have all utilized a similar recombinant adenovirus approach, new modalities like in vivo CAR T technology or adeno-associated virus (AAV) and lentivirus (LV) combination therapies are gaining traction.

Startups Making a Splash

Companies like Moderna and Pfizer received media coverage for data from their programs in rare diseases, but several small startups earned attention for their promising new programs:

  • Kelonia Therapeutics is advancing a gene therapy platform powered by “iGPS™ technology” (built on a lentiviral vector engineered with a targeting molecule). With an aim to deliver allogeneic CAR-T therapies unincumbered by traditional toxicity and manufacturing hurdles, Kelonia shared preclinical data indicating that their platform technology effectively delivered CAR molecules to T cells in humans and non-human primates.
  • Rocket Pharmaceuticals is developing gene therapies that utilize both AAV and LV modalities, sharing data from multiple clinical-stage programs at the conference. Notably, data from a Phase 1/2 study in its severe leukocyte adhesion deficiency-I (LAD-1) program showed 100% overall survival at 12 months, and Phase 2 data from its Fanconi anemia program demonstrated substantial genetic and phenotypic correction in patients with at least a year of follow-up; the company plans to file both programs for FDA approval by the end of the year.
  • Tune Therapeutics’ TEMPO platform leverages an alternative form of CRISPR called “genetic tuning” which directly alters the epigenome rather than slicing DNA to make targeted changes to DNA packaging and accessibility within cells. In the clinical data presented at the conference, the company’s technology was effective in targeting a gene associated with cholesterol to reduce levels of LDL, a type of cholesterol connected to heart disease, by more than 50% in monkeys.

By 2024’s annual meeting, the face of CGT could be further transformed – the industry is anticipating 10 potential regulatory decisions around cell and gene therapies this year alone, and there are countless programs that could be ready to unveil exciting data in the next 12 months.

Fall is a busy time of year for conferences. Need help refreshing your investor deck or identifying target events? Reach out to the MacDougall team here and be sure to reference our continuously updated conference calendar.