Lexeo Therapeutics is teaming up with Big Pharma Johnson & Johnson's unit Abiomed to tap its Impella heart pump technology in search of a potentially safer and more effective route for cardiac genetic medicines.
Impella, which J&J touts as the world’s smallest heart pump, has a range of devices used for various heart conditions. Lexeo is seeking to use the technology as a novel preclinical method for delivering an early-stage gene therapy candidate to the heart.
Gene therapies have been at the cutting edge of life sciences for 30 years but have continued to be dogged by safety and efficacy issues.
Just in the past year, a spate of companies, including Big Pharmas Takeda and Biogen, have culled all their gene therapy work because of a host of issues.
Drugmakers have also reported new deaths tied to approved and experimental medicines, including Sarepta Therapeutics, which last year disclosed three patient deaths linked to liver toxicity from two of its drugs.
Gene therapy remains an exciting area of research, but the method of delivery is still a central piece of the puzzle. These therapies typically use modified viruses to deliver therapeutic genes to certain cells or tissues, with the aim of treating genetic diseases at the source.
While this adeno-associated virus (AAV) delivery system can work well, it can also trigger unwanted immune responses and raise targeting concerns, with the therapy sometimes reaching organs beyond those intended, reducing efficacy and tending to accumulate in the liver, which can create safety issues.
Lexeo is looking to get around this with Impella. The key idea is to use the heart pumps to provide a “localized cardiac delivery of gene therapy by concentrating AAV delivery to the heart by investigating cutting-edge routes of administration,” according to a Jan. 8 release.
By going directly to the heart, its intended target, “we aim to substantially reduce required AAV doses and improve gene therapy safety while maximizing transgene expression and clinical efficacy,” R. Nolan Townsend, Lexeo’s CEO, explained in the release.
Three months ago, J&J began a serious Class I recall after cybersecurity vulnerabilities came to light from its automated Impella heart pump controllers, although the pump remains on the market.
Early last year, Lexeo laid off 15% of its staff as part of a wider plan to siphon $20 million from its pipeline, all so it could fund its lead cardiac programs. It’s also working on a gene therapy for Friedreich's ataxia, with a registrational trial planned for this year.