When the term “gene therapy” comes up, the first thing that comes to mind is often some sort of gene replacement or gene editing (either a DNA correction or disruption) to treat a well-defined Mendelian genetic disorder like sickle cell disease or phenylketonuria—not some broad, genetic, complex condition like inflammation or osteoarthritis. Well, unless you are Diem Nguyen, PhD, CEO of Xalud Therapeutics in New York City.

“Symptoms depend on the stage of disease and also which organs are affected,” said Dr. Emma Harvey, Global Head of Medical Affairs at the clinical-stage biopharmaceutical company F2G. “It starts as a respiratory disease and can be indistinguishable on symptoms from viral or bacterial pneumonia.”

Akihiro Ko is the CEO and Co-Founder of Elixirgen Therapeutics, which was founded with the mission of treating patients using RNA technology. Their c-srRNA, controllable self-replicating RNA, technology is being applied to telomere biology disorders where patients have inherited short telomeres and a failure to develop bone marrow. Elixirgen is looking to restore the ability to create blood cells in patients suffering from this rare disease.

My biggest challenge was financing Canary. I think the concept behind Canary is timely, highly medically relevant, and an obvious next step in the evolution of medical devices—life-saving

John Rex, MD, chief medical officer at drug discovery company F2G, told Becker’s he also feels the solution is two-part: legislative and research-driven.

If data is the new oil, CytoReason is one of its barons. Combining all available information about a disease – literature, data sets, all data types – the company builds computational disease models utilized by its pharma partners to help them determine which drug avenues to pursue next.

Elicio is merging with Angion Biomedica, a Uniondale, NY-based biotech that began searching for strategic alternatives last July after Phase II setbacks forced it to shutter all R&D work.

In 2021, PepGen raised $113 million to challenge Sarepta in DMD and build its neuromuscular and neurologic pipeline.