With its potential to treat an enormous range of diseases, genetic medicine has become a focal point for many large drug companies. Novartis, for instance, just last month announced plans to spend $800 million to purchase privately held Gyroscope Therapeutics, which is developing a gene therapy for an eye disorder that can lead to blindness.
Lilly, too, has been upping its investments. The company dove into genome editing research in late 2020 through a collaboration with Precision Biosciences. Another deal came shortly thereafter, with Lilly shelling out nearly $900 million to acquire Prevail Therapeutics, a gene therapy maker whose work centers on diseases of the brain and central nervous system, namely Parkinson’s disease and dementia.
Such deals, alongside a growing list of approvals from the Food and Drug Administration, have bolstered confidence in the viability of genetic medicine. But like most cutting-edge technologies, the field is still working through challenges.
Some of the biggest hurdles have to do with getting these therapies inside the right cells. It’s an already complex process, and is made more so by the series of defenses the body deploys to restrain or destroy foreign genetic material.
Doctors from the University of British Columbia recently detailed these obstacles in an article published in Nature, describing how therapies made from nucleic acids — the building blocks of DNA — can be broken down by enzymes, trigger undesirable immune system responses or have chemical properties that make it difficult for them to travel into cells.
“Safe and effective nucleic acid therapeutics therefore require sophisticated delivery platform technologies,” the doctors wrote.
A number of small biotechs now operate on the premise that their platforms are advanced enough to overcome these problems. Ring Therapeutics, which is trying to create gene therapies using an unconventional type of virus, raised $117 million last summer through a financing round that attracted interest from a handful of big-name venture capital firms, including Moderna founder Flagship Pioneering.
Other biotechs, such as Dyno Therapeutics, Intergalactic Therapeutics and Ensoma, have raised tens of millions of dollars on the belief that they, too, could solve some of the delivery issues currently hindering gene therapy development.
Entos is part of this group as well, and now has the backing of a large partner in Lilly. The new deal represents “an important validation of the potential value of [Entos’] expertise and technology platform,” the company’s founder and CEO, John Lewis, said in a statement Thursday. It’s the second partnership in two months for Entos, which began working with BioMarin Pharmaceutical in November.
Moving forward, Entos will create and optimize the proteo-lipid vehicles that will be used in the partnered programs. Lilly, meanwhile, will select the vehicles that advance into clinical development and commercialization.
