This Company Raised $100 Million To Bring Gene Therapy To The Masses – Forbes

Kriya Therapeutics cofounder and CEO Shankar Ramaswamy.

Over the past several years, breakthroughs in gene therapy have led to treatments for rare diseases that were deadly just a decade ago. Take Zolgensmain 2019, it was the first gene therapy approved by the FDA to treat spinal muscular atrophy, a rare genetic disease that affects the mobility of infants and children. But gene therapies have historically had two drawbacks: They are only used for rare diseases, and they carry a hefty price tag (treatment with Zolgensma costs $2.1 million).

Kriya Therapeutics is trying to overcome these obstacles by creating gene therapies for the massesand manufacturing them at a lower cost. On Wednesday, the startup announced that it had raised a $100 million Series B funding round to get it closer to this goal. The round was led by investors from Patient Square Capital and also involved investors from QVT, Dexcel Pharma, Foresite Capital, Bluebird Ventures, Transhuman Capital, Narya Capital, Amplo and the Juvenile Diabetes Research Foundation T1D Fund.

We think this is going to be an extraordinarily important therapeutic class that will revolutionize the treatment of many diseases, says Jim Momtazee, managing partner at Patient Square Capital.

The Silicon Valley-based company was founded in late 2019 by three pharmaceutical industry alums, including a former cofounder of Spark Therapeutics and the former president of United Therapeutics Corp. Shankar Ramaswamy, Kriyas CEO, was part of the foundational team at Roivant Sciences. The new round brings the companys total funding to $180 million; the company declined to reveal its valuation.

Kriyas main focus is its uniquely designed Adeno-associated virusesviruses that are harmless when they enter the body, but deliver instructions to cells that then pump out genes that are missing in some people with genetic diseases. Though the company still plans to develop treatments for rare diseases, what sets it apart is its focus on more common genetic diseases, like some forms of diabetes and obesity. So far, gene therapy has been really constrained in many respects from achieving its full potential, Ramaswamy says. We are believers in gene therapy being applied to rare diseases as well as prevalent diseases.

Ramaswamys goal is to build a company that can go from genetic target discovery to manufacturing and then full commercialization of new therapies, unlike a typical biotech startup that might partner with a large pharmaceutical company for the later stages of development (Ramaswamy says the company will be open to partnerships, but can bring a drug to commercialization on its own). Once the company discovers and develops new gene therapies, its 51,000-square-foot manufacturing facility in North Carolina can produce Adeno-associated viruses at scale to deliver the genes to patients in need. Ramaswamy says that capability will bring down the cost, with savings passed along to patients. I think the innovations that were delivering will make gene therapies much more affordable and accessible to patients, he says. We are very committed to not being a burden on the healthcare system.

The companys current pipeline of products are all preclinical, though Ramaswamy says that they plan to submit Investigational New Drug applications to the FDA for several products in late 2022 and early 2023. So far the company is developing gene therapies for type 1 diabetes, solid tumors and two eye conditions: geographic atrophy and uveitis. In the U.S., more than 3 million people combined have at least one of these conditions, meaning Kriya has a huge pool of potential customers. By comparison, there are fewer than 25,000 children and adults with spinal muscular atrophy in the country.

Ramaswamy says that the new capital will go toward continuing the companys explosive growthit now has 80 full-time employeesas well as refining its vector delivery platforms and manufacturing capabilities. In the future, the money will allow the company to continue to develop new gene therapies for diseases both common and rare. Were taking a very new approach, which is to think more broadly, Ramaswamy says.

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This Company Raised $100 Million To Bring Gene Therapy To The Masses - Forbes