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Start-Up Developing Regenerative Liver Disease Treatments

Damaged liver cells

Liver cells damaged by cirrhosis from alcoholism (Ed Uthman, Flickr)

8 August 2018. A new enterprise founded by scientists, is underway developing treatments for advanced liver disorders that combine new drugs with cell and gene therapies to restore or replace liver functions. Ambys Medicines in Redwood City, California begins operations with $140 million from its first venture funding round and a deal with drug maker Takeda gaining access to the company’s first products.

Ambys Medicines aims to discover and create new treatments for advanced liver disease. The company cites statistics showing some 600,000 people in the U.S. have cirrhosis or scarring of liver tissue from conditions such as hepatitis and alcoholism. Development of scar tissue in the liver prevents regeneration of new liver cells, a process considered irreversible, resulting in life-threatening complications. The only option to restore liver function is with a liver transplant, of which 7,000 are done in the U.S. each year. However, says Ambys, about 40,000 people die each year from chronic liver disease and cirrhosis.

The company aims to create liver disease treatments based on research by its scientific founders:

Martin Burke, professor of chemistry at University of Illinois in Urbana, who studies small molecule compounds that function like proteins

Markus Grompe, cell biologist at Oregon Health & Science University in Portland, and director of its stem cell center

Juan Carlos Izpisua Belmonte, who studies gene therapy and stem cells at the Salk Institute in La Jolla, California

Holger Willenbring, professor of surgery at University of California in San Francisco, who studies stem cell therapies for liver diseases

The company plans to combine cell and gene therapy with protein chemistry to develop restorative treatments for advanced liver diseases.  Gene therapies are expected to transfer healthy genes to damaged hepatocytes, or liver cells, to reprogram the cells’ functions. Cell therapies aim to grow healthy hepatocytes in the lab in large enough quantities for transplanting in patients. And Ambys plans to develop small molecule drugs that act like proteins to restore lost liver functions.

Ambys was formed by life science investment company Third Rock Ventures, in Boston and San Francisco. Jeffrey Tong, a partner with Third Rock, is Ambys’s interim CEO. “Major advances in the fields of gene and cell therapy and gain-of-function drug therapies,” says Tong in an Ambys statement, “coupled with deeper understanding of liver biology, create the potential to develop drug therapies that can now restore or replace natural liver function.”

The company’s first venture funding round of $60 million is provided by Third Rock and Takeda Pharmaceuticals in Osaka, Japan. Takeda is also gaining an option to license Ambys’s first 4 products that complete preclinical steps and reach the stage of investigational new drug applications to FDA, in effect permission to conduct clinical trials. If Takeda exercises its options, it will gain commercialization rights for these products outside the U.S., pay half of their development costs, and provide payments to Ambys for development and regulatory milestones. Ambys will retain full U.S. rights on its products.

Takeda says it’s committing $100 million to its partnership with Ambys Medicines. A first payment from Takeda along with its first venture funding round is providing Ambys with $140 million in initial financing.

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