HONG KONG – U.S.-based biotech firm Egenesis Inc., headquartered in Cambridge, Mass., has received $100 million through a series B funding round for the development of safe and effective human-compatible organs through gene editing.

"With this new round of financing from industry leaders, Egenesis is well-positioned to continue to advance the development of human-compatible organs to address the dire shortage in the U.S. and around the world," said Paul Sekhri, president and CEO of Egenesis.

According to the company, more than 110,000 patients are on the organ transplant waitlist in the U.S. alone. And from that pool, around 20 patients pass away each day because they are unable to find a suitable organ donor. Globally, it is estimated that there are between 1.5 million to 2 million people on the organ transplant waitlist currently.

Egenesis plans to use animal organs to address that problem, starting with pig kidneys.

"The concept of cross-species organ replacement, known as xenotransplantation, has re-emerged due to recent advancements in gene editing led by Egenesis, and will become a safe and effective solution for the hundreds of thousands of patients currently on the organ transplant waitlist globally," said Sekhri.

The cash infusion will enable the acceleration of Egenesis' kidney xenotransplant program into clinical testing, as well as support the development of a range of its other xenotransplant programs across islet cells, livers, hearts and lungs. The focus of the company is to rapidly advance an entirely new set of options across the transplantation field.

The series B was led by Fresenius Medical Care Ventures (FMCV), with new investors including Leaps by Bayer and Wellington Partners getting in on the action. Existing investors for the round include companies such as Arch Venture Partners, Biomatics Capital, Alta Partners and Khosla Ventures.

"We believe Egenesis is poised to revolutionize the entire organ transplantation market," said Jürgen Eckhardt, the head of Leaps by Bayer. "This could save lives in a way that was previously not thought possible just a few years ago."

Sustainable organ replacement is one of the 10 areas of engagement and investment, called "leaps," for Leaps by Bayer.

Olaf Schermeier, the CEO for global research and development at Fresenius Medical Care, hailed Egenesis' approach as "a truly transformational option for patients with kidney disease."

Overcoming the hurdles

The idea of xenotransplantation is not new. The first serious attempts, known as heterotransplantation, were first recorded in scientific literature in 1905, and it has been explored with limited success over the last century.

Pigs have long been considered the most suitable donor for humans. But there have been two significant barriers, namely endogenous retroviral transmission and immunology compatibility.

Luhan Yang, the co-founder and chief scientific officer of Egenesis, told BioWorld that the capability of multiplexable genome engineering enables Egenesis to address the two fundamental hurdles of xenotransplantation.

"We have demonstrated that we can use clustered regularly interspaced short palindromic repeats (CRISPR) to eradicate all the active endogenous retroviruses, and we are also using genome editing tools to modify the immunology makeup of the organ to achieve compatibility with the human immune system," said Yang.

"We are in the preclinical testing stage of the kidney program. We are using a nonhuman primate model and transplant pig kidneys into this animal model to test the efficacy and safety," said Yang.

But she said it was too early to comment on how the trials would develop. However, Egenesis is working with closely with the FDA to facilitate its kidney xenotransplantation IND-enabling study.

Yang also stated that islets would be the secondary indication for the firm's xenotransplantation efforts. And it would involve bringing together leading scientists from China and the U.S.

"We have close collaboration with Hangzhou Qihan Bio Inc., our sister company in China for islet indication. They are also working diligently with the local regulatory entity to facilitate the islet xenotransplantation IND-enabling studies," she said.

Hangzhou, China-based Qihan Bio raised $20 million in its own series B funding round in July. It had also received $7.8 million in series A financing in August 2018. The company was established by Yang in 2017. (See BioWorld, Aug. 7, 2018.)

Despite the promising outlook, Yang is still cautious about revealing a business strategy just yet.

"We are in the early stages of conducting patient research and are hearing about the tremendous need for additional organs that can work. It's too early to comment on the exact commercial strategy at this time," she said.

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