Moving a great idea targeting diabetes out of the lab and into a company

Tejal Desai in her lab at UCSF: Photo courtesy Todd Dubnicoff

It’s always gratifying to see research you have helped support go from being an intriguing idea to something with promise to a product that is now the focus of a company. It’s all the more gratifying if the product in question might one day help millions of people battling diabetes.

That’s the case with a small pouch being developed by a company called Encellin. The pouch is the brainchild of Tejal Desai, Ph.D., a professor of bioengineering at UCSF and a CIRM grantee.

Encellin’s encapsulation device

“It’s a cell encapsulation device, so this material can essentially protect beta cells from the immune system while allowing them to function by secreting insulin. We are placing stem cell-derived beta cells into the pouch which is then implanted under the skin. The cells are then able to respond to changes in sugar or glucose levels in the blood by pumping out insulin.  By placing the device in a place that is accessible we can easily remove it if we have to, but also we can recharge it and put in new cells as well.”

While the pouch was developed in Dr. Desai’s lab, the idea to take it from a promising item and try to turn it into a real-world therapy came from one of Dr. Desai’s former students, Crystal Nyitray, Ph.D.

Crystal Nyitray: Photo courtesy FierceBiotech

After getting her PhD, Nyitray went to work for the pharmaceutical giant Sanofi. In an article in FierceBiotech she says that’s where she realized that the pouch she had been working on at UCSF had real potential.

“During that time, I started to realize we really had something, that everything that pharma or biotech was looking at was something we had been developing from the ground up with those specific questions in mind,”

So Dr. Nyitray went to work for QB3, the institute created by UC San Francisco to help startups develop their ideas and get funding. The experience she gained there gave her the confidence to be the co-founder and CEO of Encellin.

Dr. Desai is a scientific advisor to Encellin. She says trying to create a device that contains insulin-secreting cells is not new. Many previous attempts failed because once the device was placed in the body, the immune system responded by creating fibrosis or scarring around it which blocked the ability of the cells to get out.

But she thinks their approach has an advantage over previous attempts.

“This is not a new idea, the idea has been around for 40 or more years but getting it to work is hard. We have a convergence of getting the right cell types and combining that with our knowledge of immunology and then the material science where we can design materials at this scale to get the kind of function that we need.

Dr. Nyitray ““If we can reduce fibrosis, it really helps the cells get nutrients better, survive better and signal more effectively. It’s really critical to their success.”

Dr. Desai says the device is still in the early stages of being tested, but already it’s showing promise.

“We have done testing in animals. Where the company is taking this is now to see if we can take this to larger animals and then ultimately people.”

She says without CIRM’s support none of this would have happened.

“CIRM has been really instrumental in helping us refine the cell technology piece of it, to get really robust cells and also to support the development to push the materials, to understand the biology, to really understand what was happening with the cell material interface. We know we have a lot of challenges ahead, but we are really excited to see if this could work.”

We are excited too. We are looking forward to seeing what Encellin does in the coming years. It could change the lives of millions of people around the world.

No pressure. 

CIRM-funded medical research and development company does $150M deal to improve care for dialysis patients

Fresenius & Humacyte

Nearly half a million Americans with kidney disease are on dialysis, so it’s not surprising the CIRM Board had no hesitation, back in July 2016, in funding a program to make it easier and safer to get that life-saving therapy.

That’s why it’s gratifying to now hear that Humacyte, the company behind this new dialysis device, has just signed a $150 million deal with Fresenius Medical Care, to make their product more widely available.

The CIRM Board gave Humacyte $10 million for a Phase 3 clinical trial to test a bioengineered vein needed by people undergoing hemodialysis, the most common form of dialysis.

Humacyte HAV

The vein – called a human acellular vessel or HAV – is implanted in the arm and used to carry the patient’s blood to and from an artificial kidney that removes waste from the blood. Current synthetic versions of this device have many problems, including clotting, infections and rejection. In tests, Humacyte’s HAV has fewer complications. In addition, over time the patient’s own stem cells start to populate the bioengineered vein, in effect making it part of the patient’s own body.

Fresenius Medical Care is investing $150 million in Humacyte, with a plan to use the device in its dialysis clinics worldwide. As an indication of how highly they value the device, the deal grants Fresenius a 19% ownership stake in the company.

In an interview with FierceBiotech, Jeff Lawson, Humacyte’s Chief Medical Officer, said if all goes well the company plans to file for Food and Drug Administration (FDA) approval in 2019 and hopes it will be widely available in 2020.

In addition to being used for kidney disease the device is also being tested for peripheral artery disease, vascular trauma and other cardiovascular indications. Lawson says testing the device first in kidney disease will provide a solid proving ground for it.

“It’s a very safe place to develop new vascular technologies under clinical study. From a regulatory safety standpoint, this is the first area we could enter safely and work with the FDA to get approval for a complete new technology.”

This is another example of what we call CIRM’s “value proposition”; the fact that we don’t just provide funding, we also provide support on many other levels and that has a whole range of benefits. When our Grants Working Group – the independent panel of experts who review our scientific applications – and the CIRM Board approves a project it’s like giving it the CIRM Good Housekeeping Seal of Approval. That doesn’t just help that particular project, it can help attract further investment in the company behind it, enabling it to expand operations and create jobs and ultimately, we hope, help advance the field as a whole.

Those benefits are substantial. To date we have been able to use our funding to leverage around $2 billion in additional dollars in terms of outside companies investing in companies like Humacyte, or researchers using data from research we funded to get additional funding from agencies like the National Institutes of Health.

So, when a company like Humacyte is the object of such a lucrative agreement it’s not just a compliment to the quality of the work they do, it’s also a reflection of our ability to pick great projects.