NEW HAVEN A Yale neurology and neuroscience professor who hopes to regrow neurons in patients with spinal cord injuries has secured enough funding for a human clinical trial.

Stephen Strittmatters New Haven-based company, ReNetX, also has a new CEO, Erika Smith, who joined the biomedical venture in June after leaving her post as director of Yale Universitys $10-million Blavatnik Fund for Innovation.

The company has raised about $20 million from the National Institutes of Health and other sources to carry out the first stages of a trial involving people with tetraplegia, paralysis of all four limbs and the torso.

Strittmatter said his approach works like a double negative. He identified a receptor that inhibits the growth of nerve fibers and then created a decoy to block it. That leaves the neurons free to grow or regrow, in this case naturally, as they do in early development.

We have this huge hospital and medical complex, Strittmatter said last week at his office in the Boyer Center for Molecular Medicine, 2 miles from the Yale New Haven Hospital Spine Center. We dont have a single drug that promotes neuroconnection. So if we get it to a trial and if the trials successful, it opens up a huge number of doors to all kinds of additional therapies and multiple diseases.

Yale

Erika Smith, CEO of ReNetX and former director of the Yale Blavatnik Fund for Innovation.

Erika Smith, CEO of ReNetX and former director of the Yale Blavatnik Fund for Innovation. (Yale)

ReNetX, formerly known as Axerion Therapeutics, went public with its new name, its funding progress and Smiths hire on Monday, though she joined the company June 30.

Sitting outside a New Haven coffee shop the next week, she recalled how people have told her the new logo looks retro, a bit 90s, with its teal and blue, space-age lettering.

I said, I like that. Its kind of Star Treky like this brave new world and thats kind of how we think about the company, too, Smith said.

Strittmatter the companys founder and science adviser is certainly exploring one of the final frontiers in medicine. No treatments available today can regenerate nerve cells in the adult brain and spinal cord, where neuron regrowth is extremely limited.

Spinal cord injury is one of the most significant unmet medical needs with an annual cost of more than $5 billion per year, Smith said. A treatment that could mitigate even only a part of the condition could improve quality of life of these patients.

ReNetXs treatment would be delivered as an injection into the spinal column, similar to an epidural.

The trial, which is pending regulatory approval, could begin in as little as 15 months and, if successful, the treatment could begin to restore some sensation and control to patients limbs within six months to a year. From there, the company would seek to apply the therapy to glaucoma and then stroke damage.

I wish things could move faster but on the other hand, its very encouraging to see the progress we do have, Strittmatter said, adding that he and others have spent 20 years trying to solve the problem of nerve fiber disconnection.

Its really sort of the culmination of probably hundreds of peoples work, hundreds of man-hours, woman-hours, he said. But thats what it takes to actually make a difference.

The fact that Smith has joined the company as CEO is further evidence that Strittmatters work is one step closer to leaving the lab for the marketplace.

Rebecca Lurye

Stephen Strittmatter, a neuroscience and neurology professor at Yale University and founder of ReNetX, works in his New Haven laboratory on Friday, July 21.

Stephen Strittmatter, a neuroscience and neurology professor at Yale University and founder of ReNetX, works in his New Haven laboratory on Friday, July 21. (Rebecca Lurye)

The two met when Strittmatter was applying for a grant from the fund that she directed, which seeks to bridge the gap between breakthrough research and commercialization. As Smith says, the fund tries to prevent an innovation from existing as just a paper.

Let it be something thats life changing, she said.

Strittmatter won the grant in May, $300,000 toward another of his areas of interest, Alzheimers disease. Soon after, Smith learned there was an opening for the top job at his company.

Shed spent more than 25 years as an investor and entrepreneur in life sciences, both at Yale and as senior director of investments with the Center for Innovative Technologies.

But no company tempted her away from her behind-the-scenes roles until ReNetX.

Theres amazing companies I had a chance to engage with, but this was just the right one at the right time with the right team, she said. It was just serendipity.

Excerpt from:

Yale Scientist Aiming To Reverse Spinal Cord Injuries - Hartford Courant