The Rochester biotech start-up scene is getting a new member as a Mayo Clinic professor seeks to tap the commercial potential of regenerative medicine and stem cells in the battle against eye diseases such as macular degeneration.
 
Alan Marmorstein, a professor of ophthalmology at the Mayo Clinic Medical School, is the founder of LAgen Laboratories LLC, which was established last year. He is now in the process of raising $275,000 to launch the venture, according to documents submitted to the city by Rochester Area Economic Development Inc. (RAEDI).
 
The economic development group this summer sought and won a $100,000 loan from the city of Rochester’s Economic Development Fund for LAgen to retrofit a 3,500-square-foot leased commercial space on the city’s northwest side into a biologics manufacturing facility. The terms of the subsidy deal require LAgen to create at least four new full time-equivalent jobs within two years.
 
Marmorstein says he’s confident the new company, which until now has been basically a “bootstrap” operation based on stem cell technology he developed at Mayo, will soon be cash-flowing and contributing to Rochester’s quickly developing bio-business infrastructure.
 
“I’ve been in Rochester for three years now after living all over, and I can say that I’ve never seen a place that’s more supportive of start-ups,” he told TCB. “A lot of the credit goes to RAEDI.”
 
Marmorstein’s research at Mayo is focused on the goal of restoring or preventing vision loss due to common and inherited eye diseases and trauma. His studies include how mutations in certain genes cause inherited forms of macular degeneration; how pressure is regulated within the eye; and how some organisms regenerate their eyes in response to injury.
 
The regenerative medicine element comes in with the goal of developing new therapies that make use of “induced pluripotent” stem cells (iPSC) for diseases such as macular degeneration and glaucoma. One promising way of doing so is through bioengineering new retinal pigmented epithelium (RPE) cells, which, once they degenerate in the eye, can lead to severe visual impairment and blindness.
 
There are currently no consistently effective treatments for RPE degenerative diseases, so the disease burden of these conditions are expected to continue to rise. Age-related macular degeneration and retinitis pigmentosa are both leading causes of blindness in the developed world, affecting up to one-third of people over the age of 75.
 
Stem cell therapies, however, are providing new hope. Backers say cell transplantation into the human retina has the potential to restore vision and provide treatment for diseases that feature significant RPE loss. Since these diseases spare the inner retina and optic nerve, retinal transplantation trials have focused on replacement of the photoreceptors and RPE, and retinal stem cells have been shown to be efficient at integrating into the degenerative host retina.
 
The encouraging research into these therapies has created a growing demand for stem cell-derived RPE, and that’s where LAgen comes in. Marmorstein will be licensing the methodology he has developed at Mayo to produce RPE on a large enough scale to sell to research labs and pharmaceutical companies, thus relieving those users of what is for them is a long and cumbersome process of developing their own RPE lines.
 
One reason LAgen was able to land the support of RAEDI, the development group stated, is that its ready to produce revenues immediately, thanks to customer interest generated during its official launch at the May 2016 meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Vancouver, Canada. While LAgen did not take orders at the event, it collected interest and received verbal commitments for $30,000 to $40,000 in orders, and has the inventory in hand.
 
LAgen this summer announced one of its first customers – iPS Academia Japan, a company that commercializes iPSC research for the Japanese market, where human trials for RPE transplantation are underway.
 
“The long-range business plan is to eventually expand from the research market into the therapeutic market, perhaps through a spin-off,” Marmorstein said. “That, of course, would be a much bigger market.”