New Funding Boosts URMC Biotech Start-Up for Neurological Disorders

Oscine Therapeutics, a new biotechnology company based on discoveries made and developed at the University of Rochester Medical Center (URMC) has received a significant multi-year investment to support both research and development of cell-based therapies for neurological disorders.  The funding represents the largest-ever investment in a URMC start-up company.

The new venture is based on decades of research in the lab of Steve Goldman, M.D., Ph.D., co-director of the URMC Center for Translational Neuromedicine. Goldman’s work has focused on understanding the basic biology and molecular function of support cells in the central nervous system, devising new techniques to precisely manipulate and sort these cells, and studying how cell replacement could impact the course of neurological diseases.

Goldman has developed techniques to manipulate the chemical signaling of embryonic and induced pluripotent stem cells to create the brain’s support cells, called glia. A subtype of these, called glial progenitor cells, gives rise to the brain’s main support cells, astrocytes and oligodendrocytes, which play important roles in the health and signaling function of nerve cells.

“Neurological disorders are complex diseases, but in many instances it appears that faulty support cells of the brain are driving the disease process,” said Goldman, the URMC Distinguished Professor of Neuroscience and Neurology. “These diseases represent promising targets for cell-replacement therapies because we know a great deal about the role these cells play, how to create them, and how to get them to the areas of the brain where they are needed.”

The investment in Oscine is being made by Sana Biotechnology, a new company focused on creating and delivering engineered cells as medicines for patients. The company is led by a team of biotechnology industry veterans with extensive experience in cell therapy, gene therapy, and gene editing. The company is backed by visionary investors including Arch Venture Partners, Flagship Pioneering, and F-Prime Capital Partners.

In many neurological diseases, such as multiple sclerosis and Huntington’s, glial cells have impaired development and function, or are simply lost during the course of the disease. This results in the disruption of communication between nerve cells, leading to the motor, cognitive, and behavioral symptoms of these disorders.

Goldman’s lab has developed new methods to replace the sick glial cells found in these diseases with healthy ones. In research involving animal models of these diseases, this approach has slowed, and for some disorders even reversed, disease progression.

The new investment – the terms of which were not publicly disclosed – will support R&D by Oscine focused on bringing these cell-based therapies to the clinic. The research will be conducted at URMC under a sponsored research agreement and will support 21 full-time staff, with researchers in Rochester and additional staff in Seattle and New York City.  The manufacturing of cells for clinical delivery will use protocols developed at Rochester.

“Cell-based therapies hold significant promise, and while progress has been made in areas such as cancer, there is a significant unmet need in diseases of the central nervous system,” said Christina Trojel-Hansen, Ph.D., the CEO of Oscine, who co-founded the company along with Goldman and spearheaded its organization and fundraising. “The support from Sana will enable us to advance important research in this field and work with an established team that has experience in bringing cell-based therapies through clinical trials and into clinical practice. I am also deeply grateful for the team at the University of Rochester for their efforts to ensure that these important scientific discoveries can now advance toward a clinical application.”

“Our bread and butter at ARCH is in starting companies with top researchers in world-class universities across the globe who are working on transformative discoveries, and pairing those seminal innovations with hard-charging entrepreneurs,” said Paul S. Thurk, managing director with Arch Venture Partners. “We found that golden mix with Dr. Goldman and Dr. Trojel-Hansen at Oscine.”

The University of Rochester and Cornell University have licensed intellectual property to Oscine, and both the University of Rochester and Goldman hold equity stakes in the company. Goldman also serves as Oscine’s President. The negotiation of the license and sponsored research agreement terms was led by Matan Rapoport, Ph.D., M.B.A., senior licensing manager with UR Ventures, the University of Rochester’s technology transfer office.

“This investment underscores the cutting-edge nature of the neurological cell therapy research program at URMC,” said Steve Dewhurst, Ph.D., vice dean for Research at the University of Rochester School of Medicine and Dentistry. “We are excited that the basic science discoveries made here at the Medical Center are the basis for innovative, first-in-class therapies that hold the potential to change the lives of people with these devastating neurodegenerative diseases.”

Goldman and the Center for Translational Neuromedicine maintain labs in both Rochester and at the University of Copenhagen in Denmark. Goldman’s research for cell-based therapies has received support from the National Institute of Neurological Disorders and Stroke, the National Institute of Mental Health, the Dr. Miriam and Sheldon G. Adelson Medical Research Foundation, the Lundbeck Foundation, the Novo Nordisk Foundation, CHDI, and NYSTEM.


Media Contact
Mark Michaud
(585) 273-4790

VPG Medical featured in Rochester Business Journal

University of Rochester start-up, VPG Medical, was recently featured in the Rochester Business Journal:

Among the companies in the Luminate NY accelerator, VPG Medical Inc. is a bit unique.

While most companies in the accelerator are based around developing hardware products in the optics, photonics and imaging space, VPG is a software company, developing a platform that can be used with built-in smartphone, tablets and computer cameras. In a software atmosphere where virtually every possible use of a phone’s capabilities has been explored, it’s tough to imagine what kind of tech a company could produce to put them in the largest optics accelerator in the world without even having a physical product.

That is, until founder and CEO Jean-Phillippe Couderc explains just what they do.

“What is quite amazing about this technology is that it uses the embedded camera from the smart devices to capture the electrical activity of the heart,” Couderc said. “It actually detects subtle changes in the color of your skin that occurs each time your heart beats.”

Read the full feature here.

Handy Gelbard Authors STAT News Piece Touting Multi-Target Drugs

Precision medicine is saving lives, but Harris A. (Handy) Gelbard, director of Center for Neurotherapeutics Discovery at URMC, believes we’re overlooking another class of extremely important treatment candidates: multi-target drugs. In STAT News, he shares his views on why U.S.-based pharmaceutical companies should pay closer attention to these drugs—including a promising compound discovered right here at URMC.

Read the full article here. 

Startup Company Aims to Improve Music Education

Learning a musical instrument requires dedication, practice, and time. Making performance errors is a natural part of the learning process; however, learning delays can occur when errors go uncorrected during individual practice. A recently established startup company, Shenzhen Mango Future Education Technology Co (Mango Future), has developed a product to address this problem.

Mango Future aims to improve and revolutionize the way people learn to play musical instruments. The company’s first product is Lian – a smartphone-application-based intelligent assistant for instrument practicing. The application, which is based on core technologies developed by University of Rochester Professor Zhiyao Duan, can listen to and track a musician’s performance on the score in real time, providing feedback on pitch and rhythm accuracy. The University licensed the rights to that technology to Mango Future.

The smartphone application enables the student to identify and correct errors as they practice and is intended to supplement musical instruction. Currently, the application is available to users in China and the United States. The company intends to expand its application to other string instruments, and eventually to brass and wood instruments.

Read more and download the application here.

Registration Open for Small Business Owners Boot Camp

The Ain Center for Entrepreneurship’s Buzz Lab Boot Camp: Four Saturdays for a Better Business is accepting applications for its second cohort. Designed for entrepreneurs, small business owners and aspiring innovators from the Greater Rochester community, the series of interactive workshops will run Saturdays, February 9, 16, 23, and March 2 from 10 a.m. to 3 p.m. on the River Campus. Accepted applicants will receive scholarships provided through a grant from the Economic Development Administration within the US Department of Commerce. The cost to participate is $25 and includes lunch for all four Saturdays. Those who complete the program will receive a certificate from the University. View the brochure and register online by February 6.

Rochester represents at Nobel Prize ceremony

The groundbreaking work of three scholars with ties to the University of Rochester took center stage when pioneering laser scientist Donna Strickland ’89 (PhD) and her Rochester advisor Gerard Mourou formally received the Nobel Prize in Physics, and Paul Romer, whose early career tenure-track appointment was in Rochester’s Department of Economics, received the Nobel Prize in Economic Sciences.

Strickland, who is now an associate professor of physics and astronomy at the University of Waterloo in Ontario, Canada, and Mourou, now a professor at the École Polytechnique in France, are being recognized for their Rochester work to develop “chirped-pulse amplification,” a technology that harnesses the power of lasers as precision tools and helped pave the way for laser-eye surgery, the machining of key parts for cell phones and other devices, tools for cancer treatment, and other clinical and commercial applications. The technology was the basis for Strickland’s 1988 doctoral dissertation at Rochester, where she was a graduate student working with Morou at the University’s Laboratory for Laser Energetics. They both receive a share of the Nobel Prize in Physics.

Romer, who was an assistant professor in the Department of Economics from 1982 to 1988 shortly after receiving his PhD from the University of Chicago, will be awarded the Nobel Prize for his work to assess the interaction of technology, productivity, and economic growth. He shares the prize with economist William Nordhaus of Yale University.

Watch the Nobel Prize ceremony online and read messages of congratulations from members of the University of Rochester community.

From left, Paul Romer, whose early career tenure-track appointment was in Rochester’s Department of Economics, received the Nobel Prize in Economic Sciences. Pioneering laser scientist Donna Strickland ’89 (PhD) and her Rochester advisor Gerard Mourou formally received the Nobel Prize in Physics. (Getty Images photos)



Koning Breast CT Nominated for Best Medical Technology

Koning Corporation’s breast computed tomography (KCBT 1000) system was nominated for Best Medical Technology at the 12th annual Prix Galien awards. The Prix Galien awards recognize outstanding achievements in improving the human condition through the development of innovative therapies. Worldwide, the Prix Galien is regarded as the equivalent of the Nobel Prize in biopharmaceutical and medical technology research.

Koning Corporation’s breast CT device, based on the patented research of Dr. Ruola Ning, is the only FDA-approved breast CT imaging system on the market. A unique modality, the KBCT provides 3D images of the breast – without compression and with high dose efficiency within the 10-second scan. This technology allows for precise detection, assessment, and quantification of abnormalities, especially in women with dense breast tissue. With the potential to dramatically improve the way clinicians visualize and evaluate breast tissue, Koning hopes to be able to improve survival rates and outcomes for millions of patients.

The KCBT’s nomination for Best Medical Technology represents a significant confirmation of the technology’s value. The Koning Corporation was honored to be recognized by the Prix Galien.

(L-R) Global Chief Operating Officer, Lutao Ning, Global Chairman and CEO, Dr. Ruola Ning, North American President, David Georges, and Global Chief Financial Officer, Matt Stack, attend the 12th annual Prix Galien awards on October 24, 2018.


Latest Drug Development Pilot Awards Announced

Five projects received Drug Development Pilot Awards in the latest round:

Development of UR214-7 and UR214-9 for cancer treatments
Kyu Kwang Kim, Department of Obstetrics and Gynecology

Small Molecule Modulators of U2AF-Dependent Splicing in Myelodysplastic Syndromes
Clara L. Kielkopf, Department of Biochemistry and Biophysics

Innate Immunity guided screening for apicomplexan profilins
Felix Yarovinsky, Department of Microbiology and Immunology

Creation and development of fully-transmembrane receptor effectors
Omar Bakht and Omar Aljitawi, Department of Medicine, Hematology / Oncology

A new alarmin S100A11 in host defense and inflammation
Felix Yarovinsky, Department of Microbiology and Immunology

The Drug Development Pilot Award Program (DDPA) supports projects in drug development research that result in technology development, extramural funding, and commercialization. This program is restricted to SMD faculty and awards funds up to $10,000 or $50,000, depending upon the award type.

To learn more about the DDPA, visit:

Latest Technology Development Fund Awards Announced

The Technology Development Fund (TDF) made four awards in the latest round. Awards ranged from $50,000 – $100,000 for projects of one-year in length.

Interested to learn more about the projects funded in this round? Contact us.

Fiber Array to Silicon Photonic Chip Optical Packaging
Jaime Cardenas, Institute of Optics

Pre-clinical development of a human monoclonal antibody cocktail (URnFluhmAb) for the universal prevention and treatment of influenza infection
James Kobie, Department of Medicine and Luis Martinez-Sobrido, Department of Microbiology & Immunology

Development of URML-3881, a small molecule MEK inhibitor for the treatment of cancers with high MAP kinase pathway activity
Rachael Turner, Department of Hematology/Oncology

Inscriptr Gene Therapy
Douglas Anderson, Department of Medicine

The TDF solicits proposals twice a year. Qualified applicants include University of Rochester faculty, students, or staff who have filed an invention disclosure with UR Ventures. Eligible projects propose the development of a technology to a more commercial endpoint. Awards range from $40,000 to $100,000.

To learn more about the TDF, visit: