CT aims to compete in precision medicine. What will it take?

By, Arielle Levin Becker, CTmirror.org

HARTFORD: Connecticut has the potential to become a national leader in the growing field of precision medicine, which aims to tailor disease prevention and medical treatment to individuals’ unique genetic code, environment and experiences, scientists and industry officials told a state economic competitiveness panel Friday.

But that potential comes with caveats, several said: Connecticut is far from alone in seeking to be a top player in the field. Other states have put money into supporting the field, and Connecticut must too, some researchers said.

drpeter bowers anthem Alpern Robert wks YaleNews McGee Joe Fairfield Business council 2014 Dr Murat Gunel
Dr. Peter Bowers, Anthem Blue Cross Blue Shield Dr. Robert Alpern, Yale School of Medicine  Joe McGee, Fairfield County Business Council Dr. Mural Gunel, Yale Center of Genomic Analysis

“This opportunity is huge from an economic perspective. I think the question is whether Connecticut wants to play,” Dr. Peter Bowers, chief medical officer at Anthem Blue Cross and Blue Shield, told members of the Connecticut Commission on Economic Competitiveness and the legislature’s Commerce Committee Friday.

During the meeting at the Legislative Office Building in Hartford, commission members wrestled with issues including Connecticut’s ability to compete and whether there’s a role for people without college degrees in a field based on advanced science and medicine.


Genetic risk of autism spectrum disorder linked to evolutionary brain benefit

By Bill Hathaway

New Haven: Genetic variants linked to autism spectrum disorders (ASD) may have been positively selected during human evolution because they also contribute to enhanced cognition, a new Yale study suggests.

A study based on a genome-wide association study of ASD conducted by the Psychiatric Genomics Consortium and information regarding evolutionary gene selection showed that inherited variants linked to ASD were found under positive selection in larger numbers than would have been expected by chance.

The final version of the paper was published Feb. 27 in the journal PLOS Genetics.   

By, Mitchell Young

NEW HAVEN: When a company reports organic revenue growth of 18.6% year over year, hats typically get tipped to the company, all over Wall Street.
New Haven’s Alexion Pharmaceuticals [ NASDAQ: ALXN] did just that and more, its fourth quarter income was up substantially, from $67 million to $93 million. That’s real money, in anyone’s book, but there remain Alexion doubters still.

Alexion logo colorOnly a couple of months back the company parted ways with its CEO David Hallal and CFO Vikhas Sinha and installed an interim CEO, David Brennan a board member and former CEO of Astra Zeneca.


cancer Cell with nucleus 22123754NEW HAVEN:  A Yale-led research team has found that a melanoma cell and a white blood cell can fuse to form a hybrid with the ability to metastasize. The hybrid contains the properties of a white blood cell allowing it to migrate through the body taking the Cancer DNA in the hybrid along with it.

According to senior research scientist and study author John Pawelek collaborated with colleagues at the University of Colorado Anschutz Medical Center and the Denver Crime Laboratory to examine how cancer spreads from solid tumors.

rosenberg uconnFARMINGTON: CaraGen an emerging immunotherapy company is collaborating with researchers at the University of Connecticut in Farmington to develop a vaccine for treatment of patients with colon cancer.

CaroGen employs what it says is a “transformative virus-like vesicle (VLV) platform technology” developed by Professor John Rose at Yale University School of Medicine  

and exclusively licensed to CaroGen.

The UCONN effort is being led by UConn Health Professor of Medicine, Daniel Rosenberg, PhD, the collaboration has a specific target studied by UConn researchers Kepeng Wang, assistant professor of immunology, and Anthony T. Vella, professor and Boehringer Ingelheim Chair in Immunology.

Internal Investigation, Lawsuits, Management Changes, A Failed Clinical Trial and New Competition, Heighten Speculation About the Future.

AlexionHQWinter2016NEW HAVEN: The Elm City’s new flagship company Alexion Pharmaceuticals [NASDAQ: ALXN] is finding itself under the microscope of lawyers, financial regulators, investment bankers and even bio-scientists as it reacts to significant financial and drug discovery events in the past 30 days.

In late November, the company announced an internal investigation into sales practices of its main revenue producing drug, Soliris. To date Soliris, with 2015 revenues of 2.5 billion has been the company’s sole financial driver.

A Novel Business Approach and New Technology Bring Funds To Two Companies

By Mitchell Young

NEW HAVEN: Biohaven, a privately held Hew Haven-based “pharmaceutical holding company,” has secured $80 million in new funding. Biohaven has created a diverse portfolio of potential drugs and drug platforms through investments and licensing. 

The company has licensing and drug development agreements with Yale University, Massachusetts General Hospital and Rutgers University, among others.

The company says its role is to apply its “expertise in late stage clinical development” to bring drug development forward.

Vlad Coric

Vlad Coric, M.D., CEO of Biohaven is marshalling funds and new drug candidates to company.

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Yale researcher and Kleo CEO found a local partner to help push his “novel” technology forward.

The FDA gave the company the Orphan Drug status this summer and the authority to begin a clinical trial for a drug to treat Spinocerebellar Ataxia [SCA], a rare, debilitating neurodegenerative disorder that is estimated to affect approximately 150,000 people in the United States. 

Vlad Coric, M.D., CEO of Biohaven, explained why the FDA moved forward with the designation and trial plan, “our clinical development program in Spinocerebellar Ataxia is particularly important because there are no currently approved drugs for this neurological disorder.”

The Orphan Drug designation is granted for drugs that can treat, prevent or diagnose diseases for fewer than 200,000 people in the U.S. Drugs granted that status, if eventually approved, receive an extended marketing exclusivity.

The financing was led by the venture capital firms Venrock, RA Capital Management, Vivo Capital, Aisling Capital, Rock Springs Capital, John W. Childs, Knoll Capital Management, Osage University Partners, Aperture Venture Partners, Connecticut Innovations, Greg Bailey and Litmore Capital, and two undisclosed “blue chip” pharmaceutical companies. Biohaven’s announcement said the offering was “oversubscribed.”

An investment by Biohaven in September in another New Haven biotech is likely one reason that investors were attracted. Biohaven provided an undisclosed amount of Series A financing to Kleo Pharmaceuticals.

Kleo’s CEO and Co-founder, David Spiegel, is Professor of Chemistry and Pharmacology at Yale University where he heads the Spiegel Research Group and has received wide acclaim and awards for his lab’s research.  While Spiegel co-founded Kleo, he was only appointed CEO in September in connection with the financing.

Biohaven’s investment in Kleo will help the company develop its “novel” Antibody Recruiting Molecules (ARMs) and Synthetic Antibody Recruiting Molecules (SyAMs). Both drug platforms are considered by the company to be able to create entirely new drug types.

The company plans to develop ARMs and SyAMs to treat cancer and infectious diseases.  

Spiegel explained the role he hopes his technology will take, saying, “Biologics have been the gold standard for immunotherapies. With ARMs and SyAMs, we have the opportunity to raise that bar. Our molecules are hundreds of times lighter than their biological counterparts and thus may infiltrate tissue more efficiently than large proteins.”

Of the investment by Biohaven he said, “Combining our expertise in ARMs and SyAMs with Biohaven’s investment and development expertise represents an ideal collaboration.”