The Bio Report

Gensight Targets Gene Therapy on Diseases that Blind

The France-based biotech Gensight Biologics is developing gene therapy treatments for rare, mitochondrial, and neurodegenerative diseases of the eye. Its lead therapeutic candidate is in late-stage trials for Leber’s Hereditary optic neurophathy, a sudden condition that afflicts young adults and leaves almost all of them blind. We spoke to Gensight’s CEO Bernard Gilly about the company, the challenges and promises of gene therapy, and why eye disease is a particularly attractive target for this emerging area of treatment.

A Vial Problem: How We Waste $3 Billion of Cancer Drugs a Year

Nearly $3 billion a year is wasted on cancer drugs because of the way they are packaged, according to a study in BMJ. These expensive drugs that are injected or infused come in fixed size vials and patients often require less drug than what is sold as a single dose. Because of restrictions on the ability to use leftover drug, the result is that it is usually thrown out. Nevertheless, drugmakers have little incentive to do things differently because the waste enhances their profitability. We spoke to Leonard Saltz, an oncologist with Sloan Kettering Memorial Cancer Center and coauthor of the study, about the findings, why Europe doesn’t have the same problem, and what can be done to stop the waste.

The Havoc-Wreaking Zombies Lurking in Life Sciences Boardrooms

The walking dead live, but it's value, not brains, they are eating. So-called zombie funds, life sciences venture capital funds that are fully invested and unable to raise new money, still maintain their board seats. But Outcome Capital’s Managing Directors Oded Ben-Joseph and Arnie Freeman say their divergent interest from their fellow board members often lead to disputes that can end the life of promising technologies and lead to acquisitions that leave much value on the table. We spoke to Ben-Joseph and Freeman about the boardroom dynamics were zombies lurk, the consequence of this board misalignment, and how acquirers may seek to leverage the situation

New FDA Chief Faces Challenges

Last month, the Senate confirmed Robert Califf, a cardiologist and clinical researcher from Duke University, as commissioner of the U.S. Food and Drug Administration. Though he was overwhelming confirmed, some expressed concerns about his ties to the pharmaceutical industry. Others used the confirmation process to protest the FDA’s handling of opioid painkillers as the problem of addiction to these drugs have become a growing concern. We spoke to Peter Pitts, a former FDA associate commissioner and president of the Center for Medicine in the Public Interest, about Califf, why he’s well suited for the job, and the challenges he will face in his new role.

Seeking a Best-in-Class Antibody at Bargain Basement Prices

Bird Rock Bio, a small, San Diego-based biotech, is planning to take aim at some of the biggest biologics on the market with an antibody in development to treat rheumatoid arthritis that it says it expects to market at an annual cost of just $2,000 a year. That compares to around $30,000 a year for drugs such as Humira, Remicade, and Enbrel that are TNF inhibitors. The experimental therapeutic known as Gerilimzumab targets IL-6. We spoke to Paul Grayson, president and CEO of Bird Rock Bio about the company, the benefits of targeting IL-6, and how the company expects to be able to offer the drug at such a disruptive price point.

Pharma Bro, Price Controls, and the Quest to Balance Innovation with Affordability

The embattled pharma executive Martin Shkreli, whether intentional or not, has ignited a long smoldering public policy discussion about the pricing of pharmaceuticals. As there are growing calls for steps to restrain drug prices, there are also industry concerns about the effect such actions would have on the ability to attract investment and pursue the development of high-risk, innovative therapies. We spoke to Denis Corin, CEO of Q BioMed, an acceleration and development company, for a perspective on the fallout from Shkreli, how to balance innovation with affordability, and whether he expects any lasting impacts on small drug developers.

California’s Stem Cell Agency Plots Its Future

The California Institute for Regenerative Medicine, California’s voter-established stem cell agency, is thinking about its future. The institute recently issued a strategic plan for the next five years that it says represents a “radical overhaul of the way the institute conducts business” and emphasizes “coordination, speed, partnerships, and patients.” We spoke to Randy Mills, president of the California Institute for Regenerative Medicine, about the strategic plan, the ambitious goal it has set for the next five years, and whether CIRM will have a life beyond the $3 billion voters approved for its funding.

ViaCyte Pursues ”Functional Cure” for Type 1 Diabetes

About 1.3 million people in the United States have type 1 diabetes, which requires constant monitoring and regular injections of insulin. The autoimmune disease attacks the insulin producing beta cells in the pancreas and replacement of those cells has long been viewed as an ideal approach to treating the disease. The problem, though, has been finding a reliable supply or replacement cells and protecting them from the body’s immune system while allowing them to recieve needed nutrients, as well as deliver insulin to the body. San Diego-based ViaCyte is now in human clinical trials with a promising approach to treating the disease. We spoke to Paul Laikind, president and CEO of ViaCyte, about the company’s cell therapy, its critical financial support from the California Institute for Regenerative Medicine and JDRF, and why its investigational product may represent a functional cure.