The Bio Report

The Broad Institute has emerged victorious in a battle with researchers at the University of California, Berkeley over patents covering breakthrough gene-editing technology that allows scientists to easily and inexpensively alter genetic material with precision. Last month, the Patent Trial and Appeal Board of the U.S. Patent and Trademark Office ruled that patents held by the Broad Institute relating to certain aspects of CRISPR-Cas9 gene editing did not interfere with those being sought by UC Berkeley researchers. We spoke to Kevin Noonan, Partner and Chair of the Biotechnology & Pharmaceuticals Practice Group of McDonnell Boehnen Hulbert & Berghoff and founding author of the Patent Docs blog, about the decision, what it means, and to what extent it resolves patent issues regarding the gene-editing technology.

Earlier this month the National Academy of Sciences and the National Academy of Medicine issued a report that considered the scientific, ethical, and governance issues surrounding human genome editing. The report comes as new gene editing technologies have reduced the cost and increased the ease of manipulating the human genome. We spoke to Alta Charo, co-chair of the study committee that wrote the report and professor of Law and Bioethics at University of Wisconsin-Madison, about the study, the potential for this science, and where the committee thought limits should be imposed.

The emergence of cancer immunotherapies, drugs that activate and enlist the immune system to fight cancer, has emerged as a promising approach to combat the disease in its many forms. Lawrence Fong, co-leader for the Cancer Immunotherapy Program at the University of California, San Francisco, stands at the intersection of several initiatives involving UCSF to better understand the immune system’s response to cancer and develop more effective drugs in the battle against it. We spoke to Fong about the potential of immunotherapies, the various efforts underway at UCSF, and what we known and don’t know about the immune system.

The start of Trump administration, a new cabinet, and a new Congress are raising concerns within the scientific community about how the changes in Washington will affect the health of science and innovation in the United States. At issue is not only funding for research, but a range of policy decisions relating to everything from public health to climate change that some fear will not be informed by science. We spoke to Mary Woolley, president of Research!America, about the new administration, why it is critical for scientists to become advocates, and the planned March for Science in Washington, D.C. this April.

For a number of diseases that require chronic administration of a substance naturally produced by the body, patients may have to rely on regular injections or infusions. Sernova is working to free patients from these types of regimes with it combination device and cell therapy that implants a pouch that contains living cells that produce the missing hormone, factor or other substance. We spoke to Phil Toleikis, CEO of Sernvoa, about its technology, the challenges involved in such an approach, and the company’s initial focus on diabetes and hemophilia.

Telehealth, which encompasses an array of technologies to deliver healthcare, is a rapidly growing area. Stratus Video, a language services company, is making a push into telehealth where it sees a big opportunity to leverage its expertise and apply it to healthcare. We spoke to Lee Horner, president of the Stratus Telemedicine division, about telehealth trends, how its altering the practice of medicine, and whether it can deliver on the promise of improving care while reducing costs.

Drug discovery and development is a slow and costly process, but the Regeneron Genetics Center represents a drugmakers’ bet that harnessing large amounts of genetic data can point the way to better targets, greater success rates, and ultimately better drugs. We spoke to Aris Baras, vice president and head of the Regeneron Genetics Center ahead of the Precision Medicine World Conference 2017 in Silicon Valley about how large genetic studies are brining fundamental change to the drug development process, the approach Regeneron is taking, and why its becoming a necessary element of drug development today.

Last year, KemPharm was on its way to winning an FDA approval for its opioid Apadaz designed to deter users from snorting, injecting, or otherwise abusing it. But the regulatory agency stymied the company’s efforts when it declined to label it as an abuse-deterrent opioid. Apadaz is a prodrug that is converted to its active form through an enzyme present in the intestinal tract that releases the hydrocodone in it. The decision sent shares in KemPharm tumbling. Now, as the company works to resolve regulatory issues around Apadaz, it’s moving forward with the rest of its prodrug pipeline. We spoke to KemPharm CEO Travis Mickle about abuse-deterrence, the ongoing discussion with the FDA, the company’s prodrug pipeline.

In the realm of mental health, a lack of biologic measures for diagnosing and treating patients has at times made this an area where doctor’s subjective decisions can play a disproportionate role. Ehave is seeking to improve diagnosis and treatment of patients with its data-rich management, assessment, and remediation tools that sit on its cloud-based software platform. The company’s initial product focuses on ADHD, although it plans to roll-out other applications in the future. We spoke to Prateek Dwivedi, CEO of Ehave, about its platform, the problems the company is trying to address, and how digital technologies are changing the way doctors diagnose, treat, and interact with patients.

With 2016 drawing to a close and the biotech industry gearing up for the annual JPMorgan Healthcare Conference in San Francisco, we continued our holiday tradition of checking in with Adam Feuerstein, senior columnist for TheStreet. We spoke to Feuerstein about the year in biotech that was, the winners and losers of 2016, and what to look for at the JPMorgan Healthcare Conference and beyond.

While there has been much buzz in the industry over the potential for harnessing the microbiome and using it as a way to treat disease, it remains early days for the therapeutic pipeline. Rebiotix, which is delivering human-derived microbes to treat recurrent Clostridium difficile infections, is ready to move its lead experimental therapeutic into late-stage clinical testing. The company says it is the most advanced human clinical program evaluating a microbiota-based drug conducted in coordination with a regulatory authority. We spoke to Lee Jones, founder and CEO of Rebiotix, about the microbiome, the opportunity for drugs targeting the colonies of bacteria in the gut, and the indications beyond c. diff Rebiotix is pursuing.

Investors bid up biopharmaceutical stocks following the election of Donald Trump as President of the United States. It reflected a belief that a Trump administration will remove the threat of price controls on drugs and bring about a friendlier regulatory environment at the FDA. We spoke to Amy Brown, senior reporter and author of the EP Vantage 2017 Preview, about what’s in store for the biopharmaceutical sector in the year ahead, what to look for, and why it may turn out to be a big year for M&A activity.

The advent of digital health devices and mobile health apps has the potential to improve care, change patient behavior, and permit doctors to intervene early when the health of a patient changes. But the American Medical Association wants to ensure that as these devices proliferate, doctors have a role in guiding their design and determining their worth. We spoke to James Madara, CEO of the AMA, about new principles its adopted to promote safe and effective mHealth applications, the concerns physicians have about the use of these apps, and how he sees them reshaping healthcare.

A clinical trial of an experimental vaccine against HIV that just began in South Africa is raising new hopes of making new strides against the disease. The trial follows recent news of tests in animals that suggest the vaccine, in combination with an innate immune stimulant may be able to put infected patients into remission. We spoke to Colonel Nelson Michael, director of the U.S. Military HIV Research Program at the Walter Reed Army Institute of Research, about the new trial, how this vaccine builds on what was learned from a clinical trial in Thailand from an earlier version of the vaccine, and why there’s hope of related work moving researchers towards a functional cure for the disease that could free patients from the need for antiretroviral therapy for extended periods.

Pain takes both a personal toll on individuals who suffer from it and an economic toll on society more broadly. Though there is a wide range of drugs available to treat pain, it’s a problem that is still not well addressed. The need for new pain therapies has become even more acute as the problem of opioid addiction has grown. We spoke to Kerrie Brady, founder and chief business officer of Centrexion Therapeutics, which is developing a pipeline of non-opioid pain drugs. Brady discussed the problem of today’s existing pain therapies, how the company is using Big Data in its pursuit of new drugs, and its strategy of building out its pipeline.

A number of studies have recently called attention to the lack of gender diversity in leadership positions in the life sciences industry. Women in BIO and LifeSci Advisors have taken steps to increase the presence of women in the boardroom of biotech companies through a new training program to get women boardroom ready, as well as the creation of an initiative to help place qualified women onto the boards of companies. We spoke to Dawn Hocevar, president elect of Women in Bio and Michael Rice, founding partner of LifeSci Advisors about their efforts, why there aren’t more women on the boards of biotech companies today, and what has to be done to remedy the problem.

Perlara is a drug discovery company focused on using model organisms to find treatments for diseases previously believed to be too rare to cure. Recently Perlara entered into a drug discovery and development collaboration with Novartis, a deal that included an equity investment and is seen as validating Perlara’s platform. We spoke to Ethan Perlstein, CEO of Perlara, about the company’s unique approach to drug discovery, why it focuses on the diseases it does, and the significance of its agreement with Novartis.

There’s been much buzz about the microbiome, but what that constitutes can mean different things to different people. Biosortia Pharmaceuticals is focusing on the aquatic microbiome as a rich source of potential first-in-class small molecules to address unmet medical needs. We spoke to Ross Youngs, founder and CEO of Biosortia, about what makes the aquatic microbiome compelling as a source of new drugs, how the company goes about mining it for potential compounds, and what the company’s growing pipeline looks like.

It can take up to six years to accumulate enough evidence to support advancing an experimental drug into clinical development. twoXAR is working to rapidly accelerate that process through the use of Big Data and its proprietary algorithms to find screen large public and proprietary data sets to indentify new drug candidates and determine their efficacy. We spoke to Andrew Radin, co-founder and CEO of twoXAR, about the company’s technology, how it’s being applied today, and the evolution of the company’s strategy.

Robert Goldman had been a successful Internet age inventor having developed some of the critical technology underlying the way digital music is stored, searched, and purchased online. But when his sister developed cancer, he found himself turning his skills as an inventor into an unchartered area for him. Eventually he developed a new type of catherter to deliver cancer medicines directly to where they are needed in the body. But despite his company’s success at winning marketing clearance from the U.S. Food and Drug Administration, his company Vascular Designs has had difficulty raising money. On October 17 Vascular Designs launched a campaign to raise $500,000 through the crowdfunding site Indigogo. We spoke to Goldman about his IsoFlow catheter, the funding environment for innovative medical device companies, and his decision to turn to crowdfunding.