BioPharm America - Hot in Boston

September 9, 2013

These companies are representative of the quality of innovation and creative business models that characterize the Boston-area biotech region. Meet with these companies, and many others, at BioPharm America 2013, Sept. 17-19.


RaNA Therapeutics is leveraging 20 years of oligonucleotides research to target a new, unexplored, druggable space. Fundamentally, RaNA’s approach selectively activates therapeutic protein expression to treat or prevent disease.

“We’re trying to expand beyond the starting point of the platform and identify its limits. This approach has so much potential,” said President and CEO Arthur Krieg. “We have eight programs we’re actively working on and taking forward. We want to develop this as broadly as we can.”

RaNA plans to have the first program in clinical trials in 2015. Ultimately, the company would like to keep a few programs in-house and partner the rest.

To expand partnering and acquisition options, RaNA was formed as a limited liability corporation. This enables partners to acquire the programs they want without also purchasing other, unwanted programs or acquiring staff. Instead, the staff remains with

RaNA’s operating company and continues to work on other programs. This structure enables RaNA and its partners to gain maximum value and increases the flexibility for the involved companies, thus efficiently generating returns to shareholders.


When Moderna Therapeutics and AstraZeneca partnered this spring to develop a disruptive early stage therapeutic technology, it signaled a significant shift in the life science investment ecosystem. “This shows that when pharmaceutical teams see a company with innovative science and an experienced management team to make the promise a reality, they are willing to invest and take risks,” noted Stéphane Bancel, President and Founding CEO, Moderna Therapeutics.

Going against the trend to focus upon late-stage development, AstraZeneca paid Moderna Therapeutics USD $240 million up front to access Moderna’s messenger RNA Therapeutics™ platform, which is still in preclinical development. The technology enables the body to produce therapeutic protein in vivo, making it possible to treat thousands of diseases that otherwise are undruggable.


All previous tetracyclines now on the market were developed with semi-synthetic methods that begin in bacteria and were followed by limited chemical modification. That traditional process limited the chemical diversity, he explained. That lack of diversity makes it very difficult to create tetracycline antibiotics that address multidrug resistant bacteria. As a case in point, only one tetracycline antibiotic has received FDA approval in the past 42 years.

In contrast, since its inception in 2006, Tetraphase has identified more than 50 novel chemical scaffolds and more than 2,800 novel analogs of tetracycline, enabling them to address many more infectious threats than the tetracyclines currently on the market. This explosion of possibilities is based upon the discoveries of Andy Myers at Harvard University. Tetraphase licensed exclusive, worldwide rights to Myers’ proprietary technology and has further developed this in-house. Guy Macdonald, President and CEO of Tetraphase Pharmaceuticals, Inc. said, “we have the opportunity to take an efficacious and safe class of drugs and then reinvigorate it.”

Registration for BioPharm America is now open and partnering has begun.

Click here to register. MassBio members receive $200 off of registration by using the code “MassBio” in the comments field.


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