PALO ALTO, Calif.–(BUSINESS WIRE) — BridgeBio Pharma uit Californië hult zich niet langer in stilzwijgen door meer details te onthullen over haar nieuwe aanpak in de ontwikkeling van nieuwe therapieën voor genetische ziekten. Deze ziekten, vaak erfelijk en zeldzaam, hebben verwoestende gevolgen voor tientallen miljoenen patiënten in de Verenigde Staten, onder wie kinderen.
Het in 2015 opgerichte bedrijf is primair gericht op pre-commerciële producten door gebruik te maken van de voordelen in de verschillende fases van ontwikkeling, van oorsprong tot exploitatie.
BridgeBio Pharma combineert een traditionele focus op geneesmiddelen met een nieuw corporate model om in een grote behoefte van de geneesmiddelenindustrie te voorzien. Dat doet BridgeBio Pharma door in een vroeg stadium van genetische ziekten geneesmiddelen te ontwikkelen waar patiënten baat bij hebben.
PALO ALTO, Calif.–(BUSINESS WIRE)– California-based BridgeBio Pharma has come out of stealth mode to reveal more detail about its novel approach to developing new therapies for genetic diseases. These diseases, often inherited and individually rare, collectively exact devastating effects on tens of millions of patients in the US alone, many of whom are children. The company, formed in 2015, is primarily focused on pre-commercial products, utilizing its advantages in sourcing and operating to find and build value at each stage of development.
BridgeBio Pharma combines a traditional focus on drug products with a new corporate model to tackle a big industry need – translating early stage genetic disease science into drugs that matter for patients. “There’s a lot of money and interest in new technology companies, like those focused on gene editing, and then again for late stage clinical products,” says co-founder and CEO Neil Kumar. “But if you have a small molecule for a single genetic disease that is pre-clinical, it is harder to attract interest. We started BridgeBio to focus on those single product opportunities that target well defined genetic drivers of disease. BridgeBio is set up to move those programs forward efficiently and at scale.”
The company sees itself as a drug product engine as opposed to a novel science platform – “we partner closely with academic and clinical leaders to help move insights they have already made into the clinic. We are not trying to discover new biology so much as to take what is already known and develop therapies from it,” says co-founder Frank McCormick, former co-founder of Onyx Pharmaceuticals. BridgeBio’s team members have collectively been responsible for over a dozen marketed products and include drug development veterans Charles Homcy, Frank McCormick, Philip Reilly, Hoyoung Huh, Uma Sinha, and Robert Zamboni.
BridgeBio’s novel corporate structure was designed in collaboration with MIT Sloan Professor Andrew W. Lo, who is also a founding investor and member of BridgeBio. Rather than forming large platform companies, the team forms lean and focused subsidiaries around individual assets or diseases. These subsidiaries can draw on BridgeBio’s network of world-class genetic disease expertise to complement their internal efforts. This structure is ideal for building value in product-focused investments, and allows for rapid capital reallocation should an asset fail. Lo explains: “We tried to put in place, at the outset, a corporate structure that optimized for focused R&D at the level of each asset but that still provided diversification for investors. This diversification in turn provides more predictable positive outcomes and makes these pre-commercial programs more attractive for a broader pool of capital. Ultimately, the structure also allows for liquidity in ways that are unique as compared with the traditional c-corp or fund structures seen in this industry.”
BridgeBio has seven programs to date, with two in the clinic and hopes to add to its diversified portfolio of assets based on a systematic mapping of the genetic disease landscape. It has deployed over $50 M in R&D commitments in 2016.
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