Early-generation kinase medicines are limited by poor selectivity, off-target effects and the emergence of resistance.

Our founding scientists envisioned a new generation of highly selective kinase medicines that could prolong lives, improve quality of life and prevent disease recurrences, with more potent target inhibition, fewer off-target effects and opportunities for therapeutic combinations.

They worked to build a scalable platform that could yield multiple selective kinase medicines for a wide range of diseases, bringing this innovation to as many patients as possible.

A look inside our labs

Our scientists merge expertise in bioinformatics and structural and cell biology, world-class drug design capabilities and a proprietary library of novel compounds to create a new discovery paradigm. This unique approach enables us to rapidly identify compelling disease targets and design highly selective kinase medicines.

The result is a robust and diverse portfolio of clinical- and research-stage programs targeting genomically defined cancers, rare diseases and cancer immunotherapy. With a steadfast commitment to scientific excellence and to patients, we aim to rapidly and unequivocally change the face of medicine.

Meet our Scientists

Klaus//
Kerrie//
Marion//
Fabien//
Chandra//

Klaus Hoeflich: Vice President, Biology

My path

I earned my B.S. (Hon) at Simon Fraser University in British Columbia, and my Ph.D. in Medical Biophysics from the University of Toronto; in 2000, I was awarded a Medical Research Council of Canada Next Generation Award.

My experience

Following my time in research labs for my Ph.D. and postdoctoral work, I joined Genentech in South San Francisco, CA. I ultimately was responsible for small-molecule drug discovery and disease strategy initiatives. My track record includes multiple programs to first-in-human clinical trials, an FDA-approved medicine, Cottelic®, for the treatment of metastatic melanoma, and authoring more than 65 scientific articles. Currently, my team and I are focused on discovering transformative therapies in the areas of oncology, immunology and rare genetic diseases.

My biggest achievement

Over the years, I have had the opportunity to mentor young scientists and help them to achieve higher levels of responsibility and success.

My recent scientific publications

Senior author, “Targeting cancer with kinase inhibitors,” published in the Journal of Clinical Investigation in 2015.

Senior author, “Leveraging the Pre-DFG Residue Thr-406 To Obtain High Kinase Selectivity in an Aminopyrazole-Type PAK1 Inhibitor Series,” published in ACS Medicinal Chemistry Letters in 2015.

Senior author, “Back Pocket Flexibility Provides Group II p21-Activated Kinase (PAK) Selectivity for Type I 1/2 Kinase Inhibitors,” published in the Journal of Medicinal Chemistry in 2014.

Senior author, “Phosphoproteomic characterization of DNA damage response in melanoma cells following MEK/PI3K dual inhibition,” published in the Proceedings of the National Academy of Sciences in 2013.

Senior author, “P21-activated kinase 1 (PAK1) as a therapeutic target in BRAF wild-type melanoma,” published in the Journal of the National Cancer Institute in 2013.

See Klaus' other publications

My Blueprint Medicines

This crew is enthusiastic, knowledgeable and eager to collaborate. There is a deep commitment to scientific excellence that truly fuels our work.

Kerrie Faia: Principal Scientist, Translational Medicine

My path

I earned a master’s of science degree in Animal Science at the University of New Hampshire, and completed my training in the laboratory of Dr. Thomas Foxall.

My experience

Before joining Blueprint Medicines, I worked at other pharmaceutical companies in Cambridge: Leukosite, Millennium and Infinity Pharmaceuticals. While at Infinity, I had the privilege of working on three different discovery programs; all advanced to clinical development, and one is now FDA-approved.

My biggest achievement

I am proud to have been part of the team that helped bring Copiktra®, a dual PI3K-delta/gamma inhibitor, to patients with chronic lymphocytic lymphoma, small lymphocytic lymphoma and follicular lymphoma. It is the first FDA-approved drug that I have worked on directly.

My recent scientific publications

Co-author, “Duvelisib, an oral dual PI3K-δ,γ inhibitor, shows clinical and pharmacodynamic activity in chronic lymphocytic leukemia and small lymphocytic lymphoma in a Phase 1 study,” published in the American Journal of Hematology in 2018.

Lead author, “The phosphoinositide-3 kinase (PI3K)-δ,γ inhibitor, duvelisib shows preclinical synergy with multiple targeted therapies in hematologic malignancies,” published in PLoS One in 2018.

Co-author, “The Phosphoinositide-3-Kinase (PI3K)-Delta and Gamma Inhibitor, IPI-145, Overcomes Signals from the PI3K/AKT/S6 Pathway and Promotes Apoptosis in CLL,” published in Leukemia in 2015.

Co-author, “Pharmacological inhibition of PI3K-δ and PI3K-γ by IPI-145 abrogates both the adaptive and innate immune response and suppresses autoimmune and inflammatory disease in preclinical animal models,” published in Nature Chemical Biology in 2013.

See Kerrie's other publications

My Blueprint Medicines

Everything here is time-sensitive, and it’s impossible not to be motivated by this collective sense of urgency. In cancer immunotherapy, many patients either do not respond or eventually become resistant to therapy. Our challenge is to improve our understanding of tumor-immune cell interactions to help us better predict who will benefit in the clinic, and I am proud to be part of this effort at Blueprint Medicines.

Marion Dorsch: Chief Scientific Officer

My path

I earned an M.S. and a Ph.D. in Biology from the Free University of Berlin, Germany.

My Ph.D. thesis was focused on stimulating the anti-tumor immune response  through intratumoral cytokine expression.

My experience

After completing my postdoctoral research at Columbia University, I came to Cambridge, MA and joined Millennium Pharmaceuticals. I then went on to Novartis Institutes of Biomedical Research, Sanofi, and Agios Pharmaceuticals—all in Cambridge—before joining Blueprint in 2016.

My biggest achievement

During my time at Novartis, I led efforts to target the Hedgehog pathway, a developmental  pathway mutated in medulloblastoma and basal carcinoma. My team discovered and developed a small-molecule antagonist of Hedgehog signaling that ultimately became a treatment of metastatic basal cell carcinoma under the trade name ODOMZO®. In parallel we also elucidated novel resistance mechanisms to Hedgehog pathway inhibition and identified additional indications for hedgehog pathway antagonists. Our work was published in high-impact journals.

My recent scientific publications

Co-author, “AG-221, a first-in-class therapy targeting acute myeloid leukemia harboring oncogenic IDH2 mutations”, published in Cancer Discovery in 2017

Co-author, “Targeted inhibition of mutant IDH2 in leukemia cells induces cellular differentiation”, published in Science in 2013

Senior author, “Loss of the tumor suppressor Snf5 leads to aberrant activation of the Hedgehog-Gli pathway”, published in Nature Medicine in 2010

Senior author, “Targeting resistance to Smoothened antagonists by inhibiting the PI3K pathway”, published in Science Translational Medicine in 2010

Senior author, “Discovery of NVP-LDE225, a potent and selective biphenyl-3-carboxamide Smoothened antagonist”, published in ACS Medicinal Chemistry Letters in 2010

See Marion's other publications

My Blueprint Medicines

Our discovery platform –and our immensely talented cross-functional team –empowers us to pursue targets of interest quickly and nimbly. As a result, we’ve been able to deliver multiple clinical programs with proof-of-concept in defined patient populations and a strong portfolio of discovery programs. As a scientist, I find that incredibly motivating.

Fabien Llambi: Senior Scientist I, Biology

My path

I completed my scientific education in France, earning a B.Sc. at Université Jean Monnet and a M.Sc. and Ph.D. at Université Claude Bernard.

My experience

I came to the U.S. in 2006 as a postdoctoral fellow at St. Jude Children’s Research Hospital in Memphis, Tennessee. In 2012, I joined St. Jude’s as a scientist and remained until 2016.

My biggest achievement

My research at St. Jude’s focused on understanding apoptosis, or programmed cell death, in cancer cells. My work led to a better comprehension of the signaling pathways that regulate apoptosis, and how this process is intrinsically tied to the levels of stress experienced by cancer cells. I also uncovered an unconventional cell death pathway intricately wired to the proteasome pathway that controls the degradation of unfolded proteins.

My recent scientific publications

Lead author, “BOK is a non-canonical BCL-2 family effector of apoptosis regulated by ER-associated degradation,” published in Cell in 2016.

Lead author, “A unified model of mammalian BCL-2 protein family interactions at the mitochondria,” published in Molecular Cell in 2011.

Lead author, “Apoptosis and oncogenesis: give and take in the BCL-2 family,” published in Current Opinion in Genetics & Development in 2011.

Co-author, “Interfering with multimerization of netrin-1 receptors triggers tumor cell death,” published in Cell Death and Differentiation in 2009.

See Fabien's other publications

My Blueprint Medicines

Coming from the world of academia, I wondered if I would find working on the industry side as fulfilling as my previous research. Here, I’ve discovered not only molecules, but also skilled leaders, unique expertise and a fast-paced, collaborative environment.

Chandra Miduturu: Principal Scientist, Chemistry

My path

After completing my undergraduate education and a master’s degree in Chemistry in India, I earned my Ph.D. in Chemistry from the University of Illinois, Urbana-Champaign, followed by postdoctoral research training in cancer biology and biological chemistry at Dana-Farber Cancer Institute/Harvard Medical School in Boston.

My experience

I’m a founding employee of the Blueprint Medicines discovery team, and one of the company’s first hires.

My biggest achievement

During my post-doctoral training period, I showed the utility of screening a small library of kinase inhibitors across the human kinome as a strategy to discover new scaffolds and starting points for kinase drug discovery. At Blueprint Medicines, I played a key part in validating the role of the FGFR4 kinase in hepatocellular carcinoma (HCC, the most common form of liver cancer), using a covalent small-molecule FGFR4 inhibitor.

My recent scientific publications

Co-author, “GNF-2 Inhibits Dengue Virus by Targeting Abl Kinases and the Viral E Protein,” published in Cell Chemical Biology in 2016.

Co-author, “First Selective Small Molecule Inhibitor of FGFR4 for the Treatment of Hepatocellular Carcinomas with an Activated FGFR4 Signaling Pathway,” published in Cancer Discovery in 2015.

See Chandra's other publications

My Blueprint Medicines

Our approach to kinase drug discovery is truly unique. As a founding member, I was fascinated by the potential of our approach to kinase drug discovery—especially in designing and understanding small molecule inhibitors, validating novel targets and identifying clinical candidates.

Klaus
Kerrie
Marion
Fabien
Chandra

“We’re entering a new era of precision medicine… we can now rapidly and reproducibly design potent and selective kinase medicines for previously undruggable as well as newly discovered drivers of disease. Combined with recent advances in molecular profiling, we believe these next-generation kinase medicines can quickly achieve clinical proof-of-concept and, ultimately, transform patient care.”

Marion Dorsch, Ph.D., Chief Scientific Officer

Scientific Advisory Board

Giulio Draetta, M.D., Ph.D.

Director, Institute for Applied Cancer Science at MD Anderson Cancer Center, and Professor of Molecular and Cellular Oncology

Brian Druker, M.D.*

Director, Oregon Health & Science University Knight Cancer Institute, and JELD-WEN Chair of Leukemia Research

Carlos Garcia-Echeverria, Ph.D.

Global Head of Research Platforms, Sanofi

Scott Lowe, Ph.D.*

Member, Cancer Biology and Genetics Program at Memorial Sloan Kettering Institute Cancer Center, and Chair, Geoffrey Beene Cancer Research Center

Nick Lydon, Ph.D.*

Founder, Granite Biopharma LLC

Charles L. Sawyers, M.D.*

Director, Human Oncology and Pathogenesis Program at Memorial Sloan-Kettering Cancer Center

Alice T. Shaw, M.D., Ph.D.

Associate Professor of Medicine, Harvard Medical School, and Thoracic Oncologist, Massachusetts General Hospital