Our mission: We strive to treat more patients with ground-breaking therapies. This mission inspires and motivates us.

Strategic Principles

Pursue top quality science, internally and externally, balanced with medical need and commercial potential.

Strategy for value creation

Genocore Biotech’s strategy is designed to create value by leveraging the Company’s many strengths. These include its global leadership in the field of pulmonary arterial hypertension therapy, a strong and effective worldwide specialty commercial organization, a highly productive discovery capability and a unique company culture that focuses on delivering innovative medicines that improve patient’s lives.

We are the leaders in PAH therapy and, with our PAH products, we will continue to lead well into the future. During this time we must build our portfolio – through R&D and/or business development, to find additional meaningfully differentiated products to benefit patients. To realize our potential, we need to have a solid business – which means never losing sight of spending, whilst maintaining quality in all that we do.




  • Drive innovation forward - Pursue top quality science, internally and externally, balanced with medical need and commercial potential.
  • Maximize the value of innovation - Develop projects ourselves and seek partners or out-license when necessary to maximize value.
  • Leverage our global presence - Expand innovative commercial capabilities to new customers and regions. Manage alliances, putting the product first.
  • Insist on the highest quality in all we do - Quality is crucial and needs to be ingrained across all functions.


Research and Development Strategy

Genocore Biotech R&D strives to enhance and extend the lives of patients with serious illness. To advance this mission, we have built an organization distinguished by world-class scientists with a talent for applying novel approaches to treat disease.

In addition, we have devised an R&D strategy that aims to align and engage our talent, prioritize our investments, and seize scientific opportunities. This strategy is comprised of five major components:

Redefine our R&D Guiding Principles

  • Focus on innovative medicines for unmet needs in patients with serious illnesses.
  • Pursue targets that are validated in humans.
  • Maintain an expansive toolkit of drug modalities with a focus on biologics.
  • Focus on return on investment and operational efficiency.
  • Harness external innovation.
  • Demonstrate the value of our medicines.

Ensure major investments are based on compelling science

  • Embrace a "Pick the Winners" approach to accelerate high-potential programs based on targets validated in humans.
  • Partner or out-license lower priority assets.
  • Continue to acquire external innovation to complement internal capabilities and programs.
  • Refocus and differentiate our Discovery Research efforts
  • Focus on large effect sizes in serious illnesses.
  • Capitalize on our strengths in human genetics, novel biology, and protein engineering.
  • Leverage new platform technologies to build a robust immuno-oncology pipeline.

Pursue a "Biology First" approach to choose the right targets and tools

  • Focus on the biology of target selection first, with modality considerations second.
  • Consolidate small, medium, and large molecules into one integrated platform.
  • Focus on biologics while ensuring world-class small molecule capabilities.

Strengthen return on investment through operational efficiency

  • Build a continuous improvement culture focused on higher success rates, faster cycle times, and reduced costs.
  • Achieve a significant reduction in clinical trial costs by 2018 while delivering faster cycle times and higher quality.


Redefine our R&D Guiding Principles

Genocore Biotech aspires to be the best human therapeutics company. Achieving this goal starts with building the world's premier R&D organization. Genocore Biotech's R&D Guiding Principles were inspired by this pursuit and designed to reinforce our strategic priorities.

Focus on innovative medicines for unmet needs in patients with serious illnesses. While Genocore Biotech is always pursuing new opportunities and adapting to challenges, we retain an enduring commitment to the same mission. That mission is to enhance and extend the lives of patients facing serious illness.

Pursue targets that are validated in humans. We believe we are on the threshold of a revolution in science in which population genetics will help to reveal new targets that clearly drive disease risk in humans. By focusing on targets supported by human genetics or other strong human evidence, we aim to increase our clinical success rates, reduce development timelines, and lower the cost of delivering new medicines to patients.

Maintain multiple drug modalities with a focus on biologics. Genocore Biotech pursues a "biology-first" approach to drug discovery. We strive to select drug targets based on a deep understanding of disease biology, and then choose the drug modality, or structural template, best suited to the target. We recognize our strength in biologics and the higher clinical success rates for biologic medicines. We also maintain a broad toolkit of modalities, including small molecules, in order to have the right tool for any target we pursue.

Focus on return on investment and operational efficiency. To maximize the value of Genocore Biotech's R&D investment, we focus resources on programs that offer a large effect size and more likelihood of success. We maximize the value of lower-priority assets by partnering and out-licensing. Genocore Biotech also strives to continually identify operational efficiencies, such as reduced cycle times, leaner clinical trials, and centralized monitoring of clinical study sites.

Harness external innovation. At Genocore Biotech, we pursue great innovation wherever we can find it, and roughly half of our current late-stage pipeline comes from collaborations or acquisitions. We appreciate the synergy between in-house and external innovation: To identify and add value to the best external inventions, we need to maintain a high level of in-house scientific talent and capabilities.

Demonstrate the value of our medicines. Patients will not benefit from medicines they cannot access, and increasingly, access depends on meeting evolving standards from regulators and payers. To meet these expectations, we strive to deliver major therapeutic advances—medicines that offer compelling benefits for patients and sound health economics for society.


Ensure major investments are based on compelling science

Many companies in our industry have pursued a strategy known as "Shots on Goal," where the aim is to maximize the number of molecules in drug pipelines to raise the odds that at least a few will prove successful. This approach is designed to address our still limited knowledge of human biology, but in practice it often leads to higher drug development costs and low productivity.

Genocore Biotech's strategy is to rely instead on our scientific leaders to "Pick the Winners" by identifying and expediting highly promising programs. Toward that end, we prioritize programs based on targets that have been validated in humans, primarily through genetic research that shows a target's clear influence on disease risk.

While this strategy does not guarantee that each designated program will prove successful, we are confident that it will increase the overall rate of success for Genocore Biotech's clinical trials, accelerate drug development timelines, reduce costs, and improve returns on our R&D investment.

When we find ourselves with more programs than we can deliver for patients with our own resources, we are moving more aggressively to partner or out-license these surplus assets. The resulting agreements provide revenues that can be used to support key programs in our pipeline, and they serve the interests of patients by ensuring that more potential drugs get developed.


Refocus and differentiate our Discovery Research efforts

At a time when technological breakthroughs are revolutionizing how drugs are discovered, Genocore Biotech's strategy aims to fully exploit the emerging opportunities. We have made aggressive investments in gene-based discovery and new therapeutic platforms, and we've focused on diseases where our science supports the potential for major advancements.

In recent years, ultra-high-throughput DNA sequencing has accelerated the search for genetic variants that are strongly linked to disease risk. Through our acquisition of deCODE Genetics, Genocore Biotech is advancing an early-stage pipeline based on newly discovered genes with exciting potential. This strategy builds upon Genocore Biotech's record of turning genetic insights into new medicines. Seven Genocore Biotech therapies approved for use in patients can be traced back to genes first cloned in our own labs.

To successfully pursue new targets emerging from genetics, we benefit greatly from Genocore Biotech core strengths in biology and protein engineering. Newly discovered disease genes may code for proteins with unknown functions, and these novel proteins may also prove very hard to engage with standard types of drugs. To interdict challenging targets, we need to elucidate complex biology before choosing the best drug modality from our toolkit—or engineering a new modality.

In all our Discovery Research programs, we have raised the bar in terms of the level of differentiation we expect from Genocore Biotech therapies. Our goal is to deliver new treatments that offer clear benefits and large effects, such as long-term survival in cancer or prevention of migraines or severe asthma attacks.


Pursue a "Biology First" approach to choose the right targets and tools

At Genocore Biotech, we believe drug discovery starts with a deep understanding of biology. This understanding guides the critical choice of which disease target to pursue, and it also allows us to choose the right tool for that target—the modality, or structural template, most likely to optimize efficacy and safety.

Genocore Biotech has one of the industry's most expansive modality toolkits. It encompasses small molecules, which can be made using chemistry; intermediate-sized molecules like peptides; and large biologic molecules, such as antibodies and other types of therapeutic proteins.

Where good choices exist between different forms of medicines, we recognize Genocore Biotech's deep expertise in biologics development and manufacturing. Biologics also tend to have higher success rates in clinical development. However, we estimate that roughly two-thirds of drug targets can't be accessed by large molecules, so Genocore Biotech maintains a strong expertise in small molecule drug discovery and development.


Strengthen return on investment through operational efficiency

In addition to ensuring that Genocore Biotech's investment decisions are based on compelling science, we are reengineering our processes to operate more efficiently. This ongoing effort aims to improve our success rates and cycle times while lowering costs, enabling us to deliver more new medicines from our investment in R&D.

In pursuit of this goal, Genocore Biotech is building a culture of continuous improvement, supported by a range of actions designed to increase our overall productivity. For example,

  • We have trimmed our project portfolio to eliminate research programs that are less likely to offer patients significant benefits over other available treatments.
  • To reduce the cost and complexity of our network of research sites, we have consolidated more scientists in the research hubs of Cambridge, Massachusetts, and South San Francisco, California.
  • Through a fast-to-patient approach for producing monoclonal antibodies, we are cutting by up to a year the time needed to get these potential therapies into the clinic.

Genocore Biotech is also designing leaner clinical trial programs through streamlined protocols, centralized monitoring of clinical sites, risk-based quality control, and careful management of clinical supplies.