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Nonprofits: Innovators in Preclinical Drug Development

03/27/2019

Moving a therapeutic from the bench to the bedside is wrought with challenges, with a substantial bottleneck occurring during the translation of scientific discoveries into clinic-ready drugs. These difficulties are compounded by the high risk of this stage of development and resulting low investment. What is driving these challenges and who is best suited to support this part of the process?

In this webinar, the Center for Strategic Philanthropy and FasterCures team up to identify these challenges and explore how nonprofits are advantageously poised to tackle them. Through this session, we will highlight fresh and innovative models adopted by some venturous nonprofit organizations, and engage the participants on how to spark the development of new therapeutics for patients.

Speakers include:

  • Debra Miller, CEO and Founder, CureDuchenne 
  • Shobha Parthasarathi, Vice President, Strategic Alliances and Business Development, Harrington Discovery Institute
  • Carol Routledge, Director of Research, Alzheimer’s Research UK
  • Kirstie Keller, Senior Associate, Center for Strategic Philanthropy, a center of the Milken Institute (Moderator)  

Summary 

On March 27, two centers of the Milken Institute, the Center for Strategic Philanthropy and FasterCures, co-hosted a webinar entitled “Nonprofits: Innovators in Preclinical Drug Development.” The webinar featured guest speakers from the Harrington Discovery Institute, Alzheimer’s Research UK, and CureDuchenne to discuss the roles of nonprofits in translating scientific discoveries into life-saving drugs. 

Panelists touched on a number of critical issues, including:

How Nonprofits Can Help Overcome the “Valley of Death”

Kirstie Keller, Ph.D., Senior Associate at the Center for Strategic Philanthropy, kicked off the webinar by discussing the “valley of death”—an area of high risk and low investment that often occurs when translating academic discoveries to a clinic-ready therapeutic. The Center for Strategic Philanthropy uncovered six challenges to advancing candidates through the preclinical stage of drug development and identified six unique ways that nonprofits can use either alone or together to address these challenges as addressed in their report titled Nonprofits: A Growing Force for Drug Development. Their findings are based on their analysis of expert interviews from academia, nonprofits, venture capital, and industry.

Following Dr. Keller’s introduction, three nonprofit leaders shared their organization’s distinct roles in advancing the drug development process.

Collaboration and Funding to Drive Early Discoveries: Harrington Discovery Institute (HDI)

Shobha Parthasarathi, Ph.D., Harrington Discovery Institute’s (HDI) Vice President of Strategic Alliances and Business Development, shared HDI’s model for advancing discoveries to benefit society:

The organization offers financial, intellectual, and human capital to accelerate and de-risk early drug discovery and development through a disease- and modality-agnostic approach.
HDI has a network of advisors with previous experience in the pharmaceutical industry to help guide their approach and mentor their awardees.
Since 2012, HDI supported 110 drug discovery projects at 50 institutions, five of which are in clinical trials. Their involvement aided the foundation of 20 companies and another five projects have been licensed to large pharmaceutical companies.

Dropping Barriers Through Collaboration: Alzheimer’s Research UK

Carol Routledge, Ph.D., Director of Research at Alzheimer’s Research UK, discussed her organization’s three approaches to advancing drug discovery through the collaboration of nonprofits, academia, and industry with varied financing structures: the Dementia Consortium, Drug Discovery Alliance, and Dementia Discovery Fund.

The Dementia Consortium and Drug Discovery Alliance bring together researchers and experts from pharma to fund dementia-focused projects.
The Dementia Discovery Fund, a venture capital fund, supports the discovery and development of novel disease-modifying drugs for dementia.

When Advocacy is Personal: CureDuchenne

Co-Founder and CEO of CureDuchenne, Debra Miller, explained how her son’s diagnosis inspired her to start CureDuchenne—a patient advocacy organization with a mission to improve and extend the lives of those living with Duchenne muscular dystrophy.

CureDuchenne has raised over $30 million and has funded 29 research projects, leading to 16 clinical trials and the first FDA-approved drug for Duchenne.
The venture philanthropy arm of CureDuchenne, CureDuchenne Ventures has invested more than $1.3 billion in follow-on financing from venture capital, biotech, and pharmaceutical in promising companies.