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Increasing The Value Of Academic Discoveries Through First-in-human Translation
Sashank Reddy, MD, PhD.
Johns Hopkins University, Baltimore, MD, USA.

PURPOSE: Academic medical centers (AMCs) have long been at the forefront of basic biomedical discovery. Increasingly they are presented with opportunities to translate those discoveries into new therapeutics. Examples such as Children’s Hospital of Philadelphia’s development of the first FDA-approved gene therapy - licensed to Spark Therapeutics and later sold to Roche for $4.3B - demonstrate how academic research can facilitate important new therapies and create significant economic value for sponsoring institutions. Yet, meaningful advances from target discovery to preclinical and clinical translation are rare in AMCs, leaving much of this value and impact unrealized. We sought to identify roadblocks to effective therapeutic translation and discover methods for improvement.
METHODS: Our methodology centered on four elements. First, we assessed faculty engagement by conducting interviews with 40 researchers engaged in therapeutic translation across our institution. We also culled the last 5 years of technology disclosures to the Johns Hopkins Technology Transfer office to identify therapeutically translatable discoveries. Second, we examined infrastructure across the institution that supports therapeutic translation including core facilities and advisory services. Third, we visited and interviewed leaders at Children’s Hospital of Philadelphia, University of Pennsylvania, and Harvard Medical School who have made major commitments to therapeutic translation. Finally, we spoke to leaders in the pharmaceutical, biotechnology, and venture capital industries to better understand how professionals approach drug development and academic partnership.
RESULTS: More than 500 therapeutically-relevant discoveries were disclosed to the Technology Transfer office since 2015, confirming a broad appetite among faculty for therapeutic translation. The University received nearly $200M in licensing revenue over that period. Yet, relatively few of the 500 discoveries resulted in significant deals, with the majority deemed too early for partnership. The value of discoveries increases substantially as they advance into preclinical and early clinical development (Figure 1) suggesting that AMCs should invest in therapeutic translation to better create and capture that value. Current impediments to doing so include lack of education and faculty guidance, needed improvements in policy and infrastructure, and financial constraints under current granting mechanisms. We identified a dozen steps AMCs can take to overcome these impediments, falling into three broad categories of investment: (1) leadership and governance, (2) therapeutics infrastructure, and (3) translational financing.
CONCLUSION: The time is propitious to advance basic discoveries into preclinical and clinical development at AMCs. The emergence of new therapeutic modalities that more directly link basic discovery and therapeutic intervention, the democratization of drug development by contract research organizations, and earlier alliance seeking by pharmaceutical and biotechnology partners combine to lower barriers to entry. Yet, significant, generalizable challenges remain, requiring investments in the three areas identified above. Success in turning basic discoveries into novel therapeutics at AMCs can bridge their research and patient care missions, redounding to the benefit patients, faculty, and the institutions.


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