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Gene therapy has emerged as the most popular approach employed by academic institutes, biotechs, and NGOs in the treatment of rare diseases, according to the latest findings in Inpart’s rare disease Global Challenge report.
Inpart’s Global Challenge campaigns present an opportunity for academic researchers and professionals to have their projects, technologies or breakthroughs relating to a current global health or sustainability challenge proactively disseminated to industry, with the aim of starting new conversations with the help of Inpart’s partnering platform, Connect.
The results of the latest campaign demonstrated the increase in therapeutic strategies to treat rare diseases, with small molecule orphan drug discovery, and protein or other biologics following closely behind on the list of focus areas for research collaborations. Among these types of innovations, gene therapy makes up almost a quarter (24%) of total interactions from companies who engaged with the campaign, either by viewing the technology or requesting an introduction to the scientists behind it.
“These results support what we know about the continued emergence of new therapeutic approaches to treat rare diseases. Therapeutic development and gene therapy are at the frontier of rare disease R&D because they hold the key to unlocking the potential of personalised medicine. Most rare diseases result from genetic mutations, and gene therapy offers a promising avenue to not only alleviate the symptoms of those affected, but also address the root causes,” says Dr Robin Knight, Global Head of Product, Inpart.
Of the specific rare diseases submissions that received the most interest from industry R&D, ‘DNA-Guided Gene Editing Tools’, a technology developed by scientists at Purdue University, and ‘AAV2/9-Mediated Gene Therapy for SPLIS Treatment’ from the University of California, San Francisco, were the most viewed technologies in this campaign (5% of total reads each). While the most popular approach to rare disease treatment amongst industry was gene therapy, proteins or other biologics (18%) and small molecule orphan drug discovery (17%) were also prominent approaches on the interactions list.
These innovations can also be found on the R&D priority list of the campaign partners featured in the report. Sanofi, General Inception, Mitsubishi Tanabe Pharma, and Ultragenyx provide their interests to help paint a more detailed picture of R&D in the rare disease space.
“It’s great to see broad participation from academic institutions worldwide who are looking to make connections with rare disease industry partners to drive future developments. Product development in the rare disease space, such as in the very promising field of gene therapy, will ultimately create new opportunities to develop therapies with transformative potential, addressing unmet needs for patients living with rare diseases,” says Dr Chris Hamblett, Vice President, Business Development at Ultragenyx.
“Out of the 181 new conversations our teams at Inpart started via our campaign on rare diseases, 37 are between teams in academia and industry exploring new gene therapy innovations. We hope that these new connections will accelerate groundbreaking treatments that address key R&D challenges in the field,” concludes Dr Knight.
Download the full report for all the key stats and findings.