A case study on partnering between patient advocacy organizations and biotech

June 23, 2022

Last week's 2022 Biotechnology Innovation Organization (BIO) International Convention in San Diego gave patient advocacy organizations and biotech companies the first opportunity in three years to meet in person with the potential for partnering. However, it was a meeting at BIO 2019 that could serve as a model for such partnerships.

Susan Ruediger, founder and chief mission officer of the CMT Research Foundation (CMTRF), recounted for ScienceBoard.net that fateful meeting three years ago between biotech company DTx Pharma and her nonprofit. Their discussion focused on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease, a hereditary disorder of the peripheral nervous system.

CMTRF's scientific advisory board early on identified drug delivery to the peripheral nervous system as a key barrier in CMT research. With that as a research priority, the patient advocacy organization leveraged the one-on-one partnering system at BIO 2019 to set up meetings with companies working in drug delivery. That led to a meeting with DTx Pharma.

"It took a few months for our advisors and their scientific team to get together, and we designed a program that we then, at the research foundation, funded. It was $125,000 and it was really a concept: will their technology get into the peripheral nervous system?" Ruediger said.

With funding from CMTRF, DTx Pharma designed and tested a library of novel RNA-based compounds called small interfering RNA (siRNA) which target PMP22, the gene that when overexpressed causes CMT1A. Six months later, DTx Pharma had enough data to secure a Small Business Innovation Research grant from the National Institutes of Health and the biotech was soon after able to raise $100 million to take their pipeline of therapeutic candidates, including for CMT1A, forward toward clinical trials, according to Ruediger.

She credited BIO with making the finding of partners such as DTx Pharma "in such a targeted way so easy." Ruediger said the company is now preparing for clinical trials and "the data continues to grow and expand."

Watch the video below to learn more.


NIH seeks industry partners for technology transfer
The National Institutes of Health (NIH) is the world’s largest public funder of biomedical research. ScienceBoard.net spoke with Michael Salgaller...
Estonia’s ambitious plans to grow its biotech industry
The Baltic state of Estonia's biotech industry is expected to grow tenfold this decade and offers significant opportunities for Estonian companies. ScienceBoard.net...
Using the power of plants to develop novel biopharmaceuticals
Biologics manufacturing company iBio is using plants as bioreactors to develop and manufacture therapeutics and vaccines. ScienceBoard.net spoke...
Fauna Bio leverages animal genomics, hibernation biology to target human diseases
Biotech company Fauna Bio is leveraging animal genomics and hibernation biology to develop novel translational therapeutics for disease prevention and...
Overcoming the challenges of off-the-shelf allogeneic cell therapies
Chimeric antigen receptor T-cell therapy has emerged as one of the major breakthroughs in cancer immunotherapy in the last decade. In a video interview...
Apeiron Biologics' spinout invIOs targets solid tumors with novel cell therapy platform
Apeiron Biologics' spinout invIOs has a proprietary cell therapy platform for the modulation of intracellular immuno-oncology targets that enables rapid...
Struggle to include women in clinical trials continues despite gains
Though female participation in clinical trials has been steadily improving over the years, most of the women enrolled in trials are white and not members...
Patient advocacy organizations look to partner with pharma companies
Patient advocacy organizations are looking to partner with pharmaceutical companies, offering resources and expertise to maximize the impact and efficiency...
How to develop a sustainable portfolio for cell, gene therapy platforms
Michael Retterath, a Biotechnology Innovation Organization International Convention 2022 panelist and chief business officer at Spark Therapeutics, spoke...
Biotech industry execs, not scientists, remain male-dominated
A panel at the 2022 Biotechnology Innovation Organization International Convention will explore how to close the gender gap in the biotech industry and...
The ‘voice of the patient’ needs to be heard in clinical trials: study
Patient recruitment and retention are among the biggest challenges facing clinical research, particularly in studies targeting rare diseases that have...
New era of nonviral DNA-delivered gene therapy seen emerging
Advancements in nucleic acid-encoded therapeutics over the past decade have sparked the emergence of a new era in nonviral DNA-delivered gene therapy,...
Biotech innovator recounts decades-long journey to create drug discovery technology
Dr. Stan Crooke, PhD, founder, chairman, and chief executive officer of RNA therapy company Ionis Pharmaceuticals, pioneered development of the antisense...
Biotech industry to gather at BIO International Convention in San Diego
More than 11,000 attendees from around the globe will be in San Diego, June 13-16, for the 2022 Biotechnology Innovation Organization (BIO) International...

Copyright © 2022 scienceboard.net

Last Updated 7/20/2022 2:28:57 PM