BETHESDA, Md. and BALTIMORE, Nov. 30, 2020 (GLOBE NEWSWIRE) — Gain Therapeutics, Inc. (“Gain”), today announced a research collaboration with the University of Maryland School of Medicine (UMSOM), to investigate Gain’s structurally targeted allosteric regulators (STARs) in cellular models of neuronopathic Gaucher disease (nGD) and Parkinson’s disease (PD). STARs are proprietary small molecules targeting novel allosteric binding sites on enzymes. These small molecule drug candidates are designed to cross the blood brain barrier and penetrate other hard to treat organs such as bone and cartilage, stabilize the effective enzyme to restore function and reduce toxic substrate. Research will be led by Ricardo A. Feldman, Ph.D., Associate Professor, of Microbiology and Immunology in UMSOM.
Under the terms of the collaboration, UMSOM will investigate Gain’s STAR candidates in macrophage and neuronal models of nGD and GBA-associated PD. These diseases are characterized by mutations in the GBA gene, where misfolding of the enzyme encoded by GBA (beta-glucocerebrosidase (GCase)) interferes with its normal transport to the lysosome. The research program will aim to further elucidate the mechanism of action of Gain’s STAR candidates by studying their effect on GCase, including GCase’s enzyme activity and transport to the lysosome. Additionally, other effects such as prevention of alpha-synuclein aggregation in PD dopaminergic neurons will be evaluated.
“We are exceedingly proud to be advancing our work in nGD and Parkinson’s in close collaboration with the University of Maryland School of Medicine,” said Eric Richman, Chief Executive Officer at Gain. “The expertise and experience of UMSOM and Dr. Feldman will be instrumental as we work to further validate the exciting potential of Gain’s STAR candidate for these devastating diseases. I am confident these foundational studies will bring us closer to a potential new treatment option for those with these disorders.”
Dr. Feldman added, “Our laboratory has used human induced pluripotent stem cell (iPSC) models of GD and GBA-associated PD to uncover the molecular mechanisms leading to these diseases. We have also developed very sensitive assays to evaluate the therapeutic efficacy of small molecules in reversing the phenotypic abnormalities caused by mutant GBA in the cell types affected by these diseases, including macrophages and neuronal cells. I have been impressed by Gain’s initial results evaluating the potential of STARs in correcting enzyme misfolding and restoring function, and look forward to working with Gain’s team to further advance its program to treat these diseases.”
Gain and UMSOM intend to report initial data from the collaboration in the first half of 2021.
About Gain Therapeutics, Inc.
Gain Therapeutics is redefining drug discovery with its SEE-Tx™ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was originally established in 2017 with the support of its founders and institutional investors such as TiVenture, 3B Future Health Fund (previously known as Helsinn Investment Fund) and VitaTech. It has been awarded funding support from The Michael J. Fox Foundation for
FDA opens private Covid vaccine meetings to the public in bid to gain trust as Trump pressures for fast approval
The FDA took the unusual step Thursday in opening to the public a routine meeting with an advisory group that’s weighing in on approving the coronavirus vaccine as the agency battles public concerns about its safety as well as political pressure from President Donald Trump to approve it before the Nov. 3 election.
The Vaccines and Related Biological Products Advisory Committee, an outside group of researchers and physicians who are advising the Food and Drug Administration on whether to approve a Covid-19 vaccine, debated the standards needed to ensure a Covid-19 vaccine is safe and effective in a meeting broadcast on YouTube and C-SPAN. Those are key questions among medical experts who worry the U.S. will approve a vaccine before it has been adequately tested.
Officials at the meeting Thursday said the public forum was “critical” to build public trust and confidence in the development of potential vaccines, which are being developed in record time. FDA officials promised that any vaccine would undergo rigorous testing before being distributed to the public.
“Vaccine development can be expedited. However, I want to stress that it cannot, and must not, be rushed,” said Dr. Marion Gruber, director of FDA’s Office of Vaccines Research and Review, adding the agency would not lower its standards.
Trump has pushed the FDA to approve a drug in time to distribute by the Nov. 3 election — a daunting task even his closest advisors have said is near impossible.
“I think we should have it before the election, but frankly the politics gets involved and that’s okay. They want to play their games, it’s going to be right after the election,” Trump said in a video he posted on Twitter on Oct. 7. “The FDA has acted as quickly as they’ve ever acted in history. There’s never been a time, and no president’s ever pushed them like I’ve pushed them either, to be honest.”
The agency is approving drugs “in a matter of weeks” that used to take years, he added.
Video: Dr. Patel on how the White House should be dealing with the virus outbreak: ‘This should go well beyond what’s standard’ (MSNBC)
Four drugmakers backed by the U.S. are still conducting their late-stage trials, and medical experts don’t expect to see trial data needed for FDA authorization until later this month at the earliest.
Because of the pandemic, U.S. health officials and researchers have been accelerating the development of vaccine candidates by investing in multiple stages of research even though doing so could be for naught if the vaccine ends up not being effective or safe.
The FDA, under pressure from the White House, has faced skepticism from medical experts that the vaccine approval