GTPCH1

SHEFFIELD, United Kingdom, Jan. 8, 2025 /PRNewswire/ -- BlackfinBio Ltd (BFB) a clinical stage gene therapy company focused on the development of treatments for rare neurological diseases has announced today that it has acquired the Parkinson's patent portfolio from OXB. BFB intends to initially develop this technology as part of its BFB-201 program focused on dopamine deficiency diseases. Continue Reading BlackfinBio Ltd announces the acquisition of Parkinson's IP patent portfolio from OXB Post this Peter Nolan CEO of BFB said "We are delighted to take assignment of this patent portfolio which we look forward to developing as part of our BFB-201 program." Professor Mimoun Azzouz, Academic Founder of BFB said "We are excited to attract this assignment to the BFB pipeline, offering a fantastic opportunity to apply a single product across a wide range of dopamine deficiency diseases." About Blackfin Bio Ltd BlackfinBio is a clinical gene therapy company focused on the development of treatments for rare neurological diseases. The pipeline comprises BFB-101, an IND stage AP4B1 replacement AAV gene therapy for Hereditary Spastic Paraplegia, sub-type 47 - an ultra-rare genetic neurological disease for which no treatment currently exists. BFB-201 is a preclinical stage gene therapy approach using a three gene fusion (AADC-TH-CH) to treat several dopamine deficiency disorders. For more information, please visit: For media enquiries, please contact: Peter Nolan, Chief Executive Officer [email protected] SOURCE BlackfinBio WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

A new study points to possible new treatments for chronic pain with a surprising link to lung cancer. Findings of the research, conducted in laboratory mouse models, open up multiple therapeutic opportunities that could allow the world to improve chronic pain management and eclipse the opioid epidemic. Pain is an important alarm system that alerts us to tissue damage and prompts us to withdraw from harmful situations. Pain is expected to subside as injuries heal, but many patients experience persistent pain long after recovery. Now, a new study published in Science Translational Medicine points to possible new treatments for chronic pain with a surprising link to lung cancer. The work was spearheaded by an international team of researchers at IMBA -- Institute of Molecular Biotechnology of the Austrian Academy of Sciences, Harvard Medical School, and Boston Children's Hospital. Their findings of the research, conducted in laboratory mouse models, open up multiple therapeutic opportunities that could allow the world to improve chronic pain management and eclipse the opioid epidemic. Acute pain is an important danger signal. By contrast, chronic pain is based on persistent injury and can even be experienced in the absence of a stimulus, injury, or disease. Despite the hundreds of millions of people affected, chronic pain is among the least well-managed areas of healthcare. To improve how persistent pain is managed and considering the raging opioid crisis, it is paramount to develop novel drugs based on a fundamental understanding of the underlying mechanisms. "We had previously shown that sensory neurons produce a specific metabolite, BH4, which then drives chronic pain, such as neuropathic pain or inflammatory pain," says project lead and co-corresponding author Shane Cronin, a staff scientist in the Penninger lab at IMBA and a former postdoc in the Woolf lab at Harvard Medical School and F.M. Kirby Neurobiology Center, Boston Children's Hospital. "The concentrations of BH4 correlated very well with the pain intensity. So, we naturally thought that this was a great pathway to target." To identify drugs that reduce BH4 levels in pain neurons, the researchers performed a "phenotypic screen" of 1000 target-annotated, FDA-approved medications. This approach allowed the scientists to start their search using medications that are currently in use for various indications, and to identify undescribed, off-target analgesic properties. Among the first findings of this hypothesis-driven search, the team was able to link the previously observed analgesic effects of several drugs, including clonidine and capsaicin, to the BH4 pathway. "However, our phenotypic screen also allowed us to 'repurpose' a surprising drug," says Cronin. The drug 'fluphenazine', an antipsychotic, has been used to treat schizophrenia. "We found that fluphenazine blocks the BH4 pathway in injured nerves. We also demonstrated its effects in chronic pain following nerve injury in vivo." The researchers also found that the effective analgesic dose of fluphenazine in their experiments in the mouse model is comparable to the low end of the doses safely indicated for schizophrenia in humans. In addition, the screen uncovered a novel and unexpected molecular link between the BH4 pathway and EGFR/KRAS signaling, a pathway involved in multiple cancers. Blocking EGFR/KRAS signaling reduced pain sensitivity by decreasing the levels of BH4. The genes of EGFR and KRAS are the two most frequently mutated genes in lung cancer, which prompted the researchers to look at BH4 in lung cancer. Surprisingly, by deleting an important enzyme, GCH1, in the BH4 pathway, the mouse models of KRAS-driven lung cancer developed fewer tumors and survived much longer. Hence, the researchers uncovered a common signaling pathway for chronic pain and lung cancer through EGFR/KRAS and BH4, thus opening up new avenues of treatment for both conditions. "Chronic pain is currently subjected to often ineffective palliative treatments. Furthermore, effective painkillers such as opioids can lead, if used inappropriately, to severe addiction. It is therefore critical to find and develop new and repurposed drugs to treat chronic pain," says co-corresponding author Clifford Woolf, professor of neurology and neurobiology at Harvard Medical School and director of the F.M. Kirby Neurobiology Center at Boston Children's Hospital. One intriguing aspect of the study is the mechanistic link between pain and lung cancer. "The same triggers that drive tumor growth appear to be also involved in setting the path to chronic pain, often experienced by cancer patients. We also know that sensory nerves can drive cancer, which could explain the vicious circuit of cancer and pain," adds co-corresponding author Josef Penninger, IMBA group leader and founding director, who is currently also the director of the Life Sciences Institute at the University of British Columbia (UBC), Vancouver, Canada. "Understanding these cross-talks is therefore not only critical for cancer treatments but might also help to improve the quality of life for cancer patients towards less pain."

October 23, 2014 By Riley McDermid , BioSpace.com Breaking News Staff Up and coming biotech Quartet Medicine today announced a Series A funding round of $17 million from financing by Atlas Venture with Novartis Venture Funds , Pfizer Venture Investment and Partners Innovation Fund . As part of the deal, Henry Skinner from Novartis Venture Funds will now join Quartet ’s board of directors. Cambridge, Mass.-based Quartet said it plans to use the funding to advance its lead program targeting the modulation of tetrahydrobiopterin (BH4) synthesis by taking its research to human subjects. “Human genetics and clinical biomarker data implicate a role for BH4 pathway up-regulation in neuronal excitability and on immune cell activity after nerve injury,” said Quartet co-founder Clifford Woolf in a statement. “New treatments that safely restore BH4 levels at the nexus of the peripheral nervous system and the immune system have potential to address the tremendous unmet need faced by patients with chronic pain.” Quartet was co-founded in late 2013 by Kevin Pojasek , who was then an entrepreneur-in-residence at Atlas Venture along Woolf, who had previously held positions at Harvard Medical School and Boston Children’s Hospital , and Kai Johnsson , of École Polytechnique Fédérale de Lausanne ( EPFL ). The three agreed to launch a biotech startup in order to further explore the therapies provided by understanding BH4’s role in chronic pain and inflammation. Quartet ’s primary research focuses on how to regulate BH4 synthesis. Injury or inflammation increases cellular levels of BH4, an important co-factor for several classes of enzymes, resulting in neuronal hypersensitivity and chronic immune cell activation. Thus far, multiple human genetic studies have shown a link between a haplotype in the gene encoding GTP cyclohydrolase I, a BH4 synthetic enzyme, and a reduced risk of developing chronic pain after nerve injury or chronic disease. Quarter hopes to develop a drug that will modify either GTP cyclohydrolase I or sepiapterin reductase, another enzyme in the BH4 synthesis pathway, in an effort to restore BH4 to normal levels leading to an improvement of pain symptoms in preclinical models. The venture capitalist involved said they were proud to be getting in on the ground floor of such promising research. “ Atlas Venture is proud to be leading this strong syndicate of investors after providing seed capital to support the formation of Quartet and to achieve early target validation,” said Bruce Booth , chairman of Quartet and partner at Atlas Venture . “We are capitalizing on recent advances in human genetics that are revealing new mechanisms and, ultimately, novel targets for drug discovery and development in the challenging and underserved therapeutic area of chronic pain.” var switchTo5x=true; stLight.options({publisher: "0341611e-38a4-415d-9e18-75e093ff27e0", doNotHash: false, doNotCopy: false, hashAddressBar: false});