Leukodystrophy, Globoid Cell

YONGIN, South Korea I November 25, 2024 I GC Biopharma and Novel Pharma today announced that the first patient in the US has been dosed in multinational clinical trial with ‘GC1130A’, an innovative new drug for Sanfillippo syndrome type A (MPS IIIA). GC Biopharma and Novel Pharma have received IND clearance for Phase I clinical trial of ‘GC1130A’ in the US, Korea, and Japan and are currently conducting multi-national clinical trial. Through this Phase I clinical trial, they plan to evaluate the safety, tolerability, and efficacy of ‘GC1130A’ over a two-year period for children aged between two and six diagnosed with Sanfillippo syndrome type A (MPS IIIA). This Phase I clinical trial will be conducted at two to three institutions in the US including University of California San Francisco (UCSF) Benioff Children’s Hospitals, Samsung Medical Center and Ajou University Hospital in Korea, and one institution in Japan. Patients who meet eligibility criteria after screening will undergo surgery to implant an intracerebroventricular (ICV) access device and will receive ‘GC1130A’ once every two weeks. Sanfillippo syndrome type A (MPS IIIA) is an autosomal recessive genetic disorder that damages the central nervous system due to the accumulation of heparan sulfate. The main clinical manifestation is brain deficit, and it is a devastating disease where patients die around the age of 15. As no treatment option currently exists, there is a huge unmet medical need. ‘GC1130A’ is a first-in-class treatment that uses GC Biopharma’s proprietary platform to produce concentrated, high quality recombinant protein which is administered via ICV injection to bypass the blood brain barrier. GC Biopharma developed the first ICV administered enzyme replacement therapy (ERT) for patients with Hunter syndrome and marketed in Japan under the brand name Hunterase ICV. In a non-clinical study, GC Biopharma has proven that ICV injection is up to 47 times more effective than that of intrathecal (IT). SooKyung Shin, Head of Medical Division at GC Biopharma commented, “The initiation of patient dosing in the US is a significant milestone for ‘GC1130A’ global clinical trials and we will do our best to expedite the clinical development process.” About GC Biopharma Corp. GC Biopharma Corp. (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma Corp. is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century. About Novel Pharma Inc. Novel Pharma is an emerging biotechnology company that focuses on developing first-in-class treatments for rare pediatric diseases (MPS/LSD) utilizing ICV (intracerebral ventricular) administration.  Headquartered in Seoul, South Korea, it is currently engaged in the development of (i) MPSIIIA, (ii) GM1, (iii) MPSIVB and (iv) Krabbe disease. SOURCE: GC Biopharma

YONGIN, South Korea, Nov. 25, 2024 /PRNewswire/ -- GC Biopharma and Novel Pharma today announced that the first patient in the US has been dosed in multinational clinical trial with 'GC1130A', an innovative new drug for Sanfillippo syndrome type A (MPS IIIA). GC Biopharma and Novel Pharma have received IND clearance for Phase I clinical trial of 'GC1130A' in the US, Korea, and Japan and are currently conducting multi-national clinical trial. Through this Phase I clinical trial, they plan to evaluate the safety, tolerability, and efficacy of 'GC1130A' over a two-year period for children aged between two and six diagnosed with Sanfillippo syndrome type A (MPS IIIA). This Phase I clinical trial will be conducted at two to three institutions in the US including University of California San Francisco (UCSF) Benioff Children's Hospitals, Samsung Medical Center and Ajou University Hospital in Korea, and one institution in Japan. Patients who meet eligibility criteria after screening will undergo surgery to implant an intracerebroventricular (ICV) access device and will receive 'GC1130A' once every two weeks. Sanfillippo syndrome type A (MPS IIIA) is an autosomal recessive genetic disorder that damages the central nervous system due to the accumulation of heparan sulfate. The main clinical manifestation is brain deficit, and it is a devastating disease where patients die around the age of 15. As no treatment option currently exists, there is a huge unmet medical need. 'GC1130A' is a first-in-class treatment that uses GC Biopharma's proprietary platform to produce concentrated, high quality recombinant protein which is administered via ICV injection to bypass the blood brain barrier. GC Biopharma developed the first ICV administered enzyme replacement therapy (ERT) for patients with Hunter syndrome and marketed in Japan under the brand name Hunterase ICV. In a non-clinical study, GC Biopharma has proven that ICV injection is up to 47 times more effective than that of intrathecal (IT). SooKyung Shin, Head of Medical Division at GC Biopharma commented, "The initiation of patient dosing in the US is a significant milestone for 'GC1130A' global clinical trials and we will do our best to expedite the clinical development process." About GC Biopharma Corp. GC Biopharma Corp. (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma Corp. is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century. About Novel Pharma Inc. Novel Pharma is an emerging biotechnology company that focuses on developing first-in-class treatments for rare pediatric diseases (MPS/LSD) utilizing ICV (intracerebral ventricular) administration. Headquartered in Seoul, South Korea, it is currently engaged in the development of (i) MPSIIIA, (ii) GM1, (iii) MPSIVB and (iv) Krabbe disease. This press release may contain biopharmaceuticals in forward-looking statements, which express the current beliefs and expectations of GC Biopharma's management. Such statements do not represent any guarantee by GC Biopharma or its management of future performance and involve known and unknown risks, uncertainties and other factors. GC Biopharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. SOURCE GC Biopharma WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Unique international partnership marks significant momentum for new gene therapy entity PHILADELPHIA, Oct. 8, 2024 /PRNewswire/ -- Today, GEMMA Biotherapeutics ("GEMMABio") – a new therapeutics company founded by gene therapy pioneer Dr. Jim Wilson – announced a major agreement with the Oswaldo Cruz Foundation ("Fiocruz"), a public health research institution that is part of the Ministry of Health of Brazil and has extensive experience in biomanufacturing. GEMMABio partnered with Fiocruz – which is the main supplier of immunobiologics, biopharmaceuticals and diagnostics to the Brazilian Ministry of Health – to sponsor research that will bring gene therapy treatments to the people of Brazil under the Sistema Único de Saúde (SUS), Brazil's publicly funded health care system. Fiocruz will fund up to $100 million, which will be used to conduct clinical research and manufacturing – ultimately securing approval for programs that will allow the country to increase distribution of rare disease treatments and at a more affordable cost. "By combining our resources and knowledge, this collaboration represents a major advancement in making gene therapies accessible to more patients quickly and at a fraction of the typical cost," said Dr. Wilson, who will serve as President and CEO of GEMMABio. "We are optimistic that this deal will serve as a global example for expanding access to advanced medical therapies as well as pursuing non-traditional ways to capitalize biotech companies." A key component of the partnership will involve tech transfer, replicating GEMMABio's manufacturing technologies and process so that the companies can collaborate on programs and production in Brazil and for Fiocruz to utilize with other sponsors and programs. The agreement will also fund clinical development activities in Brazil for six core programs, including GM1 gangliosidosis (GM1), Krabbe Disease, Metachromatic leukodystrophy (MLD), and three unnamed programs. In return for their financial support, Fiocruz will be able to license related treatments to supply the Brazilian public health system. "Brazil has the largest public health system in the world, offering health services and products to more than 200 million people. We are very proud to bring this innovation to the Brazilian system, a technology that was previously restricted to those with higher incomes, will now be accessible to the entire Brazilian population" said Marco Krieger, Vice President of Production and Innovation in Health, Fiocruz. About GEMMABio GEMMABio is a therapeutics company focused on speeding the research of and global access to life-changing advanced therapies for those living with rare diseases. The company will provide research and product development functions to bring gene therapy discoveries from the bench to the bedside faster and affordably. GEMMABio is led by gene therapy industry pioneer Jim Wilson and his team of experts, who previously conducted their work in academia. Wilson is also the Chairperson of Franklin Biolabs, a Contract Research Organization. The collective goal of the two affiliated companies is to translate innovative scientific work into clinical trials, to then commercialize and distribute the new therapies around the world to patients who need them most. For more information, please visit gemmabiotx.com. About Fiocruz The Oswaldo Cruz Foundation (Fiocruz) is the largest biomedical research institution in Latin America, which also produces vaccines, medicines and in vitro diagnostics (IVD) to supply the Unified Health System (SUS, acronym in Portuguese for the Brazilian public health system). Linked to the Brazilian Ministry of Health, the Foundation was created on May 25, 1900 to initially manufacture serums and vaccines against bubonic plague. Since then, the institution has experienced an intense trajectory, which is intertwined with the development of public health in Brazil. Currently, Fiocruz is installed in 10 states, in addition to the Federal District, and has an office in Maputo, capital of Mozambique, Africa. In addition to the institutes based in Rio de Janeiro, the Foundation maintains units in the Northeast, North, Southeast and South regions of Brazil, and offices in Ceará, Mato Grosso do Sul, Piauí and Rondônia. In total, there are 16 technical-scientific units, focused on teaching, research, innovation, assistance, technological development and extension in the field of health. For more information, visit . Contact: Scott Hoeflich [email protected] SOURCE GEMMA Biotherapeutics and Franklin Biolabs WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED