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CAMBRIDGE, Mass., Jan. 26, 2023 /PRNewswire/ -- HBM Alpha Therapeutics (HBMAT), Inc., an innovative biotechnology company incubated by Harbour BioMed (HKEX: 02142), announced that it completed seed financing to advance its leading programs, novel antibody therapies to treat congenital adrenal hyperplasia (CAH) and polycystic ovary syndrome (PCOS), with the lead candidate currently in IND-enabling stage. HBMAT was founded on the expertise of Harbour BioMed's antibody discovery and development capabilities, and expertise in the molecular pathogenesis and treatment of endocrine disorders of HBMAT's Scientific Founder Dr. Joseph Majzoub, Professor of Pediatrics and Medicine at Boston Children's Hospital. HBMAT has licensed relevant technologies from Boston Children's Hospital. Dr. Majzoub chairs the Scientific Advisory Board for HBMAT and is a shareholder of the company. At present, HBMAT's Scientific Advisory Board comprises Dr. Majzoub, Dr. Frank Grosveld, Co-founder and CSO of Harbour Antibodies and the inventor of Harbour Mice®, and Dr. Ieuan Hughes, a paediatric endocrinologist and an emeritus professor of paediatrics at the University of Cambridge. HBMAT's programs aim to deliver precision therapies for endocrine disorders. The leading programs target a pathway for CAH and PCOS. The financing will be used to advance these programs into the clinical stage. CAH is a rare-autosomal recessive genetic disease with a defective CYP21A2 gene. CAH patients have life-threatening deficiencies of glucocorticoid and mineralocorticoid, with limited treatment options and no new therapeutic modalities in over 50 years. While the current standard of care is associated with inevitable treatment-related side effects, specifically Cushing Syndrome and hyperandrogenism, HBMAT is developing HAT001 which focuses on treating hyperandrogenism without causing Cushing Syndrome. HAT001 would effectively cause reversible pharmacologic adrenalectomy, converting CAH patient treatment into that for primary adrenal insufficiency, which can be easily treated with much lower, physiological doses of glucocorticoid. As a common and chronic endocrine disorder, PCOS affects over 10% women of reproductive age around the world and is the most common cause of anovulatory female infertility. HBMAT is also developing HAT002 for PCOS, targeting a pathway shared with CAH. "I am very excited about this unique opportunity of developing antibody therapeutics for CAH, a rare genetic disease and PCOS impacting women's health," said Jingsong Wang, Founder, Chairman & CEO of Harbour BioMed, and Chairman of BOD of HBMAT. "The company is thrilled to take these exciting programs to the next level and translate this therapeutic antibody with very impressive biological functions into clinical benefits for patients with significant unmet medical needs." "I've worked closely with patient groups and tried to help those suffering from rare diseases over the past decades. HBMAT has already identified a clinical development candidate with excellent characteristics in preclinical functional activities including in vivo efficacy in various relevant animal models. I look forward to moving the development candidate into clinical trials and beyond." said Dr. Philip Reilly, a renowned clinical geneticist and biotech entrepreneur, and board member of HBMAT. About congenital adrenal hyperplasia (CAH) CAH is a group of autosomal recessive diseases characterized by cortisol deficiency and androgen excess. It is one of the most common autosomal recessive disorders in humans. Current standard of care treatment of CAH patients with supraphysiologic glucocorticoids leads to Cushing syndrome, a collection of signs and symptoms due to prolonged exposure to glucocorticoids. The CAH market had sales of $46 million in 2016, with new therapeutics is expected to generate around $5 billion in 2035. About polycystic ovary syndrome (PCOS) PCOS is one of the most common endocrine and metabolic disorders in premenopausal women. PCOS is frequently associated with abdominal adiposity, virilization, insulin resistance, obesity, metabolic disorders and cardiovascular risk factors. The economic burden of PCOS has been estimated at approximately $3.7 billion annually in 2020 when considering only the costs of the initial diagnosis and of reproductive endocrine morbidities, without considering the costs of pregnancy-related and long-term morbidities. About Harbour BioMed Harbour BioMed (HKEX: 02142) is a global biopharmaceutical company committed to the discovery, development, and commercialization of novel antibody therapeutics focusing on immunology and oncology. Harbour BioMed is building its robust portfolio and differentiated pipeline through internal R&D capability, collaborations with co-discovery and co-development partners, and select acquisitions. Harbour BioMed's proprietary antibody technology platforms Harbour Mice® generate fully human monoclonal antibodies in two heavy and two light chain (H2L2) format, as well as heavy chain only (HCAb) format. Building upon the HCAb antibodies, the HCAb-based immune cell engagers (HBICE®) can deliver tumor-killing effects unachievable by traditional combination therapies. Integrating Harbour Mice®, HBICE® with single B cell cloning platform, the antibody discovery engine is unique and efficient for the development of next-generation therapeutic antibodies. For more information, please visit www.harbourbiomed.com. About HBM Alpha Therapeutics HBM Alpha Therapeutics is a company presently developing antibody therapeutics aimed at the rare genetic disease congenital adrenal hyperplasia (CAH) and polycystic ovary syndrome (PCOS). Its therapeutics could also be indicated for other endocrinological diseases related to the dysregulation and function of the hypothalamus-pituitary-adrenal (HPA) axis. HBMAT was founded and incorporated in Delaware, USA in 2019. It is a joint venture between Harbour BioMed and Boston Children's Hospital, an affiliate of Harvard Medical School. For more information, please visit www.hbmalphatx.com or contact BD@harbourbiomed.com. Leadership & Advisory TeamJingsong Wang, M.D., Ph.D., Chairman of BODFounder, Chairman & CEO of Harbour BioMedFormer Head of China R&D & Head of Translational Medicine, Asia Pacific, Sanofi Joseph Majzoub, M.D., Scientific FounderProfessor of Pediatrics, Professor of Medicine, Harvard Medical SchoolChief Emeritus, Division of Endocrinology, Vice Chair for Research, Department of Pediatrics, Boston Children's Hospital Philip Reilly, M.D., J.D., Board MemberAdvisory Board to Boston University School of Public HealthFormer Overseer Weill Cornell Medical CollegeAuthor of the book "Orphan: The Quest to Save Children with Rare Genetic Disorders" Michael Rosenblatt, M.D., Board MemberMember of the Harvard Medical School Board of FellowsMember of the research advisory committees of the Massachusetts General Hospital,Brigham and Women's Hospital and Boston Children's HospitalFormer Chief Medical Officer at Flagship PioneeringFormer Chief Medical Officer at Merck Robert Kamen, Ph.D., Board MemberAdvisory Partner at Third Rock VenturesCo-founder and former Chairman of BioAssetsFormer Director of Neon Therapeutics and Harbour AntibodiesFormer President and Unit Head of Abbott Bioresearch Centre, Former SVP at Genetics Institute, Inc.
SEOUL, South Korea, Jan. 26, 2023 /PRNewswire/ -- GenKOre, Inc., a Korea-based biotech company with hypercompact CRISPR-Cas tools, announced that it has entered into a strategic research collaboration with a US-based biopharma company for the development of in vivo gene editing therapies. This collaboration will utilize GenKOre's proprietary CRISPR-Cas platform, TaRGET (Tiny nuclease, augment RNA-based Genome Editing Technology). The TaRGET platform is distinct from the most popular genome editing technology, CRISPR-Cas9, in that the whole editing module can be delivered with a single AAV vector. Based on their TaRGET platform, GenKOre has developed different modalities of editing tools including TaRGET-CUT, TaRGET-Adenine Base Editing (ABE), TaRGET-AI (Gene Activation and Inhibition system). and TaRGET-FREE (Gene knock-in) . "This collaboration reflects the strength and potential utility of TaRGET platform in the application of in vivo gene-editing therapy", said Yong-Sam Kim, CEO of GenKOre. "Not only will this collaboration provide an opportunity to validate the applicability of TaRGET platform to in vivo therapy, but we will also expand the utility of our technologies to a wider spectrum of rare diseases." The collaboration was built on the mutual interest in forging transformative therapeutics for patients with rare diseases. Under the terms of the agreement, GenKOre will receive up to 300 million USD including an upfront, option exercise fees and milestone payments upon successful achievement of R&D and commercial milestones across two in vivo disease targets. In addition, GenKOre will receive research funding and tiered royalties up to a double-digit percentage of net sales.
NANJING, China, Jan. 26, 2023 /PRNewswire/ -- As the tissue engineering and regenerative treatment industry has shown tremendous potential in clinical practices in recent years worldwide, China's blue ocean market welcomes the first wave of acquisitions. ReLive Biotechnologies, Ltd. ("ReLive" or "Company", a leading industry player, announces it has completed the acquisition of all operating assets of Germany-listed biotech company Co.Don AG (CNW.DF) ("Acquisition"), taking over the global patent rights of its flagship products "Spherox" and "Chondrosphere". "Spherox" is one of the only two authorized cell therapy for the regenerative treatment of articular cartilage defects globally. It was authorized by the European Medicines Agency (EMA) for distribution across Europe in 2017. Simultaneously with the Acquisition, ReLive also completed its Series A financing ("Series A") of $36 million to fund the Company's global ambition. ReLive plans to use the fund for R&D, licensing, manufacturing, distributions, and other operational needs in China, U.S. and Europe. Series A was led by Shanghai Healthcare Capital (SHC), with contributions from two existing angel investors, Indaco Capital and Jiuyo Capital, and a new joiner Watson Capital. G&G Capital serves as the exclusive financial advisor. Aside from capital financing, SHC is also expected to support ReLive in both research innovation and commercialization with its robust industry resources. Backed by Shanghai Industrial Investment (Holdings) Co., Ltd (SIIC), Shanghai Pharma and other key players in the healthcare ecosystem, SHC is able to connect ReLive with research institutes, clinical institutions, industrial parks, and the whole medical supply chain. "ReLive's acquisition of Co.Don AG will underpin ReLive's multiple R&D projects on the universal cell therapies for cartilage treatments. It also marks one of ReLive's internationalization efforts, as the company aims at building a true global platform for regenerative medicine." said Dr. Zhao, ReLive's founder, Chairman and CEO. He added that, ReLive will independently research and develop cost-effective tissue engineering products based on a combination of stem cell technology and 3D printing of scaffolds, targeting treatments of hyaline cartilage, osteocartilage, meniscus in orthopedics and sports medicine, and external ear reconstruction in plastic surgery. Acquisition and Financing Fuel ReLive's Growth Plan ReLive is a leading company in tissue engineering and regenerative treatment with global aspirations. Founded by Dr. Zhao in 2021, the Company aims to fulfill the clinical unmet needs in the regenerative treatment market globally as it develops pioneering products in the area of orthopedic sports medicine and reconstructive surgery. The Company will focus equally on independent R&D and acquiring authorized products and the world's top-notch manufacturing and quality control facilities. "Spherox" is one of the only two cell therapies for regenerative treatment of articular cartilage approved by regulatory bodies. Authorized by the European Medicines Agency for distribution across Europe (including EU, U.K., Switzerland, etc.), the therapy has benefited over 17,000 patients with sufficient evidence of efficacy and safety. By taking over 100% of Co.Don AG's core assets via its subsidiary in Germany, ReLive acquires the global patent rights of Spherox, industry-leading manufacturing facilities that meet the "Good Manufacturing Practices"(GMP) standards in two locations in Germany, and quality control facilities and systems. With global patent rights and distribution rights in Europe, the acquisition marks a leap forward in ReLive's global landscape. Manufacturing and Quality Control Lab Compliant with GMP standards "Co.Don AG owns the world's top commercial 'GMP' production quality control system and the world-leading fully automated production lines. We believe the CMC system and the product line will underpin ReLive's universal upgrade of cell therapies for cartilage treatments. ReLive will also introduce the CMC system to China and U.S. to support the Company's global operations," Dr. Zhao said. May Liang, Partner of SHC said, "the number of osteoarthritis patients has exceeded 400 million globally, and the incidence rate is over 50% for people older than 60 years old. Regenerative therapy is one of the most effective and vital ways to treat cartilage defects. SHC will continuously pay close attention to new innovations in the area." "ReLive, through this acquisition, not only has the world's most advanced regenerative technology but also grew with the best global blueprint. We look forward to supporting ReLive to become the leading global cell therapy platform." she added. "Meanwhile, SHC is actively seeking advanced biotech targets overseas for its planned USD fund. SHC's partnership with Hong Kong Science and Technology Parks Corporation (HKSTP) and Shanghai Pharmaceuticals Holding (SPH) will further help SHC to synergize the on-shore and off-shore assets to build a 'global investment ecosystem'." SHC's investment in ReLive aligns with Shanghai's ambition to establish an innovative biotech hub with international influence. In 2022, Shanghai released a three-year action plan to boost Shanghai's cell therapy and accelerate industrial innovation between 2022 and 2024, aiming to increase the scale of cell therapy to 10 billion yuan by 2024. SHC will exploit "the matrix of governments, industry, academics, research, treatments and capital" to support the research and development of the world's most advanced cell therapy, and benefit more patients. More to Come This Acquisition marks only the beginning of ReLive's global expansion. In the future, ReLive will exploit strengths of the U.S., Europe, and China. ReLive will locate R&D in Boston, the world's most advanced biotech hub, continue the manufacturing and global distribution of the existing products in Germany, and focus on product listing and distribution in the US and China. Moreover, ReLive will actively seek strategic partnerships for technology transfers and licensing opportunities in ASEAN, South America, the Middle East, Mexico, and Japan to continuously expand the market globally. In China, ReLive has completed the construction of a production facility for Spherox's, and the Company plans to expeditiously carry out the required bridging clinical trials before launching China's first approved cell therapy for cartilage repair. In other markets like Singapore and HongKong, ReLive is able to take the expedited evaluation route in the pre-submission phase for Spherox as it was already approved by EMA, one of Health Sciences Authority's (HSA) comparable overseas regulators. ReLive has submitted the application for listing to HSA. ReLive is also preparing for similar submission to the US FDA. Manufacturing and Quality Control Lab Compliant with GMP standards ReLive's ambition is backed by a team of top scholars and industry practitioners. The Company's founder Dr. Zhao is a seasonedscientist who completed his Ph.D. and post-docin Regenerative Medicine at the Faculty of Medicine at Utrecht University and Harvard Medical School. He served as the Director of Research & Development and Chair of Research Steering Committee at Vericel when its flagship product MACIÒ received approval from the FDA in 2017. The Company's COO, Zhang Xin is an experienced manager and operator of businesses in the healthcare industry as he held senior positions in multiple multinational medical companies and was the co-founder of Shandong WEGO Orthopedic Device Company Limited, a top-tier orthopedic device company in Asia and a STAR market-listed company. The Research and Development Director of ReLive, Dr. David Bichara, was lecturer in orthopedics at Harvard Medical School, and the director of preclinical research and development with Hyalex, a Boston-based orthopedic device company. Tilmann Bur, previously CEO of Co.Don AG, was also recruited by ReLive as Head of European Market. Mr. Bur will play a vital role in the global commercialization of ReLive's products with valuable experiences in Europe and connections within the industry. The Company also introduced the Strategy and Science Advisory Board that is composed of world-class academics, clinical experts, and top industry leaders, including a professor of Orthopedics at Harvard and the Director of Sports Medicine at Mayo Clinic. Besides the current therapies, a universal version of the cell therapy researched and developed by ReLive is now in the pipeline. The new features are more patient-friendly as the new generation does not require a second surgery. This upgrade will also make scale manufacturing possible. ReLive will continue to cooperate with top scholars from Harvard and M.I.T, and top industry practitioners to expand the Company's innovative pipelines and applications. It will focus on the cross-disciplinary collaboration of the most advanced cell and gene therapies and novel scaffold materials in the application in Orthopedics including treatments of articular hyaline cartilage, osteochondral and meniscus, and in the area of plastic and cosmetic surgery such as ear reconstructions. The US$36 million fund as a vital addition will also stimulate the company's global expansion. Dr. Zhao said: "2022 has been very tough for fundraising given the unprecedented challenges posed to political and business environments. However, we managed to complete our Series A as well as the asset acquisition of Co.Don AG in 2022, laying the ground for our global landscape, and for our ambition to become a leading global regenerative biotech company." "Special thanks for the trust and support from all our investors, especially SHC as the lead investor and a key supporter of the asset acquisition, for empowering ReLive with all-round tremendous resources. We believe ReLive will be a phenomenal success with our joint efforts," Dr. Zhao added.
PlumCare RWE and Lifebit enter a three-year partnership to support Greece's pioneering national newborn genomic sequencing program, BeginNGS. Researchers will be able to access and analyse data securely in combination with global cohorts, whilst ensuring data is kept safe, private and in place in their secure environment using Lifebit's federated technology. This will help detect approximately 400 early onset but actionable genetic conditions in newborns, ensuring appropriate treatments can be given as early as possible, to help limit the impact of the diseases. LONDON, Jan. 25, 2023 /PRNewswire/ -- Lifebit, a leading UK-based precision medicine software company, was awarded a three-year contract with PlumCare RWE to support the scaling of BeginNGS Greece, a pioneering newborn sequencing program. PlumCare RWE partners with Lifebit on Greece’s newborn genomic sequencing program, BeginNGS. The partnership will help detect approximately 400 early onset but actionable genetic conditions in newborns. "We are thrilled to partner with Lifebit as it will allow us to securely connect data from Greece's first newborn screening program with global cohorts. With this, our researchers can gain crucial insights to better understand rare diseases and ultimately save lives," said Petros Tsipouras, Chief Executive Officer at PlumCare RWE. PlumCare RWE partners with Lifebit on Greece’s newborn genomic sequencing program, BeginNGS. The partnership will help detect approximately 400 early onset but actionable genetic conditions in newborns. Worldwide, thousands of children are born with rare genetic conditions that severely affect their quality of life. For approximately 400 early onset but actionable genetic conditions, these can be detected easily using genomic sequencing and appropriate interventions given, allowing affected families to lead lives unburdened by disease. The BeginNGS program, backed by PlumCare RWE, the Rady Children's Institute for Genomic Medicine, and the National Organization of Public Health (EODY) in Greece, aims to sequence the genomes of 1,000 newborns by the end of 2023, scaling to all newborns in Greece by 2027. The program will use whole genome sequencing to diagnose many rare but actionable genetic conditions and identify treatment options before symptoms begin, a major advance over current practices that focus on children who are already critically ill. Rare genetic conditions are, by definition, uncommon within the general population, therefore, to maximise and validate insights from the data, researchers critically need access to multiple large-scale cohorts of clinical-genomic data that are often in different places. Lifebit's powerful federated technology will advance these efforts, making this data interoperable and securely connected with global data cohorts. Lifebit's Chief Executive Officer Dr Maria Chatzou Dunford commented, "We believe all biomedical data that can be used to save lives should be used. We are thrilled to support PlumCare to harness the power of securely connected clinico-genomic data. Our federated technology means that the data remains safe and private throughout by never leaving their secure environment and is used only by trusted researchers. By enabling secure data analysis, our technology will help to deliver life-saving breakthroughs in the diagnosis and treatment of rare diseases." About Lifebit Biotech Ltd. Lifebit builds enterprise data platforms for use by organisations with complex and sensitive biomedical datasets. Lifebit's patented federated technology securely unlocks access to biomedical data. From providing Trusted Research Environments for national precision medicine programmes to enabling pharmaceutical companies to discover new drug targets faster, Lifebit empowers customers to transform how they leverage sensitive biomedical data. Web: www.lifebit.ai/ About PlumCare RWE: PlumCare RWE (Delaware, USA) is a Biotechnology and Healthcare data company. It combines real world evidence and multi-omics analysis to deliver actionable insights for individuals, companies and populations focusing on children. It is building a Global Pediatric Databank that can impact individual and population health and facilitate drug development and discovery. Its European headquarters is based in Athens, Greece. Web: https://www.plumcarerwe.com/
A new bioinformatic pipeline to investigate single cell eQTL analysis is now offered in the sequencing service from Singleron Biotechnologies. COLOGNE, Germany, Jan. 25, 2023 /PRNewswire/ -- Bioinformatic experts at Singleron Biotechnologies, a leader in single cell sequencing, have developed a new single cell eQTL analysis workflow that takes advantage of single cell RNA sequencing (scRNAseq) and whole genome sequencing (WGS) data to predict cell-type specific eQTLs. eQTL (expression Quantitative Trait Loci) are specific variants regulating expression levels of local or distant genes. eQTL analysis, aims to predict the association between genetic variation and gene expression levels, to elucidate the relationship between gene regulation and phenotypic traits. This resource is ideal for customers wanting to perform population studies and requires both wet lab and computational support for single cell eQTL analysis and adds a valuable addition in understanding the molecular mechanisms of disease. It will complement the already established single cell multi-omics sequencing service from Singleron, to provide customers requiring an outsourced solution for their single cell sequencing. Samples are globally accepted to laboratories in Cologne, Germany, and Singapore, where single cell experts with extensive knowledge in both wet lab and data analysis, will support customers to produce a streamlined workflow for their projects. About Singleron Founded in 2018, Singleron develops and commercializes single-cell multi-omics products that can be used in both research and clinical settings. Its current product portfolio includes instruments, microfluidic devices, reagents, software analysis and database solutions that facilitate high-throughput single-cell analysis. The company currently has offices, laboratories, and manufacturing facilities in Germany, Singapore, China, and the US. Its products are used in over 2,000 laboratories in hospitals, research institutes, and pharmaceutical companies. Media ContactEmail: info@singleronbio.com
Discogenic progenitor cell therapy safely increased disc volume and provided rapid, durable improvements in low back pain, function, quality of life, and pain medication usage out to two years post-injection in patients with lumbar DDD. SALT LAKE CITY, Jan. 25, 2023 /PRNewswire/ -- DiscGenics, Inc., a clinical stage biopharmaceutical company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine, today announced positive two-year clinical data from its first-in-human clinical study of IDCT (rebonuputemcel), an allogeneic discogenic progenitor cell therapy for lumbar degenerative disc disease (DDD). During treatment, a single dose of IDCT is injected into the painful disc percutaneously. In the FDA-allowed prospective, randomized, double-blind, vehicle- and placebo-controlled, multicenter clinical study, high dose IDCT (9,000,000 cells/mL; n=20) met the primary safety and efficacy endpoints and demonstrated statistically significant improvements in low back pain, function, quality of life, and disc volume, suggesting a regenerative effect following a single injection into the intervertebral disc. Key findings include: The primary safety endpoint of the study was achieved with no subjects in the IDCT treatment groups experiencing treatment-emergent serious adverse events (TESAEs). As previously reported, the primary efficacy endpoint of the study was achieved, with statistically significant improvement in back pain scores by >30% as measured on a 100mm Visual Analog Scale (VAS) observed in the high dose IDCT group at 52 weeks (–62.79%, p=0.0005). A smaller, significant decrease in VAS was also observed in the vehicle group. Clinically meaningful, statistically significant improvements in low back pain (VAS), function (ODI), and quality of life (EQ-5D) were observed by 12 weeks following intradiscal injection with high dose IDCT in subjects with symptomatic lumbar disc degeneration. These clinical improvements were sustained at six months, one year, 1.5 years, and two years post-injection and statistically exceeded the Minimal Clinically Important Difference (MCID) in each respective outcome measure, which reflect changes following a clinical intervention that are meaningful for the patient. In the low dose IDCT group (3,000,000 cell/mL; n=20), there was a trend in improvement of clinical outcomes, though inconsistent. While the vehicle control group (n=10) resulted in some pain relief, it was not associated with clinically meaningful improvements in function or quality of life. No consistent or durable statistically significant or clinically meaningful outcomes were observed in the saline placebo control group (n=10). Statistically significant improvements in disc volume were also observed in the high dose IDCT group, where MRI imaging-derived mean change in disc volume increased steadily from baseline and reached statistical significance at Week 52 (249.01 mm3, p=0.0284) and Week 104 (402.1 mm3, p=0.028). In contrast, changes in disc volume for the control groups decreased, although not at a statistically significant level. Importantly, the high dose IDCT treatment group was the only group in this study to show a decrease in both opioid and nonsteroidal anti-inflammatory drug (NSAID, e.g. aspirin, ibuprofen, etc.) use. At 2 years, overall patient follow-up was 85.0%. "These clinical results demonstrate the incredible potential of DiscGenics's IDCT to safely treat not only the pain and disability associated with DDD with a single injection, but also to address the underlying cause of the disease—the degenerating disc. This is unlike any treatment I have seen in 30 years of practice and unlike any treatment currently available on the market," said Matthew F. Gornet, M.D., Board Certified Spine Surgeon at The Orthopedic Center of St. Louis and top enroller in the IDCT study. "The improvements we observed in disc volume through MRI image analysis suggest DiscGenics's IDCT produces a regenerative effect within the degenerating disc which indicates the ability to halt and possibly reverse the progression of DDD." The 60-subject study was designed to evaluate the safety and preliminary efficacy of IDCT for the treatment of symptomatic lumbar degenerative disc disease versus vehicle and saline controls. Subjects were enrolled at 13 centers across 12 states. In this study, low back pain was measured on a 100-mm Visual Analog Scale (VAS), function was measured via the Oswestry Disability Index Questionnaire (ODI), and quality of life was measured using the EQ-5D Index Score. "We are very encouraged by the final two-year results of this study," said Flagg Flanagan, Chief Executive Officer and Chairman of the Board for DiscGenics. "The significant and durable improvements we saw in pain, function, quality of life, disc volume, and concomitant pain medication usage are critical indicators of the potential for IDCT to change the paradigm of care for patients with DDD." DiscGenics has submitted a full clinical study report to the U.S. Food & Drug Administration's (FDA) Office of Tissues and Advanced Therapies (OTAT). Simultaneously, DiscGenics is scaling up its in-house manufacturing capabilities so it will have cells ready for future application, pending the FDA's review of the data. A summary of this data has been presented at: The American Academy of Neurological Surgery (AAcNS) 84th Annual Meeting by Kevin T. Foley, MD, Professor of Neurosurgery at the University of Tennessee Health Science Center and Chairman of Semmes-Murphey Clinic on September 29, 2022. The North American Spine Society (NASS) 37th Annual Meeting by Matthew F. Gornet, MD, Board Certified Spine Surgeon at The Orthopedic Center of St. Louis on October 12, 2022. The 41st Annual J.P. Morgan Healthcare Conference by Flagg Flanagan, CEO and Chairman of DiscGenics on January 11, 2023. About IDCTIDCT (rebonuputemcel) is an allogeneic injectable discogenic progenitor cell therapy intended for patients with symptomatic early to moderate degenerative disc disease. The active ingredient (Drug Substance) of IDCT is a live discogenic progenitor cell population derived from the intervertebral disc tissue of adult organ donors. These cells are enriched and expanded into Discogenic Cells through a multistep manufacturing process in a highly controlled environment under current good manufacturing practices (cGMP) that results in significant proliferation and phenotypic changes to the cells. At the completion of the manufacturing process, the Discogenic Cells are subjected to extensive testing prior to use, including identity, purity, potency, and safety evaluations. The Discogenic Cells are then mixed with a viscous Sodium Hyaluronate Solution and excipients to generate IDCT, the Final Drug Product. IDCT is cryopreserved and maintained as individual "off-the-shelf" doses for administration via percutaneous injection into the intervertebral disc in an outpatient setting. IDCT has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). Disclaimer: IDCT is an investigational product that is under development by DiscGenics and has not been approved by the FDA or any other regulatory agency for human use. About the IDCT Clinical Study (DGX-A01)DGX-A01 was a prospective, randomized, double-blinded, vehicle- and placebo-controlled, multicenter clinical study to evaluate the safety and efficacy of IDCT in subjects with single-level, symptomatic lumbar intervertebral disc degeneration. Sixty subjects were enrolled in 13 centers across 12 U.S. states. Prior to enrollment, each participating subject was screened and verified to have met all eligibility criteria, including having early to moderate symptomatic, single-level DDD from L3-S1, no previous lumbar spine surgery, no radiculopathy (pinched nerve) or leg pain, and no comorbidities, such as tumors, fibromyalgia, systemic disease, osteoarthritis, or chronic opioid usage. Upon enrollment, eligible subjects were randomized to one of four treatment cohorts: low dose IDCT (3,000,000 cells/mL; n=20), high dose IDCT (9,000,000 cells/mL; n=20), vehicle alone (n=10), or saline placebo (n=10). Each subject received a single intradiscal injection of his or her assigned treatment into the target symptomatic lumbar intervertebral disc. In accordance with the trial design, subjects in all cohorts were observed and evaluated for two years. Primary outcome measures include safety and reduction in pain. Secondary outcome measures include reduction in disability and radiographic improvement. Through this study, IDCT is being evaluated under an investigational new drug (IND) allowance by the U.S. Food and Drug Administration (FDA) and will be regulated as a drug-biologic through a therapeutics biologics license application (BLA). For more information on the U.S. study, please visit: https://clinicaltrials.gov/ct2/show/NCT03347708. About Chronic Low Back Pain and Degenerative Disc DiseaseChronic low back pain is a serious medical condition that represents a leading cause of disability worldwide and is the most common non-cancer reason for opioid prescription in the U.S. It affects 12-30% of U.S. adults at a given time and is estimated to cost the U.S. healthcare system over $100 billion each year, creating a significant burden on the economy and individual patients dealing with the condition. In nearly 40% of patients, low back pain is caused by DDD, a chronic and progressive condition where the intervertebral disc breaks down and causes pain. About DiscGenicsDiscGenics is a privately held, clinical-stage biopharmaceutical company developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine. DiscGenics's first product candidate, IDCT (rebonuputemcel), is an allogeneic, injectable discogenic progenitor cell therapy for symptomatic, mild to moderate lumbar disc degeneration. IDCT is a mixture of live Discogenic Cells, which are a manufactured progenitor cell population derived from donated adult human intervertebral disc tissue, and a viscous carrier. As the only company in the world to develop an allogeneic cell therapy derived from intervertebral disc cells to treat diseases of the disc, DiscGenics has a unique opportunity to offer a non-surgical, potentially regenerative solution for the treatment of patients suffering from the debilitating effects of back pain. For more information, visit discgenics.com. Photo - https://mma.prnasia.com/media2/1989076/Discgenics_IDCT_injection.jpg?p=medium600Logo - https://mma.prnasia.com/media2/561675/DiscGenics_Logo.jpg?p=medium600
Biotechnology
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