Sickle haemoglobin

PENNINGTON, N.J., March 12, 2025 /PRNewswire/ -- Zynext Ventures USA LLC (Zynext Ventures), the venture capital arm of Zydus Lifesciences (Zydus), today announced its investment in Illexcor Therapeutics (Illexcor), a pioneering biopharmaceutical company developing next-generation oral therapies for sickle cell disease (SCD). This investment underscores Zynext Ventures' commitment to supporting disruptive healthcare innovations that address significant unmet medical needs. Illexcor is developing a first-in-class oral drug that directly targets the root cause of SCD. Their lead asset, currently in preclinical development, is designed to bind to Hemoglobin S and effectively block polymerization and sickling, offering the potential for disease-modifying clinical benefits. Speaking on the development, Dr. Sharvil Patel, MD of Zydus Lifesciences, said, "This investment reflects our commitment to patients battling rare and orphan diseases. Recognizing the profound impact of sickle cell disease on patients' lives, we are pleased to support Illexcor in their efforts to develop a novel therapeutic solution addressing this critical unmet medical need." Emphasizing the strategic significance of the investment, Jay Kothari, Director of Zynext Ventures said, "This investment is consistent with Zynext Ventures' objective of identifying and fostering transformative early-stage healthcare innovations. Beyond the financial backing, Zynext Ventures will leverage its unique data analytics and strategic expertise to accelerate Illexcor's path to delivering this potentially life-changing therapy globally." Andrew Fleischman, CEO of Illexcor stated, "We are excited to partner with Zynext Ventures to advance our lead drug ILX002 into clinical trials later this year. We are hopeful that ILX002 will be a transformative treatment not only for SCD patients in the U.S. but also for millions around the globe. Zynext Ventures and Zydus Lifesciences are in a strategic position to help us achieve these goals." Sickle cell disease is a devastating and debilitating genetic condition that affects up to 10 million people worldwide. The disease continues to severely impact quality of life and longevity. Highly efficacious oral drug therapies are urgently needed to manage this condition on a global scale. About Zynext Ventures Zynext Ventures is the investment arm of Zydus Lifesciences. The venture capital firm focuses on identifying and investing in promising early-stage and growth-stage companies in the healthcare sector. Zynext Ventures provides financial support, strategic guidance, and industry expertise to its portfolio companies, helping them achieve their full potential and make a meaningful impact on the lives of patients. To learn more about Zynext Ventures, please visit: LinkedIn: Zynext Ventures Twitter: @ZynextVentures About Zydus Zydus Lifesciences Ltd. with an overarching purpose of empowering people with freedom to live healthier and more fulfilled lives, is an innovative, global lifesciences company that discovers, develops, manufactures, and markets a broad range of healthcare therapies. The group employs 27,000 people worldwide, including 1,400 scientists engaged in R & D, and is driven by its mission to unlock new possibilities in lifesciences through quality healthcare solutions that impact lives. The group aspires to transform lives through path-breaking discoveries. Over the last decade, Zydus has introduced several innovative, first-in-class products in the market for treating unmet healthcare needs with vaccines, therapeutics, biologicals and biosimilars. For more details visit . About Illexcor Illexcor Therapeutics is a biopharmaceutical company focused on the discovery and development of innovative oral therapies for sickle cell disease. Their lead asset is a first-in-class drug designed to directly target the root cause of SCD, offering the potential for significant disease-modifying clinical benefits. Logo - SOURCE Zydus WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SHANGHAI, China I January 20, 2025 I YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for YOLT-204, an investigational therapy for the treatment of transfusion-dependent beta-thalassemia (TDT). TDT is a severe genetic blood disorder where mutations in the beta-globin gene leads to reduced or absence of hemoglobin, a condition where regular blood transfusion is required to manage anemia and prevent other complications. YOLT-204 is a first-in-class in vivo gene editing therapy leveraging YolTech’s proprietary lipid nanoparticles (LNP). The therapy edits the regulatory region of hemoglobin to induce expression of fetal hemoglobin, potentially alleviating the imbalance of hemoglobin production and normalizing the number of red blood cells in TDT patients. In pre-clinical models, YOLT-204 showed effective and sustained induction of fetal hemoglobin, suggesting therapeutic potential for TDT. YOLT-204 may also be an effective treatment for patients with sickle cell disease (SCD), as increased expression of fetal hemoglobin in these patients has been associated with less polymerization of sickle hemoglobin as well as reduced complications and mortality. The clinical trial for YOLT-204 is a dose-escalation study to preliminarily examine the safety and efficacy of a single-dose regimen with YOLT-204 in TDT. If successful, YOLT-204 may eventually provide an off-the-shelf curative treatment for TDT patients without conditioning chemotherapy and HSCT (Hematopoietic Stem Cell Transplantation). “The initiation of clinical trial for YOLT-204 represents a significant milestone of gene editing therapy development for TDT and SCD,” said Dr. Yuxuan Wu, founder and Chief Executive Officer of YolTech Therapeutics. “We are excited to collaborate with our clinical investigators to bring this innovative therapy to patients.” For further details about the trial or YolTech’s ongoing programs, please visit [ www.yoltx.com ] or contact [ info@yoltx.com ]. About YOLT-204 YOLT-204 is an off-the-shelf in vivo gene editing therapy developed for TDT without conditioning chemotherapy and HSCT. YOLT-204 delivers a gene editor to hematopoietic stem cells through YolTech’s proprietary lipid nanoparticles. The editor together with a guide RNA targets the hemoglobin regulatory region to induce expression of fetal hemoglobin. The expression of fetal hemoglobin has the potential to normalize hemoglobin composition and the red blood cell counts in patients with transfusion-dependent beta-thalassemia, eventually making them transfusion independent. About YolTech YolTech Therapeutics is a clinical-stage in vivo gene editing company committed to pioneering the next generation of precision genetic medicines. Our approach combines innovative gene editing technologies with an advanced lipid nanoparticle (LNP) delivery system, creating a versatile platform designed to address a wide range of serious diseases. Central to our mission is the development of internal capabilities, including end-to-end manufacturing, to ensure the highest standards of quality and scalability. Our lead candidate, targeting ATTR, marks a significant milestone as China’s first LNP-mediated in vivo gene editing therapy to enter clinical development. With promising early clinical outcomes, YolTech is also advancing therapies for familial hypercholesterolemia (FH) and primary hyperoxaluria type 1 (PH1). As a company dedicated to transforming the treatment landscape, YolTech continues to push the boundaries of what is possible in gene editing. For more information, please visit: www.yoltx.com SOURCE: YolTech Therapeutics

SHANGHAI, Jan. 20, 2025 /PRNewswire/ -- YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for YOLT-204, an investigational therapy for the treatment of transfusion-dependent beta-thalassemia (TDT). TDT is a severe genetic blood disorder where mutations in the beta-globin gene leads to reduced or absence of hemoglobin, a condition where regular blood transfusion is required to manage anemia and prevent other complications. YOLT-204 is a first-in-class in vivo gene editing therapy leveraging YolTech's proprietary lipid nanoparticles (LNP). The therapy edits the regulatory region of hemoglobin to induce expression of fetal hemoglobin, potentially alleviating the imbalance of hemoglobin production and normalizing the number of red blood cells in TDT patients. In pre-clinical models, YOLT-204 showed effective and sustained induction of fetal hemoglobin, suggesting therapeutic potential for TDT. YOLT-204 may also be an effective treatment for patients with sickle cell disease (SCD), as increased expression of fetal hemoglobin in these patients has been associated with less polymerization of sickle hemoglobin as well as reduced complications and mortality. The clinical trial for YOLT-204 is a dose-escalation study to preliminarily examine the safety and efficacy of a single-dose regimen with YOLT-204 in TDT. If successful, YOLT-204 may eventually provide an off-the-shelf curative treatment for TDT patients without conditioning chemotherapy and HSCT (Hematopoietic Stem Cell Transplantation). "The initiation of clinical trial for YOLT-204 represents a significant milestone of gene editing therapy development for TDT and SCD," said Dr. Yuxuan Wu, founder and Chief Executive Officer of YolTech Therapeutics. "We are excited to collaborate with our clinical investigators to bring this innovative therapy to patients." For further details about the trial or YolTech's ongoing programs, please visit [] or contact [[email protected]]. About YOLT-204 YOLT-204 is an off-the-shelf in vivo gene editing therapy developed for TDT without conditioning chemotherapy and HSCT. YOLT-204 delivers a gene editor to hematopoietic stem cells through YolTech's proprietary lipid nanoparticles. The editor together with a guide RNA targets the hemoglobin regulatory region to induce expression of fetal hemoglobin. The expression of fetal hemoglobin has the potential to normalize hemoglobin composition and the red blood cell counts in patients with transfusion-dependent beta-thalassemia, eventually making them transfusion independent. About YolTech YolTech Therapeutics is a clinical-stage in vivo gene editing company committed to pioneering the next generation of precision genetic medicines. Our approach combines innovative gene editing technologies with an advanced lipid nanoparticle (LNP) delivery system, creating a versatile platform designed to address a wide range of serious diseases. Central to our mission is the development of internal capabilities, including end-to-end manufacturing, to ensure the highest standards of quality and scalability. Our lead candidate, targeting ATTR, marks a significant milestone as China's first LNP-mediated in vivo gene editing therapy to enter clinical development. With promising early clinical outcomes, YolTech is also advancing therapies for familial hypercholesterolemia (FH) and primary hyperoxaluria type 1 (PH1). As a company dedicated to transforming the treatment landscape, YolTech continues to push the boundaries of what is possible in gene editing. For more information, please visit: SOURCE YolTech Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED