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SAN FRANCISCO, Oct. 25, 2024 /PRNewswire/ -- Kind Pharmaceutical ("Hangzhou Andao Pharmaceutical Ltd. and Kind Pharmaceuticals LLC"), a clinical-stage biopharmaceutical company focused on developing innovative medicines to treat hematological diseases and cancers, today announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation (ODD) to AND017 in the treatment of Sickle Cell Disease (SCD). AND017 is being developed to treat various anemia indications associated with diseases including non-dialysis dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD). The results for AND017's phase 1 in healthy subjects and phase 2 clinical trials in treatment of anemia in NDD-CKD and anemia in DD-CKD are currently being presented at the annual meeting of American Society of Nephrology (ASN) Kidney Week in San Diego. The preclinical work supporting AND017's ODD in SCD will be presented in future scientific meeting and subsequently published in a scientific journal. Sickle Cell Disease (SCD) is a devasting genetic disease with US patient population around 120,000; SCD disproportionally affects Black and African Americans, comprising more than 98% of patients living with this disease. "The FDA's granting of ODD for AND017 underscores the urgent medical need for new therapies, particularly oral drugs to safely and effectively treat patients with SCD," said Dong Liu, Ph.D., Founder, Chairman, and CEO of Kind Pharmaceutical. "The granting of ODD also demonstrates the innovation capability and the vision of Kind Pharmaceutical." "Hydroxyurea and L-glutamine are the limited FDA-approved oral treatments for SCD, AND017 from Kind Pharmaceutical might not only provide a novel oral treatment with unique mechanism of action, but also an obvious better safety and efficacy profile." said Prof. Gang Huang at UT Health San Antonio, Kathryn Mays Johnson Distinguished Chair in Oncology, who is an expert in studies of hematology and various blood diseases, and involved in the preclinical studies of AND017 in SCD. "I am eager to see how a compound with such unbelievable preclinical safety and efficacy data will translate to real world SCD patients." FDA Orphan Drug Designation (ODD) is granted to a drug or biological product to prevent, diagnose or treat a rare disease or condition that affects fewer than 200,000 people in the United States. ODD qualifies KIND for incentives including tax credit for qualified clinical trials, exemption from user fees, and potential seven years of market exclusivity after AND017's approval for treatment of Sickle Cell Disease (SCD). About Sickle Cell Disease (SCD) Sickle Cell Disease (SCD) affects around 120,000 patients in US and more than 8,000,000 worldwide. As a genetic disease with a single amino acid mutation in the β-globin of mature adult hemoglobin, the hemoglobin molecules of in the red blood cells (RBCs) of SCD patients are prone to aggregate in the tissue after the dissociation of oxygen molecules, which would cause the RBCs sickling in the blood stream and eventually lead to hemolysis and occlusion of capillaries; therefore the hallmarks of SCD are hemolytic anemia and vaso-occlusive crises (VOCs); and SCD causes multiorgan failure and premature death. About AND017 AND017 is a first-in-class hemoglobin elevating agent (HbEA) that targets multiple stages of the red blood cell (RBC) life cycle and is being developed to treat anemia associated with dialysis-dependent chronic kidney disease (DD-CKD), non-dialysis dependent chronic kidney disease (NDD-CKD), cancer-related anemia (CRA), myelodysplastic syndromes (MDS) anemia, sickle cell disease (SCD), and β-thalassemia. About Kind Pharmaceutical ("Hangzhou Andao Pharmaceutical Ltd. and Kind Pharmaceuticals LLC") KIND is a clinical-stage biopharmaceutical company focused on developing innovative medicines to treat hematological diseases and cancers. The company's mission, "kind to human, humble to science, good to patients", drives its commitment to advancing current science to meet unmet medical need. KIND's lead clinical candidate, AND017, first-in-class hemoglobin elevating agent (HbEA), is being developed for treating anemia of various disorders, including dialysis dependent chronic kidney disease (DD-CKD) associated anemia, non-dialysis dependent (NDD) CKD associated anemia, cancer related anemia (CRA), myelodysplastic syndromes (MDS) anemia, sickle cell disease (SCD), and β-thalassemia. KIND's second clinical candidate, AND019, an orally available brain penetrant selective estrogen receptor degrader (SERD), is being developed to treat ER+/Her2- breast cancer. KIND is also developing promising disruptive next generation ADC technologies. For details, please visit . Contact: Dong Liu, PhD, Chairman and CEO of Kind Pharmaceutical Email: [email protected] Tel: 650-315 6151 SOURCE KInd Pharmaceuticals WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

A single intravenous administration of Gene Writer™ resulted in an estimated 79% hepatocyte genomic editing with an average of 84% of mRNA transcripts containing the edited sequence in non-human primates (NHPs) at the SERPINA1 locus responsible for alpha-1 antitrypsin deficiency (AATD)In vivo proof-of-concept achieved for rewriting at carrier level efficiency (52%) of hemoglobin beta (HBB) gene, the locus responsible for sickle cell disease (SCD), in humanized mice with a single dose of Gene Writer delivered in Tessera’s proprietary lipid nanoparticle (LNP)Michael Holmes, Ph.D., Tessera’s Chief Scientific Officer, will present these data today in the session titled In Vivo Gene Editing in Preclinical Models from 3:30 pm – 4:30 pm CEST SOMERVILLE, Mass., Oct. 24, 2024 (GLOBE NEWSWIRE) -- Tessera Therapeutics, a biotechnology company pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, is presenting updates from two of its in vivo genetic medicine programs, for AATD and SCD respectively, including updated rewriting efficiencies using a single dose of RNA Gene Writer combined with Tessera’s proprietary LNP. Additional data were presented on novel computational methods to comprehensively evaluate the genomic safety profile of RNA Gene Writers, highlighting Tessera’s advances towards integrated methods to quantify the specificity and safety of its Gene Writing platform. These new data will be shared in one oral presentation and two poster presentations at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress taking place in Rome, Italy, October 22 – 25, 2024. “The continued development of our Gene Writing and delivery platforms have resulted in rewriting efficiencies in preclinical models exceeding what we anticipate will be curative levels for AATD and SCD,” said Michael Severino, M.D., CEO of Tessera Therapeutics. “We believe these data represent an important step towards advancing potentially transformative in vivo genetic medicines for the treatment of two of the largest monogenic diseases, alpha-1 antitrypsin deficiency and sickle cell disease.” In Vivo AATD Data Tessera presented new preclinical data from NHP studies in its AATD program using a single dose of RNA Gene Writer formulated with a proprietary LNP delivery vehicle. AATD is a monogenic disease of the liver, resulting in an abnormal form of the alpha-1 antitrypsin (AAT) protein that causes both lung and liver manifestations. Analysis of genomic DNA from whole liver samples collected from treated NHPs showed an estimated 79% rewriting efficiency of the SERPINA1 locus in hepatocytes1 on average. A supporting cDNA analysis demonstrated that an average of 84% of mRNA transcripts contained the edited sequence. These data improve upon the first-time demonstration of NHP proof-of-concept presented recently at the Federation of American Societies for Experimental Biology Meeting on Genome Engineering. Data were also presented of an optimized RNA Gene Writer that achieved therapeutically relevant levels of correction at very low doses in the PiZ mouse model, including an estimated 70% genomic DNA correction in hepatocytes, an average of 70% mRNA correction, and an average of 92% of serum AAT corrected at a dose of 0.05 mg/kg, and an estimated 95% genomic DNA correction in hepatocytes, an average of 93% mRNA correction, and an average of 100% serum AAT corrected at a dose of 0.5 mg/kg. In Vivo SCD Data Updated in vivo rewriting results of the HBB locus responsible for SCD in humanized mice showed that Gene Writers formulated in a proprietary LNP developed for delivery to hematopoietic stem cells (HSCs) achieved an average rewriting efficiency of 52% in long-term hematopoietic stem cells (LT-HSCs) after a single intravenous administration. These data build upon the HBB in vivo rewriting data first presented at the American Society of Gene and Cell Therapy 27th Annual Meeting. Additional data show rewriting efficiencies in NHPs exceeding 70% using a surrogate reagent, as well as the maintenance of in vivo HBB edited HSCs and engraftment levels upon secondary bone marrow transplant in humanized mice. 1 Based on the assumption that 60% of liver cells are comprised of hepatocytes About Tessera Therapeutics Tessera Therapeutics is pioneering a new approach to genome engineering through the development of its Gene Writing™ and delivery platforms, with the aim to unlock broad new therapeutic frontiers. Our Gene Writing platform is designed to write therapeutic messages into the genome by efficiently changing single or multiple DNA base pairs, precisely correcting insertions or deletions, or adding exon-length sequences and whole genes. Our proprietary lipid nanoparticle delivery platform is designed to enable the in vivo delivery of RNA to targeted cell types. We believe our Gene Writing and delivery platforms will enable transformative genetic medicines to not only cure diseases that arise from errors in a single gene, but also modify inherited risk factors for common diseases and create engineered cells to treat cancer and potentially autoimmune and other diseases. Tessera Therapeutics was founded in 2018 by Flagship Pioneering, a life sciences innovation enterprise that conceives, creates, resources, and develops first-in-category bioplatform companies to transform human health and sustainability. For more information about Tessera, please visit www.tesseratherapeutics.com. Contact Kristin Politi, Ph.D.LifeSci Communications, LLCkpoliti@lifescicomms.com

CAMBRIDGE, Mass., Oct. 15, 2024 (GLOBE NEWSWIRE) -- Omega Therapeutics, Inc. (Nasdaq: OMGA) (“Omega”), a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines, today announced it will share new preclinical data at three upcoming scientific meetings. “These preclinical results build upon the foundation of data we have shared to date and expand our confidence in the ability of epigenomic controllers to upregulate or downregulate the expression of one or more genes,” said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. “More broadly, we believe these upcoming presentations further demonstrate the power of the OMEGA platform to prospectively engineer novel therapeutic candidates to regulate gene expression with high specificity and the potential of precision epigenomic control to offer new treatment strategies for diverse indications.” Details for the upcoming presentations are as follows: Poster at the Society for Immunotherapy of Cancer (SITC) 39th Annual Meeting taking place in Houston, TX, November 8 – 10 Title: Controlled Epigenetic Upregulation of CXCL9 and CXCL10 in Hepatocellular Carcinoma Promotes T-cell RecruitmentAbstract Number: 1360Session Date and Time: Saturday, November 9, 2024, from 12:15 to 1:45 p.m. CST Poster and Flash Talk at the 12th International mRNA Health Conference taking place in Boston, MA, November 12 – 14 Title: Programmable Epigenomic mRNA Therapeutics Enable Multi-Gene Up or Down Tuning of α-globin via Regulatory Element Targeting: A Precision Approach for β- and α-Thalassemia TreatmentPaper Number: 102E-Poster Session Date and Time: Tuesday, November 12, 2024, beginning at 9:00 a.m. ESTFlash Talk Session Date and Time: Tuesday, November 12, 2024, from 4:00 to 5:00 p.m. EST Poster at the American Association for the Study of Liver Diseases’ (AASLD) 75th Annual The Liver Meeting® taking place in San Diego, CA, November 15 – 19 Title: Durable Upregulation of P1-isoform Hepatocyte Nuclear Factor 4 alpha (HNF4α) Using Novel Epigenomic ControllersPublication Number: 3228 Session Title: MASLD/MASH - Therapeutics: New AgentsSession Date and Time: Sunday, November 17, 2024, from 8:00 a.m. to 5 p.m. PST The posters will be made available on the Omega website at https://omegatherapeutics.com/science/publications/ at the same time as the respective presentations. About Omega Therapeutics Omega Therapeutics is a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines to treat or cure a broad range of diseases. By pre-transcriptionally modulating gene expression, Omega’s approach enables precision epigenomic control of nearly all human genes, including historically undruggable and difficult-to-treat targets, without altering native nucleic acid sequences. Founded in 2017 by Flagship Pioneering following breakthrough research by world-renowned experts in the field of epigenetics, Omega is led by a seasoned and accomplished leadership team with a track record of innovation and operational excellence. The Company is committed to revolutionizing genomic medicine and has a pipeline of therapeutic candidates derived from its OMEGA platform spanning oncology, regenerative medicine, and multigenic diseases including inflammatory and cardiometabolic conditions. For more information, visit omegatherapeutics.com, or follow us on X and LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our expectations surrounding the broad potential of precision epigenomic control, the versatile capabilities of the OMEGA platform, the potential of the Company’s pipeline of therapeutic candidates, and the Company’s participation in upcoming events and presentations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the novel technology on which our product candidates are based makes it difficult to predict the time and cost of preclinical and clinical development and subsequently obtaining regulatory approval, if at all; the substantial development and regulatory risks associated with epigenomic controllers due to the novel and unprecedented nature of this new category of medicines; our limited operating history; the incurrence of significant losses and the fact that we expect to continue to incur significant additional losses for the foreseeable future; our need for substantial additional financing; volatility in capital markets and general economic conditions; our investments in research and development efforts that further enhance the OMEGA platform, and their impact on our results; uncertainty regarding preclinical development, especially for a new class of medicines such as epigenomic controllers; potential delays in and unforeseen costs arising from our clinical trials; the fact that our product candidates may be associated with serious adverse events, undesirable side effects or have other properties that could halt their regulatory development, prevent their regulatory approval, limit their commercial potential, or result in significant negative consequences; difficulties manufacturing the novel technology on which our epigenomic controller candidates are based; our ability to adapt to rapid and significant technological change; our reliance on third parties for the manufacture of materials; our ability to successfully acquire and establish our own manufacturing facilities and infrastructure; our reliance on a limited number of suppliers for lipid excipients used in our product candidates; our ability to advance our product candidates to clinical development; and our ability to obtain, maintain, enforce and adequately protect our intellectual property rights. These and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, and our other filings with the SEC, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.