Latest news with #28thAnnualMeeting
Yahoo
20 hours ago
- Business
- Yahoo
Metagenomi (MGX) Presents Latest in Gene Editing Technologies
Metagenomi, Inc. (NASDAQ:MGX) is one of the 11 Best New Penny Stocks to Buy Right Now. On May 14, Metagenomi, Inc. (NASDAQ:MGX) announced that it is presenting three abstracts at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held from May 13 to 17, 2025, in New Orleans, Louisiana. These presentations highlighted Metagenomi, Inc.'s (NASDAQ:MGX) advancements in compact nucleases for extrahepatic gene editing and CAST (CRISPR-associated transposases) for site-specific integration of large therapeutic genes. A lab scientist peering into a microscope focused on gene editing technology. The company's special metagenomics discovery platform can systematically identify and optimize novel gene editing systems through natural microbial evolution and AI-guided protein optimization. At the meeting, Metagenomi, Inc. (NASDAQ:MGX) shared early proof-of-concept data across different types of gene-editing technologies. This included compact Type II and Type V nucleases for efficient in vivo editing and a programmable Type V-K CAST system capable of targeted integration of large DNA cargoes. The small nucleases can potentially improve delivery to hard-to-reach tissues, while CAST offers promising new tools for targeted and precise large gene integration, addressing one of the greatest challenges in gene editing. Metagenomi, Inc.'s (NASDAQ:MGX) work shows promise in specifically integrating correct copies of complete genes, which could enable treatment for diseases caused by a loss-of-function mutation. Metagenomi, Inc. (NASDAQ:MGX) is a precision gene editing company focused on developing curative therapeutics using its proprietary, metagenomics-derived genome editing toolbox. While we acknowledge the potential of MGX as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 10 Best American Semiconductor Stocks to Buy Now and 11 Best Fintech Stocks to Buy Right Now. Disclosure: None. This article is originally published at Insider Monkey. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
14-07-2025
- Business
- Yahoo
Metagenomi Showcases Advanced AI-Enhanced Gene Editing for Neurological Disorders at ASGCT Meeting
Metagenomi Inc. (NASDAQ:MGX) is one of the best young stocks to buy and hold for 5 years. In mid-May, Metagenomi announced the presentation of 3 abstracts at the American Society of Gene & Cell Therapy/ASGCT 28th Annual Meeting, which was held from May 13 to 17 in New Orleans, Louisiana. The presented data highlighted Metagenomi's advancements in compact nucleases for extrahepatic gene editing and CRISPR-associated transposases/CAST for site-specific integration of large therapeutic genes. These advancements underscore the platform's potential to develop next-gen, in vivo precision medicines, particularly for neurological disorders and diseases caused by loss-of-function mutations. A scientist in a laboratory holding a test tube filled with a glowing blue solution, representing the company's nano-biopharmaceutical research and development. Metagenomi's proprietary gene editing toolbox is enhanced by AI and ML and has analyzed over 7.4 billion proteins and holds the potential to target any type of genetic mutation across the entire human genome. The company's platform offers a full spectrum of gene editing technologies, which include ultra-small nucleases, base editors, and large gene integrations using CAST and RNA-Mediated Integration Systems/RIGS. Metagenomi Inc. (NASDAQ:MGX) is a genetic medicines company that develops therapeutics for patients using a metagenomics-derived genome editing toolbox in the US. While we acknowledge the potential of MGX as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
Yahoo
06-06-2025
- Business
- Yahoo
GenEditBio Announces First Patient Dosed in Investigator-Initiated Trial of GEB‑101, World's First In Vivo CRISPR-Cas Ribonucleoprotein-Based Genome Editing Investigational Therapy for TGFBI Corneal Dystrophy
HONG KONG, BEIJING and BOSTON, June 6, 2025 /PRNewswire/ -- GenEditBio Limited ("GenEditBio"), a clinical-stage biotechnology start-up company focusing on genome editing therapeutic solutions through the discovery of novel and precise Cas nucleases and the development of safe and efficient cargo delivery platforms, today announced a key milestone in the development of its pipeline: First patient has been dosed in an investigator-initiated trial (IIT) of GEB-101, the Company's leading in vivo genome editing program for TGFBI corneal dystrophy. The ongoing IIT is in collaboration with Prof Xingtao ZHOU, MD, PhD, Professor and President, and his team at the Eye & ENT Hospital of Fudan University in Shanghai, China. GEB-101 is a wholly owned program of GenEditBio. Preclinical assessment in non-human primates demonstrated that GEB-101 was well-tolerated after local intrastromal injection and had high safety profile with virtually undetectable off-target effect ( The preclinical research recently received the Excellence in Research Award at the 28th Annual Meeting of the American Society of Gene and Cell Therapy in May 2025 ( The IIT of GEB-101 is an open-label and dose-escalation clinical study to investigate the tolerability of GEB-101 when combined with standard treatment phototherapeutic keratectomy in adults with corneal dystrophy. The first patient, who received GEB-101 in May 2025, has been discharged from the collaborating hospital with no observable adverse event. This trial marks the world's first clinical study of an in vivo CRISPR-Cas ribonucleoprotein (RNP)-based genome editing investigational therapy for TGFBI corneal dystrophy. "Today, on National Sight Day, we are proud and honored to announce that we have initiated the world's first clinical study of an in vivo genome editing investigational therapy for corneal dystrophy. This key milestone represents years of effort in technology platform development and dedication from our world-class research team and clinical partners," said Zongli ZHENG, PhD, Chairman and Co-Founder of GenEditBio, "We stand at the frontier of a new era and recognize the transformative potential of this moment is not just for the Company but for the entire field of genetic medicine because the technology has the potential to extend far beyond corneal dystrophy. Our company is committed to developing fundamentally safe, efficacious and affordable in vivo genome editing therapies for genetic diseases with unmet medical needs". "This investigator-initiated trial marks a defining moment for our award-winning research team and clinical partners. Remarkably, in just one year since initiating our proof-of-concept and preclinical studies, we have successfully integrated our genome editing and delivery technologies to advance this pipeline program from the laboratory to clinical stage, showcasing our competence and dedication to rapidly translate basic research into clinical program to ultimately benefit patients," said Tian ZHU, PhD, CEO and Co-Founder of GenEditBio, "We also express our deepest gratitude to the participant who volunteers in this early stage trial to support medical innovation. As clinical data accumulate, we are positive that GEB-101 has the potential to become a new treatment option for patients with corneal dystrophy". About TGFBI Corneal Dystrophy TGFBI corneal dystrophy is a group of genetic eye disorders caused by mutations in the TGFBI gene, resulting in abnormal protein buildup in the stromal layer of cornea. Symptoms include photophobia, gradual vision loss and recurrent corneal erosions. Current treatment options include phototherapeutic keratectomy and corneal transplantation. These procedures, however, have known limitations such as recurrence and carry risks of sight-threatening complications, underscoring the need for novel therapies. About GEB-101 GEB-101, a wholly owned program of GenEditBio, is a genome editing drug candidate designed as a once-and-done treatment for TGFBI corneal dystrophy. GEB-101 is based on the CRISPR-Cas genome editing technology that targets a particular locus in the mutated TGFBI gene. GEB-101 is encapsulated in the form of RNP in engineered protein delivery vehicle (PDV), a proprietary in vivo delivery system developed by GenEditBio. GEB-101 is to be administered by intrastromal injection and is being investigated for tolerability in the IIT. About GenEditBio Established in 2021 and headquartered in Hong Kong, China, GenEditBio is a gene therapy start-up company with an overarching strategic goal of providing potentially curative, once-and-done and programmable in vivo genome editing-based therapeutic solutions (dubbed "DNA surgery") with high safety profile, unmatched precision, and affordable access for genetic diseases with unmet needs. The Company's core areas of focus include novel Cas nuclease discovery and safe and efficient cargo delivery utilizing lipid nanoparticle (LNP) and engineered protein delivery vehicle (PDV). We have research laboratories and supporting offices in Hong Kong, Beijing, and Boston. GenEditBio is financially backed by top-tier life science investors, including Qiming Venture Partners, Fangyuan Capital, Center Biotherapeutics, Lumosa Therapeutics, HKSTP Venture Fund, and others. For more information, please visit Related Previous Press Releases GenEditBio Scientist Receives Excellence in Research Award at the 28th Annual Meeting of the American Society of Gene and Cell Therapy 2025 GenEditBio to Present Preclinical Data on its Delivery Technology Platform and Drug Pipeline at the 28th Annual Meeting of the American Society of Gene and Cell Therapy Media Contact: Investors Contact: bd@ View original content: SOURCE GenEditBio Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
17-05-2025
- Business
- Yahoo
Be Biopharma Announces New Preclinical Data for BE-102, a B Cell Medicine for the Potential Treatment of Hypophosphatasia
Preclinical research demonstrates that a single administration of BE-102 provides continuous secretion of active alkaline phosphatase (ALP) in vivo out to 6 months No safety findings observed in long-term pharmacology studies Data presented at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting CAMBRIDGE, Mass., May 17, 2025--(BUSINESS WIRE)--Be Biopharma, Inc. ("Be Bio"), a clinical-stage company pioneering the discovery and development of engineered B Cell Medicines (BCMs), today presented results from new preclinical research demonstrating a single administration of BE-102, a BCM for the potential treatment for Hypophosphatasia (HPP), produces continuous levels of active ALP long-term in vivo. The findings will be presented during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting on Saturday, May 17, at 10:45 AM CT. HPP is a genetic disease caused by deficient ALP activity, resulting from pathogenic mutations in the ALPL gene, which leads to multi-systemic clinical complications including deficient bone mineralization. Enzyme replacement therapy (ERT) is the only approved treatment for HPP which requires frequent lifelong injections, and is only available for perinatal/infantile- and juvenile-onset forms of HPP. BE-102 was developed to address these limitations by providing continuous secretion of active ALP from a single infusion, with the flexibility to be titratable and re-dosable as needed. BE-102 is manufactured from primary human B cells by isolating, activating, and precision engineering with CRISPR/Cas9 followed by AAV-mediated delivery of a DNA donor template for the insertion of human ALPL gene into the CCR5 locus (a safe harbor locus) followed by expansion and differentiation in culture into ALP-secreting B lymphocyte lineage cells. "These studies demonstrate the potential of our B cell medicine platform to deliver B cell derived active ALP with durability out to six months," said Rick Morgan, Chief Scientific Officer of Be Biopharma. "BE-102 offers a novel and durable approach that may overcome the limitations of current enzyme therapy and does not require pre-conditioning, offering flexibility for re-dosing." The presentation highlights both in vivo and in vitro data supporting the target product profile for BE-102. In vivo studies were conducted in immune-deficient NOG-hIL6 mice, confirming long-term engraftment and continuous production of B cell derived active ALP (>175 days) following a single IV administration of BE-102. No BE-102 related adverse events have been observed across multiple in vivo studies. In vitro pharmacology data presented today demonstrates that BE-102 secretes active ALP, which is capable of rescuing calcium deposit inhibited by inorganic pyrophosphate (PPi), a potent inhibitor of bone mineralization and an ALP substrate which accumulates in people with HPP. Be Bio's in vitro and in vivo pharmacology and safety data established preclinical proof-of-concept that BE-102 has the potential to be a disease-modifying therapy for people with HPP by providing continuous secretion of ALP, with the flexibility to be titratable and re-dosable as needed. A robust package of preclinical studies is planned in anticipation of submission of an IND for a first-in-human clinical trial for people with HPP. About BE-102 BE-102 is a first-in-class BCM that has been engineered using artificial intelligence-guided protein design to modify primary human B cells to produce ALP, an enzyme deficient in people living with HPP. A single infusion of BE-102 has the potential to provide continuous secretion of therapeutic ALP with the flexibility to be titrated and/or re-dosed, if needed, and without the need for pre-conditioning. BE-102 has been selected as a Development Candidate and has the potential to transform the standard of care for people living with HPP. About Engineered B Cell Medicines – A New Class of Cellular Medicines The B cell is a powerful cell that produces thousands of proteins per cell per second at constant levels, and over decades. Precision genome editing can now be used to engineer B Cells that produce therapeutic proteins of interest, driving a new class of cellular medicines – Engineered B Cell Medicines (BCMs) – with the potential to be durable, allogeneic, re-dosable, and administered without pre-conditioning. The promise of BCMs could transform therapeutic biologics with broad application — across protein classes, patient populations and therapeutic areas. About Be Biopharma Be Biopharma ("Be Bio") is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies. Be Bio was founded in October 2020, and is backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Nextech, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children's Research Institute, Pathway to Cures (the venture philanthropy fund for the National Bleeding Disorders Foundation) and others to re-imagine medicine based on the power of Engineered B cells. For more information, please visit us at and our LinkedIn page. 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Yahoo
15-05-2025
- Health
- Yahoo
Axovia Therapeutics Unveils New Preclinical Data for AXV-201, for Treatment of Genetic Obesity Caused by MC4R Mutations, at ASGCT
Positive preclinical POC data show that novel gene therapy, AXV-201, prevented obesity and metabolic disease in a monogenic model LONDON, May 15, 2025 (GLOBE NEWSWIRE) -- Axovia Therapeutics Ltd., a biotechnology company developing therapies to address the genetic causes of blindness and obesity, announced that today it will unveil new preclinical proof-of-concept data for novelly designed, AXV-201, to treat individuals with severe obesity and very high BMIs resulting from MC4R mutations, in a poster presentation at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, which is being held from May 13-17, 2025 in New Orleans, LA. 'Today's presentation highlights how a new human sequence and AAV9 vector, AXV-201, can rescue the weight gain phenotype and could prove to be a powerful treatment for individuals with severe obesity and very high BMIs resulting from MC4R mutations,' said Dr. Victor Hernandez, Co-Founder and Chief Scientific Officer. 'MC4R is a key regulator of body weight and is the most common cause of genetic early-onset monogenic obesity. A gene replacement therapy could be transformative for those suffering across the globe.' Preclinical proof-of-concept data highlights include: New codon-optimized self-complementary human cMC4R sequence was able to express the MC4R transgene activity greater than five times more efficiently than the wild-type cDNA In vivo administration of AXV-201 in Mc4r-null mice prevented the development of obesity in males and females, restoring a normal weight trajectory comparable to wild-type controls and showed normalization of neurometabolic markers No safety concerns were observed when wild-types cohorts were dosed with AXV-201 'Genetic forms of obesity, such as those linked to MC4R mutations, represent a significant and often overlooked challenge for patients and clinicians alike,' said Dr. Jesse Richards, OU Health. 'Despite advances in obesity research, there remains a critical unmet need for effective therapies that address the underlying genetic causes. These data lay the groundwork for bringing new hope to individuals and families affected by MC4R-related obesity, offering the possibility of a targeted intervention that aims to normalize weight and have a better effect than currently available options.' About Melanocortin 4 Receptor () MutationsMelanocortin 4 receptor (MC4R) mutations are the most common cause of human monogenic obesity. Individuals with MC4R loss of function mutations suffer from hyperphagia, severe obesity and hyperinsulinemia. Impact of the different type of MC4R mutation depends on how they reduce MC4R expression and the impact they have on the production of cyclic AMP (cAMP). Mutations in heterozygosity, with partial MC4R functionality are the most frequent, but homozygous mutations and double heterozygotes account for 25% of MC4R mutations. There are approximately 50,000 patients with complete MC4R loss of function (LoF) and BMI>40 in the United States, European Union and Middle East. About Axovia Therapeutics Therapeutics is leading the development of therapies that address the genetic causes of blindness and obesity syndromes that are driven by cilia dysfunction. Ciliopathies are a group of more than 55 rare inherited genetic diseases linked to more than 950 genes that impact the function of cilia which are critical for protein transport and cellular signaling. The company plans to initiate a clinical study to treat retinal degeneration for its lead program for Bardet-Biedl Syndrome (BBS), AXV-101, in mid-2025, based on robust preclinical efficacy and toxicological data with established scaled GMP manufacturing and patient registries. AXV-101 has achieved U.S. Food and Drug Administration Orphan Drug Designation and Rare Pediatric Disease Designation. The company is developing its second program, AXV-201, for genetic obesity caused by MC4R mutations. Axovia is backed by ALSA Ventures and was formed following decades of work on ciliopathies at University College London by co-founders Professor Phil Beales and Dr. Victor Hernandez. For further information, please visit Contact:Professor Phil BealesChief Executive Officerinvestors@
