Latest news with #28thAnnualMeeting
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@
Yahoo
15-05-2025
- Business
- Yahoo
Arbor Biotechnologies to Present Data on CNS Programs and Platform Advances at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
– Presentations demonstrate progress in advancing portfolio of CNS gene editing programs in SOD1 ALS and first data presented on company's Angelman syndrome program – Data showcase specificity characterization of Arbor's proprietary type V nucleases to have 10-fold greater sensitivity in off-target detection – Identification and characterization of first RT editor packageable in a single AAV CAMBRIDGE, Mass., May 15, 2025 (GLOBE NEWSWIRE) -- Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today announced the presentation of four posters showcasing Arbor's commitment to CNS diseases of high unmet need and advances in proprietary gene editing technology at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, taking place May 13-17 in New Orleans, Louisiana. CNS Program Presentations Presented data demonstrated preclinical proof-of-concept for Arbor's proprietary type V gene editing platform, which enables delivery of the nuclease and guide construct in a single AAV capsid, in amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations. In vitro and in vivo data detailed the identification and characterization of nuclease and guide constructs targeting SOD1 that efficiently reduced SOD1 protein levels while maintaining low off-target liability. Assessment of top performing editing constructs in a fast-progressing preclinical mouse model of ALS prevented a decline in muscle function which is indicative of motor neuron preservation. Moreover, an ongoing survival study demonstrated an extension of survival by approximately 100 days compared to controls. Arbor's presentations at the meeting included the first public presentation of its differentiated gene editing approach to potentially enable long-term treatment of Angelman syndrome by reactivating the paternal copy of the UBE3A gene, which is normally silenced in Angelman patients. The gene editing approach was able to restore UBE3A protein levels in Angelman patient-derived neurons to or above levels found in neurotypical control neurons or greater. In a novel multi-electrode array assay, developed to evaluate neuron activity, Arbor demonstrated the ability to reverse the hyperexcitability and hypersynchrony phenotype demonstrated in Angelman syndrome neurons to levels observed in neurotypical control neurons. 'We are excited to share our progress in advancing our type V gene editing platform beyond the liver into two CNS diseases of high unmet medical need,' said John Murphy, Ph.D., Chief Scientific Officer at Arbor. 'These data support the continued development of these programs toward the clinic as novel therapeutic approaches with the potential to durably improve patient outcomes.' Arbor's Gene Editing Platform Presentations Building on an earlier oral presentation highlighting preclinical data for ABO-101 in PH1, a separate presentation further elaborated on the in-depth on- and off-target assessment of Arbor's proprietary engineered Cas12i2 type V nuclease. Data presented showed the consistency of Arbor's engineered Cas12i2 to generate 5-20 nucleotide deletions. The similarity between on- and off-target editing patterns enabled Arbor to develop and validate a robust statistical method for off-target analysis. The limit of detection of the verification workflow was shown to be 0.012%, which is 10-fold more sensitive than the industry standard for detection of off-target editing for Cas9-based programs. Arbor showcased this approach in the off-target analysis for ABO-101, with 80% on-target editing and no off-target edits detected above 0.1% in primary human hepatocytes (PHH) and human splenic endothelial cells (HSEC). An additional presentation detailed the first identification and characterization of novel small reverse transcriptase (RT) editors deliverable via a single adeno-associated virus (AAV) vector. Arbor demonstrated robust engineering of the small RT editors (sRTE) to substantially improve RT editing activity and broader targetability with the ability to make precise corrections in induced motor neurons. 'The advances in our platform technology support the sustainability of our gene editing discovery and development platform at Arbor,' said Devyn Smith, Ph.D., Chief Executive Officer at Arbor. 'The ability to increase the sensitivity of our off-target detection allows more rigorous evaluation of the specificity and safety profile of our nucleases prior to entering the clinic. In addition, our identification of, what is to our knowledge the first small RT editors capable of being delivered via single AAV, allows us to further expand potential therapeutic targets for future development.' Details for the poster presentations are as follows: Title: Abstract Number: 1100Session: Wednesday Poster ReceptionSession Date and Time: Wednesday, May 14, 2025, 5:30-7:00 PM CDTLocation: Poster Hall, Hall I2Presenter: Dan Brogan, PhD Title: Abstract Number: 1549Session: Thursday Poster ReceptionSession Date and Time: Thursday, May 15, 2025, 5:30-7:00 PM CDTLocation: Poster Hall, Hall I2Presenter: Adele Bubnys, PhD Title: Abstract Number: 1637Session: Thursday Poster ReceptionSession Date and Time: Thursday, May 15, 2025, 5:30-7:00 PM CDTLocation: Poster Hall, Hall I2Presenter: Ivan Kristanto Title: Abstract Number: 1923Session: Thursday Poster ReceptionSession Date and Time: Thursday, May 15, 2025, 5:30-7:00 PM CDTLocation: Poster Hall, Hall I2Presenter: Amrutha Pattamatta, PhD About ABO-101ABO-101 is a novel, investigational gene editing medicine designed to be a one-time liver-directed gene editing treatment that results in a permanent loss of function of the HAO1 gene in the liver to reduce PH1-associated oxalate production. ABO-101 is currently being evaluated for PH1 in the redePHine Phase 1/2 clinical study (NCT06839235). PH1 is a rare genetic disorder in which enzyme deficiencies in the liver lead to the overproduction and buildup of oxalate, resulting in kidney stones eventually leading to end stage kidney disease and systemic oxalosis. ABO-101 is designed to knock down HAO1 gene expression in the liver, thereby providing durable reduction in oxalate production. ABO-101 consists of a lipid nanoparticle (LNP), licensed from Acuitas Therapeutics, encapsulating messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA which specifically targets the human HAO1 gene. About Arbor Biotechnologies, Biotechnologies™, a clinical stage, next-generation gene editing company based in Cambridge, MA, is advancing a pipeline of novel gene editing therapeutics to address a wide range of genetic conditions – from the ultra-rare to the most common genetic diseases. The company's unique suite of optimized gene editors, which is capable of approaches ranging from gene knockout, excisions, reverse transcriptase editing, and large gene insertion, goes beyond the limitations of early editing technologies to unlock access to new gene targets and has fueled a robust pipeline of first-in-class assets focused on diseases of high unmet need. With Arbor's lead program, ABO-101 for the treatment of primary hyperoxaluria type 1, progressing into clinical trials, the company continues to focus its research and development efforts on genomic diseases of the liver and CNS for which there are no existing functional cures. For more information, please visit: Media Contact:Peg RusconiDeerfield Groupprusconi@
Yahoo
15-05-2025
- Business
- Yahoo
Immunofoco's In Vivo CAR-T Technology Platform Debuts with Groundbreaking Innovations
MxV-G is a novel fusogen for lentiviral packaging that achieves higher vector titers and enhanced transduction efficiency. The detargeted MxV-G mutant abolishes native receptor binding while preserving its fusogenic activity. With various TCMs, the MxV-G mutant selectively transduced T cells, yielding CAR-T cells in mice with efficiency and anti-tumor efficacy comparable to wild-type. TCM3, our in-house developed T cell–targeting module, is designed for specific binding, activation, and transduction of T cells. TCM3 achieved higher T cell transduction rates than αCD3/CD80/CD58 and αCD3/CD80 across various fusogen mutants. MxV-G-mut+TCM3 exhibited superior specificity profile on a panel of human normal or malignant cells TCM3 demonstrated more potent anti-tumor efficacy in mice model. SHANGHAI, May 15, 2025 /PRNewswire/ -- Immunofoco, a company dedicated to advancing cell therapies for solid tumors, announced that its independently developed, innovative lentiviral vector-based In Vivo CAR-T Technology Platform made a remarkable appearance at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). This platform has broken the patent barriers in this field, achieving significant in-vitro and in-vivo specificity and efficacy, and providing a new strategy for tumor immunotherapy. Novel Lentiviral Vector: Overcoming Patent Barriers with Superior Performance Immunofoco's team developed a novel lentiviral vector pseudotyped with the MxV glycoprotein (MxV-G), demonstrating performance in generating CAR-T cells in vivo. Compared with the traditional VSV-G pseudotyped lentiviral vector, MxV-G pseudotyped vector not only enhances viral titer and transduction efficiency but also enables generated CAR-T cells to more effectively target and kill tumor cells. This novel envelope has good clinical application potential in both traditional ex vivo CAR-T and in vivo CAR-T. AI-Driven Optimization: Successful Construction of Precision-Engineered Tropism-Modified Mutants To eliminate the natural tropism of MxV-G and enhance its specificity, the team used an AI-driven protein model to successfully design and construct a mutant MxV-G. The mutated MxV-G eliminates the infectivity to non-T cells while retaining its membrane-fusion-mediating activity. By introducing different T-cell targeting modules, its infectivity to T cells is restored, achieving precise targeting and improving the safety and efficacy of treatment. Next-Generation T-Cell Targeting Molecules: Upgrading Specificity and Anti-Tumor Activity To target T cells precisely, the team engineered multiple T-cell-targeting molecules (TCM). TCM3 demonstrated selective T-cell transduction with no off-target effects and outperformed αCD3/CD80/CD58 (MDF) and αCD3/CD80 in efficiency when paired with different membrane fusion protein variants. CAR-T cells generated by MxV-G-TCM3 showed high specificity across cell lines and reduced T-cell exhaustion markers, supporting sustained activity and improved tumor control. In mouse models, this combination exhibited significantly stronger in vivo anti-tumor efficacy compared to αCD3/CD80/CD58. Dr. Hao Ruidong, Partner and Head of the R & D Center at Immunofoco, said, "CAR-T cell therapy has revolutionized cancer treatment, yet its complex manufacturing and high costs limit accessibility. Our novel in vivo CAR-T platform, powered by lentiviral technology, breaks foreign patent barriers in fusion proteins and T-cell targeting while showing strong in-vitro and in-vivo specificity and efficacy. With simpler manufacturing and lower costs, we aim to make this life-saving treatment accessible to more patients. Moving forward, we'll advance its clinical potential to maximize impact." View original content: SOURCE Immunofoco Biotechnology
Yahoo
14-05-2025
- Business
- Yahoo
Aspen Neuroscience to Present at American Society of Gene & Cell Therapy (ASGCT) Annual Meeting
Presentation Covers Aspen's Autologous iPSC-derived Approach for Neuron Replacement Therapy in Trial for Parkinson's Disease SAN DIEGO, May 14, 2025 /PRNewswire/ -- Aspen Neuroscience Chief Scientific Officer Xiaokui Zhang, Ph.D., will present at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held this week May 13-17, at the Ernest N. Morial Convention Center in New Orleans, Louisiana. The annual meeting, the largest gathering of cell and gene therapy professionals, hosts a global audience of attendees from academia, the biotechnology and pharmaceutical industries, government, and non-profit patient foundations. "We are excited to be part of this meeting, which covers the latest advancements in cell and gene therapies," commented Dr. Zhang. "The members of the ASGCT community share our vision to translate the potential of these therapies into safe and effective medicines to improve patients' lives." Dr. Zhang will present during the Scientific Symposium "Next Generation Strategies for Evading Immunity in Stem Cell Therapies" (Organized by the Stem Cell Committee), scheduled for Friday May 16 from 1:30 - 3:15 PM. She will discuss recent developments in personalized, autologous cell therapy research and development, including for ANPD001, Aspen's iPSC-derived neuron replacement therapy currently under investigation for Parkinson's disease in a Phase 1/2a clinical trial. About Aspen Neuroscience Headquartered in San Diego, Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on autologous regenerative medicine. The company's patient-derived iPSC platform is used to create personalized therapies to address diseases with high unmet medical needs, beginning with autologous neuron replacement for PD. Aspen combines cell biology with the latest machine learning and genomic approaches to investigate patient-specific, restorative cell treatments. The company has developed a best-in-class platform to create and optimize pluripotent-derived cell therapies, which includes in-house bioinformatics, manufacturing and quality control. For more information and important updates, please visit View original content: SOURCE Aspen Neuroscience, Inc. Sign in to access your portfolio
