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Jul 11, 2025, 06:12 ET June in Review: 14 Top Health Press Releases
– Replace a Silenced Gene Called Alpha-Klotho ("α- KLOTHO") Recent clinical and pre-clinical studies and analysis indicates the potential to increase lifespan and reduce age-associated degeneration in multiple organ systems has be realized with a focus on the human gene called Klotho.
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Jul 11, 2025, 06:02 ET 14 Top Press Releases from June
acquire Verve Therapeutics to advance one-time treatments for people with high cardiovascular riskVerve is developing a pipeline of gene editing medicines designed to address the drivers of atherosclerotic cardiovascular disease (ASCVD) through treatments that may only need to be given
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Jul 10, 2025, 11:54 ET REGENXBIO Announces Publication of Preclinical Results Demonstrating Functional Benefits of Novel Microdystrophin Construct in RGX-202 Investigational Gene Therapy for Duchenne Muscular Dystrophy
approved microdystrophin gene therapy candidate for the treatment of Duchenne muscular dystrophy (Duchenne) that includes the CT domain, a key portion of dystrophin, making it the closest to naturally occurring dystrophin. "We specifically designed RGX-202 differently from other gene therapies with the
More news about: REGENXBIO Inc.
Jul 10, 2025, 09:00 ET Sarepta Therapeutics, Inc. Sued for Securities Law Violations - Investors Should Contact The Gross Law Firm Before August 25, 2025 to Discuss Your Rights - SRPT
during the class period, Defendants issued materially false and/or misleading statements and/or failed to disclose that: (i) ELEVIDYS, a prescription gene therapy for Duchenne, posed significant safety risks to patients; (ii) ELEVIDYS trial regimes and protocols failed to detect severe side effects; (iii)
More news about: The Gross Law Firm
Jul 10, 2025, 08:54 ET LG Debuts Next-Generation Precision Medical AI
model's gene mutation prediction accuracy to a best-in-class 78.4%, establishing a new State-of-the-Art (SOTA) benchmark. 'EXAONE Path 2.0' was developed using over 10,000 matched datasets comprising Whole Slide Images and multi-omics data. This robust training enables it to predict gene expression
More news about: LG AI RESEARCH
Jul 10, 2025, 07:49 ET CS Genetics Launches 96-Sample SimpleCell™ Kit, Delivering Same-Day Single Cell RNA-Seq at Population Scale
2025 /PRNewswire/ -- CS Genetics, a privately held genomics technology company, today announced the commercial launch of its 96-sample SimpleCell 3' Gene Expression Kit, further expanding the capabilities of its SimpleCell platform for scalable, cost-efficient single cell RNA sequencing.
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Jul 10, 2025, 06:00 ET Vita Therapeutics and I Peace announced a development program to generate iPS-derived differentiated cells for transplant therapy with an initial focus on FSHD (Facioscapulohumeral Muscular Dystrophy) using universal iPS cells
provide the technology necessary for gene editing and developing iPSC-derived universal cells. The manufactured MCB through this collaboration will be registered in the FDA Drug Master File (DMF). I Peace will retain rights to supply MCB's of normal and gene-edited cells and to supply differentiated
More news about: I Peace, Inc.; VITA Theraputics
Jul 10, 2025, 05:00 ET Klotho Neurosciences, Inc. Granted FDA Orphan Drug Designation for KLTO-202 for Treatment of Amyotrophic Lateral Sclerosis ("ALS" or "Lou Gehrig's Disease")
KLTO), a gene and cell therapy company focused on the treatment of neurodegenerative and other aging-related diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company's novel secreted-Klotho (s-KL) promoter, gene and delivery system
More news about: Klotho Neurosciences, Inc.
Jul 09, 2025, 17:16 ET Berger Montague PC Investigates Rocket Pharmaceuticals (RCKT) Over Safety Disclosures and Stock Drop
New Jersey and alleges that the Company misled investors regarding critical safety disclosures tied to its RP-A501 gene therapy program.
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Jul 09, 2025, 14:45 ET Two-faced gene variant appears good for men, bad for women
rely upon muscle cells carrying glucocorticoid receptors. This study delved into what happens when a gene variant changes how those receptors function. Some studies have already shown that the gene variant rs6190 appears to enhance muscle strength, lean body mass
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Jul 09, 2025, 11:00 ET Branching out: Tomato genes point to new medicines
involve genes known as paralogs. "Paralogs emerge across evolution through gene duplication and are major features of genetic networks," Lippman explains. "We know paralogs can buffer against each other to prevent gene mutations from affecting traits. Here, we found that collections of natural and
More news about: Cold Spring Harbor Laboratory
Jul 09, 2025, 09:49 ET $14 Billion Cell and Gene Therapy Market to Double by 2030, Driven by Oncology and RNA Breakthroughs, reports Kalorama Information
currently leading the cell and gene therapy market with sales generated from its oncology CAR-T therapy Kymriah, its acquired gene therapy for muscular atrophy Zolgensma, and Leqvio. Gilead Sciences, with Yescarta and Tecartus, is the second largest provider of cell and gene therapy, specializing in oncology
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Jul 09, 2025, 09:00 ET CDI Labs Canada Launches Hormone-focused PhIP-Seq Library for Highly Multiplexed Analysis of Serum Autoantibodies
clinical deployment phases, and include: autoantibody biomarker discovery and profiling; viral exposure profiling; immunogenicity assessment of cell, gene and radioligand therapies; antibody and ADC specificity profiling; target identification for monoclonals, biologics and drugs; and custom protein and
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Jul 09, 2025, 08:30 ET Breaking Through: Systemic Genetic Medicines for Hard-to-Treat Cancers
creating transformative gene-based medicines for serious diseases, is reporting updates across its in vivo cardiovascular disease programs. These updated include new data for CTX310(TM), targeting ANGPTL3, as well as continued progress on CTX320(TM), targeting the LPA gene, and CTX340(TM), targeting
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Jul 09, 2025, 08:12 ET IASO Bio Receives Orphan Drug Designation from the Ministry of Food and Drug Safety of South Korea for Equecabtagene Autoleucel
Autoleucel（Fucaso） Equecabtagene Autoleucel（Fucaso） is an innovative fully human anti-BCMA CAR-T cell therapy which uses lentivirus as a gene vector to transfect autologous T cells. The CAR contains a fully human scFv, CD8a hinge and transmembrane domain, and 4-1BB co-stimulatory molecule
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Jul 09, 2025, 07:00 ET Genomenon and UCB Partner to Advance Thymidine Kinase 2 Deficiency Awareness and Diagnosis
Genomenon has used its unique combination of AI and team of genomic experts to curate and classify all published TK2 gene variants, making them freely available to the global clinical research community via the Mastermind Genomic Intelligence platform. To further expand
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Jul 09, 2025, 06:45 ET FDA approves updated label for Lilly's Kisunla (donanemab-azbt) with new dosing in early symptomatic Alzheimer's disease
dizziness, nausea, difficulty walking, confusion, vision changes and seizures. Some people have a genetic risk factor (homozygous apolipoprotein E ε4 gene carriers) that may cause an increased risk for ARIA. Talk to your healthcare provider about testing to see if you have this risk factor. You
More news about: Eli Lilly and Company
Jul 08, 2025, 17:43 ET Yale School of Medicine Receives $27.7 Million Grant Toward Autism Research
epilepsy, and those with Angelman Syndrome, a condition marked by severe speech and language impairments, sleep disturbances, and anxiety due to a single gene mutation. The study will collect noninvasive clinical and behavioral evaluations developed for use in multisite autism studies in parallel with
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Jul 08, 2025, 17:02 ET Colossal Biosciences, Ngāi Tahu Research Centre, and Sir Peter Jackson Launch Strategic Partnership to Revive the South Island Giant Moa and Other Taonga Species
will follow a methodical approach combining scientific rigor with Māori values, beginning with ancient DNA recovery and genomic analysis, followed by gene identification and development of reproductive technologies. Indigenous Leadership and Economic Opportunities The Ngāi Tahu Research
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Jul 08, 2025, 16:12 ET Class Action Announcement for Sarepta Therapeutics, Inc. (SRPT): Kessler Topaz Meltzer & Check, LLP Announces that a Securities Class Action Lawsuit Has Been Filed Against Sarepta Therapeutics, Inc.
alleges that, throughout the Class Period, Defendants made false and/or misleading statements and/or failed to disclose that: (1) Sarepta's gene therapy treatment, ELEVIDYS, posed significant safety risks to patients; (2) ELEVIDYS trial regimes and protocols failed to detect severe side effects;
More news about: Kessler Topaz Meltzer & Check, LLP
Jul 08, 2025, 12:45 ET ChristianaCare and Children's Hospital of Philadelphia Agree to Join Forces, Expand Access to World-Class Pediatric Care in Delaware
comprehensive stroke center and regional centers of excellence in heart and vascular care, cancer care and women's health. It also includes the pioneering Gene Editing Institute. ChristianaCare is nationally recognized as a great place to work, rated by Forbes as one of the nation's best employers for
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Jul 08, 2025, 10:30 ET $2.35 Billion by 2030: Why In Situ Hybridization Is Revolutionizing Molecular Diagnostics | MarketsandMarkets™
Technological innovations—including multiplex ISH, high-sensitivity probes, and automation—are enabling more accurate, efficient, and spatially resolved gene expression analysis in both clinical and research settings. From oncology to neuroscience, ISH is paving the way for advanced diagnostics and next-generation
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Jul 08, 2025, 09:00 ET Levi & Korsinsky Reminds Sarepta Investors of the Pending Class Action Lawsuit with a Lead Plaintiff Deadline of August 25, 2025 - SRPT
CASE DETAILS: The filed complaint alleges that defendants made false statements and/or concealed that: (i) ELEVIDYS, a prescription gene therapy for Duchenne, posed significant safety risks to patients; (ii) ELEVIDYS trial regimes and protocols failed to detect severe side effects; (iii)
More news about: Levi & Korsinsky, LLP
Jul 08, 2025, 08:11 ET Myrtelle Launches Manufacturing of First-in-Class Gene Therapy for Canavan Disease in Strategic Alliance with Charles River and Viralgen
a pioneering clinical-stage gene therapy company dedicated to revolutionizing treatment for neurodegenerative diseases, today announced the official launch of commercial-stage manufacturing for its first-in-class oligotrophic recombinant adeno-associated virus (rAAV) gene therapy product, developed specifically
More news about: Myrtelle, Inc
Jul 08, 2025, 06:00 ET Chan Zuckerberg Initiative, Innovative Genomics Institute Announce New Center for Pediatric CRISPR Cures
clinical success in treating 'Baby KJ' Muldoon, an infant born with an exceedingly rare metabolic disease, with the first-ever personalized CRISPR gene-editing therapy. The IGI, a joint effort between UC Berkeley, UCSF and UC Davis founded by Nobel laureate Jennifer Doudna,
More news about: Chan Zuckerberg Initiative