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iNtRON Bio Files Patent Applications for CRISPR-Cas9 Gene Editing Technology Targeting Pasteurella Bacteriophages

Gene Editing
  • iNtRON Bio has filed two patent applications for its IMPA™ gene editing technology that uses CRISPR-Cas9 and recombination methods to modify Pasteurella bacteriophages for therapeutic applications.
  • The technology enables precise genetic manipulation of bacteriophage capsids to create modular phages capable of carrying multiple therapeutic payloads, representing the first reported gene editing of Pasteurella bacteriophages worldwide.
  • The platform leverages an integrated database containing over 11,000 bacteriophage records and 600 whole genome sequences to develop targeted therapeutics, anticancer drugs, and vaccines.
  • The company plans to expand the technology beyond Pasteurella to other bacterial targets and develop various drug candidates including ADC drugs, anticancer agents, and vaccines.

Axelyf Closes $2.6M Seed Round to Advance Superior RNA-LNP Gene Editing Technology

Gene EditingAI
  • Axelyf Inc. secured $2.6 million in seed funding led by Brunnur Ventures to develop its proprietary AXL lipid nanoparticle technology for RNA therapeutics.
  • The company's AXL technology demonstrated superior in vivo gene editing potency compared to LP-01 benchmark in Phase 3 trials, achieving higher efficiency at lower doses without liver toxicity.
  • Axelyf combines novel ionizable lipids with ANNA™ AI platform for lipid optimization, positioning the company as an emerging leader in RNA medicine delivery systems.
  • The funding will support development of lead candidate AXL-003 targeting autoimmune conditions and advance partnerships in the rapidly evolving RNA therapeutics field.

Qihan Biotech Receives FDA Clearance for QT-019B, First Universal Dual-Target CAR-T Therapy for Refractory Lupus

CAR-TGene Editing
  • The FDA has cleared Qihan Biotech's IND application for QT-019B, enabling a Phase 1/2 clinical trial for refractory systemic lupus erythematosus in the United States.
  • QT-019B is an off-the-shelf allogeneic CAR-T therapy targeting both CD19 and BCMA antigens, engineered with gene editing to reduce immune rejection and enhance persistence.
  • The therapy has shown promising results in investigator-initiated clinical studies in China against multiple refractory autoimmune diseases, including systemic lupus erythematosus.
  • Qihan Biotech is developing next-generation CAR-T therapies that will not require lymphodepletion, potentially transforming treatment paradigms for autoimmune diseases.

Anocca Secures $46M to Advance Europe's First Non-Viral Gene-Edited TCR-T Therapy for Pancreatic Cancer

Gene Editing
  • Anocca AB raised SEK 440 million ($46 million) in financing led by Mellby Gård to advance VIDAR-1, Europe's first non-viral gene-edited TCR-T cell therapy targeting pancreatic cancer.
  • The Phase I multi-center trial is now recruiting patients across eight sites in Sweden, Denmark, Germany, and The Netherlands, targeting mutant KRAS in pancreatic ductal adenocarcinoma.
  • VIDAR-1 represents a breakthrough approach for pancreatic cancer patients, who face a five-year survival rate of less than 10% with limited definitive treatment options currently available.
  • The funding will support early-stage clinical development of VIDAR-1 and advance Anocca's preclinical pipeline of TCR-T therapies for solid tumors and other difficult-to-treat diseases.

Precision BioSciences Reports First Clinical Evidence of Hepatitis B Gene Editing Therapy PBGENE-HBV

Gene Editing
  • Precision BioSciences' PBGENE-HBV demonstrated substantial antiviral activity in all three patients in the lowest dose cohort, achieving 47-69% reductions in hepatitis B surface antigen levels.
  • The gene editing therapy showed a well-tolerated safety profile with no serious adverse events or dose-limiting toxicities observed across multiple dose administrations.
  • One patient maintained a durable 50% reduction in HBsAg levels seven months after initial treatment, providing first clinical evidence of sustained viral DNA editing.
  • PBGENE-HBV represents the first and only clinical modality designed to eliminate covalently closed circular DNA and inactivate integrated DNA for potential hepatitis B cure.

MaxCyte Partners with Anocca to Advance TCR-T Cell Therapy Manufacturing Using Non-Viral Gene Editing Platform

Gene Editing
  • MaxCyte has signed a Strategic Platform License Agreement with Anocca AB to support the scalable development and manufacturing of TCR-T cell therapies using Flow Electroporation technology.
  • Anocca will deploy MaxCyte's ExPERT platform for non-viral gene editing workflows across its therapeutic pipeline, including a lead program targeting mutant KRAS-driven advanced pancreatic cancer.
  • The partnership provides Anocca with non-exclusive research, clinical and commercial rights to MaxCyte's technology platform, while MaxCyte receives annual licensing fees and program-related revenue.
  • Anocca recently received GMP compliance certification and manufacturing license from Swedish regulators for its cell therapy production facility.

Aldevron Establishes Innovation Center in Boston Biotech Hub to Accelerate Genomic Medicine Development

Gene Editing
  • Aldevron, a Danaher-owned leader in DNA, RNA, and protein manufacturing, has opened a new Innovation Center in Waltham, Massachusetts, positioning itself at the heart of the Boston biotech corridor.
  • The facility is designed to accelerate advancements in cell-free DNA, gene editing, and mRNA analytics, enabling faster collaboration with clients and shorter project timelines for advanced therapies.
  • The expansion builds on Aldevron's recent breakthrough in producing the world's first personalized CRISPR gene editing therapy for urea cycle disorder, demonstrating its capability to translate scientific discoveries into scalable solutions.
  • The strategic move leverages Boston's unmatched biotech ecosystem, including proximity to MIT, Harvard, and the Broad Institute, while benefiting from Danaher Corporation's $40 billion life sciences portfolio.

Pluristyx Secures Strategic Investment to Advance iPSC Technologies and Gene-Edited Cell Lines

Gene Editing
  • Pluristyx completed a funding round led by BioLife Solutions and BroadOak Capital Partners to expand its induced pluripotent stem cell technology portfolio.
  • The investment will support expansion of clinical-grade cell lines incorporating proprietary FailSafe® and iACT™ engineering with hypoimmune genetic modifications.
  • Funding will also advance the recently launched PluriForm™ kit for organoid generation and complex test systems development.
  • The company's iPSC products are designed to serve as universal raw materials for next-generation cell-based therapies targeting currently untreatable diseases.

Regeneron and Intellia Advance CRISPR Gene Therapy Trial for Hemophilia B Treatment

Rare DiseaseGene Editing
  • Regeneron Pharmaceuticals and Intellia Therapeutics are conducting a two-part clinical study evaluating REGV131-LNP1265, a CRISPR/Cas9-based gene insertion therapy for hemophilia B patients.
  • The experimental therapy aims to enable patients' bodies to naturally produce clotting factor IX, potentially reducing or eliminating the need for regular factor replacement therapy.
  • The ongoing study began in September 2024 and follows a dose escalation approach in adults before expanding to adolescents and children.
  • The gene therapy uses CRISPR/Cas9 technology to insert a functional gene into patients' DNA, representing a potential long-term solution for hemophilia B treatment.

Cystic Fibrosis Foundation Commits Additional $24 Million to Prime Medicine for Gene Editing Therapy Development

Gene Editing
  • The Cystic Fibrosis Foundation has committed up to $24 million in additional funding to Prime Medicine to advance prime editing gene therapy for cystic fibrosis patients.
  • The funding will focus on developing a prime editing therapy targeting the G542X nonsense mutation, one of the most prevalent CF-causing mutations with no available treatments.
  • Prime Medicine's prime editing technology has the potential to permanently correct CFTR gene mutations and could eventually treat the vast majority of people with cystic fibrosis.
  • The company is investigating multiple approaches including hotspot editing and PASSIGE technology to address various CF mutations through a single platform.

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