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Precision BioSciences' ARCUS Gene-Editing Platform Achieves 85% Efficiency in T-Cells, Advances Hepatitis B and DMD Programs

Gene EditingRare Disease
  • Precision BioSciences' ARCUS gene-editing platform demonstrated exceptional efficiency with gene insertion rates exceeding 85% in T-lymphocytes and 40% in non-dividing human hepatocytes, according to a study published in Nucleic Acids Research.
  • The company's PBGENE-HBV program for hepatitis B treatment has initiated patient recruitment at its first U.S. clinical trial site as part of the Phase 1 ELIMINATE-B trial.
  • The PBGENE-DMD program targeting Duchenne muscular dystrophy showed compelling preclinical results, with treated mice exhibiting up to 93% of maximum force production capacity and 66% improvement in injury resistance.
  • Despite reporting net losses, the company has secured sufficient liquidity to fund operations through the second half of 2027, positioning it for potential sustainable growth as clinical programs mature.

Factor Bioscience Sues AstraZeneca and Cellectis Over Gene-Editing Patent Infringement

Gene EditingOncology
  • Factor Bioscience filed a patent lawsuit against Cellectis and AstraZeneca in Delaware federal court, alleging infringement of three foundational gene-editing patents related to mRNA TALEN technology.
  • The lawsuit claims Cellectis copied Factor's patented technology for designing gene-edited cells for cancer therapies, particularly for treating leukemia and lymphoma.
  • AstraZeneca is accused of violating patent rights through its 2023 licensing agreement with Cellectis for gene-editing technology development in oncology and other therapeutic areas.
  • Factor CEO Matt Angel warns that allowing large pharmaceutical companies to exploit smaller biotech innovations could chill future research and delay new therapy development.

Stanford Researchers Unveil CRISPR-GPT: AI-Powered System Accelerates Gene Editing Experiments

AIGene EditingPrecision Medicine
  • Stanford researchers have developed CRISPR-GPT, an AI-powered system that serves as a "copilot" for gene editing experiments, providing step-by-step guidance and reducing the time needed to develop new treatments from years to months.
  • The multi-agent system outperformed general AI models like GPT-3.5 and GPT-4 in accuracy and reliability across nearly 300 experimental scenarios, with junior researchers achieving 80-90% editing efficiency in real laboratory tests.
  • CRISPR-GPT democratizes gene editing by lowering barriers to entry for novice researchers while incorporating safety guardrails to prevent unethical applications, potentially accelerating the development of life-saving therapies.

SOHM Secures South Korean Patent for CRISPR-Based Gene Editing Platform Targeting CAR-T Therapy Development

Gene EditingCAR-TPrecision Medicine
  • SOHM, Inc. received patent approval from South Korea for its ABBIE gene-editing platform that fuses dCas9 with retroviral integrases for targeted DNA integration.
  • The patent covers "CAS9 Retroviral Integrase and CAS9 Recombinase Systems" technology designed for site-specific genome incorporation in cells and organisms.
  • This Korean patent approval follows earlier European successes and strengthens SOHM's global intellectual property portfolio for next-generation allogeneic CAR-T therapies.
  • The ABBIE platform represents a first-in-class solution for precise gene integration with applications in regenerative medicine and therapeutic development.

YolTech Therapeutics Secures $45 Million Series B Funding to Advance In Vivo Gene Editing Pipeline

Gene Editing
  • YolTech Therapeutics closed a $45 million Series B financing round led by AstraZeneca-CICC healthcare investment fund to support clinical program advancement and global strategic execution.
  • The company operates one of the most extensive in vivo gene-editing clinical pipelines globally, with four programs targeting ATTR, familial hypercholesterolemia, primary hyperoxaluria type 1, and β-thalassemia/sickle cell disease.
  • YolTech's YOLT-201 became the first in vivo gene-editing therapy in China to enter Phase I/IIa clinical trial in 2024, while YOLT-101 achieved first-in-class IND clearance in both China and the United States.

ToolGen Files Patent Infringement Lawsuit Against Lonza Over CRISPR Technology Used in CASGEVY Production

Gene Editing
  • ToolGen has filed a patent infringement lawsuit in the Netherlands against Lonza, alleging unauthorized use of its CRISPR-Cas9 RNP technology in producing CASGEVY, the world's first approved gene-editing therapy.
  • The lawsuit targets Lonza's Dutch manufacturing facility where the company produces CASGEVY under a long-term supply agreement with Vertex Pharmaceuticals for treating sickle cell disease and beta-thalassemia.
  • This legal action follows a similar UK lawsuit filed in April, representing ToolGen's strategic effort to protect its intellectual property rights across key European production sites.
  • ToolGen CEO emphasized the lawsuit aims to secure fair compensation for the company's foundational technology while remaining open to amicable resolution that doesn't restrict patient access to treatment.

Northwestern University Develops Novel Nanostructure to Triple CRISPR Gene Editing Efficiency

Gene EditingPrecision Medicine
  • Northwestern University researchers have developed lipid nanoparticle spherical nucleic acids (LNP-SNAs) that deliver CRISPR gene-editing tools up to three times more effectively than standard methods.
  • The new nanostructure combines spherical nucleic acids with lipid nanoparticles to create a protective DNA shell that enhances cellular uptake and reduces toxicity.
  • Laboratory tests showed the LNP-SNA approach improved gene-editing efficiency threefold and increased precise DNA repair success rates by more than 60% compared to current delivery systems.
  • Flashpoint Therapeutics holds exclusive global licensing rights to commercialize the technology and plans to advance it toward clinical trials for various therapeutic applications.

Vertex Pharmaceuticals Partners with Enlaza Therapeutics in $45 Million Deal to Develop Autoimmune and Gene-Editing Therapies

Gene Editing
  • Vertex Pharmaceuticals has committed $45 million upfront to partner with Enlaza Therapeutics, with potential total deal value exceeding $2 billion for autoimmune disease treatments.
  • The collaboration will leverage Enlaza's proprietary War-Lock platform to develop drug conjugates and T cell engagers targeting autoimmune conditions.
  • The partnership aims to enhance conditioning therapies for patients undergoing gene-editing treatment with Casgevy, Vertex's approved CRISPR therapy for sickle cell disease and beta thalassemia.
  • Enlaza's War-Lock platform creates highly specific warheads that covalently bind to drug targets, expanding therapeutic windows while maintaining selectivity of biologics.

Intellia Therapeutics Gains Momentum as ARK Invest and Institutional Investors Back CRISPR Gene Editing Platform

Gene EditingRare Disease
  • Cathie Wood's ARK Invest recently invested $4 million in Intellia Therapeutics, acquiring 349,930 shares as institutional confidence builds in the gene-editing company.
  • The company's lead therapy NTLA-2002 for hereditary angioedema demonstrates 98% reduction in attack rates over three years, with potential regulatory filing by second half of 2026.
  • Intellia's Phase 3 ATTR trial is expanding to approximately 1,200 participants due to accelerated patient recruitment, indicating strong clinical momentum.
  • Despite stock volatility with shares dropping from above $30 to $11.36, the company maintains over $630 million in cash to fund operations through first half of 2027.

CRISPR Gene Therapy Exa-cel Delivers Sustained Quality of Life Improvements in Blood Disorders

Drug ApprovalGene Editing
  • Treatment with exagamglogene autotemcel (exa-cel), a CRISPR-based gene therapy, led to significant and sustained improvements in health-related quality of life for patients with severe sickle cell disease and transfusion-dependent beta thalassemia.
  • Patients with sickle cell disease saw quality of life scores not only improve but exceed population norms, with adult patients experiencing the largest gains in social and emotional functioning.
  • Beta thalassemia patients reported mean gains of 14.0 points in EQ-5D-5L scores by month 48 for adults and 6.1 points by month 24 for adolescents, all exceeding minimal clinically important difference thresholds.
  • The findings represent among the first quantified patient-reported quality-of-life changes following CRISPR-based gene editing treatment, based on data from ongoing clinical trials with median follow-up of 33.6 months for sickle cell disease and 38.4 months for beta thalassemia.

A Long-term Follow-up Study in Participants Who Received CTX001

NCT04208529Unknown StatusPhase 3
Vertex Pharmaceuticals Incorporated
Posted 1/20/2021

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

NCT03745287CompletedPhase 2
Vertex Pharmaceuticals Incorporated
Posted 11/27/2018

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