MedPath

Tagged News

Synthego Launches GMP SpCas9 to Streamline CRISPR Therapeutic Development

Gene Editing
• Synthego has expanded its CRISPR portfolio with GMP SpCas9, enabling researchers to bundle the nuclease with IND-enabling or GMP sgRNAs for therapeutic development.
• The new product meets certified cGMP manufacturing standards, ensuring exceptional quality, consistency, and scalability required for clinical applications.
• Synthego provides access to Drug Master Files with unified regulatory documentation for both GMP sgRNAs and GMP SpCas9, simplifying regulatory submission processes.

Cellectis Advances Gene Editing with Non-Viral TALEN Technology and TALE Base Editors at ASGCT 2025

Gene EditingCAR-TOncology
  • Cellectis presents breakthrough research on TALEN-mediated non-viral transgene insertion technology that addresses manufacturing constraints and genomic toxicity risks associated with traditional viral methods.
  • The company's TALE base editors (TALEB) demonstrate high-fidelity C-to-T editing with no detectable off-target effects in primary cells, enhancing specificity for therapeutic applications.
  • Research shows circular single-stranded DNA templates maintain better hematopoietic stem cell fitness and provide more stable gene editing compared to viral donor templates.
  • These innovations expand Cellectis' gene editing toolbox for developing next-generation therapies targeting cancer, autoimmune diseases, and monogenic disorders.

Eli Lilly Commits $1.3 Billion to Verve Therapeutics' Cardiovascular Gene Therapy Platform

Precision MedicineGene Editing
  • Eli Lilly has made a $1.3 billion investment commitment to Verve Therapeutics, marking a significant bet on cardiovascular gene therapy development.
  • The partnership represents one of the largest investments in cardiovascular gene therapy, highlighting the potential of this emerging therapeutic approach.
  • Verve Therapeutics has received FDA Fast Track designation for its cardiovascular gene therapy programs, accelerating the regulatory pathway.
  • The collaboration positions both companies at the forefront of next-generation cardiovascular treatments targeting genetic causes of heart disease.

CRISPR Gene Editing Advances CAR-NK Cell Therapy to Overcome Clinical Bottlenecks

Gene Editing
  • CRISPR-Cas9 gene editing is being used to enhance CAR-NK cell therapy by disrupting inhibitory genes like KLRC1, TGFBR2, CISH, and CD38 to improve cytotoxicity and metabolic fitness.
  • Over 60 CAR-NK clinical trials are registered, but none have achieved approval due to challenges including short lifespan, low proliferation, poor tumor trafficking, and immunosuppressive tumor microenvironments.
  • Gene manipulation strategies are addressing key bottlenecks by reducing immunogenicity, enhancing cytotoxicity through modified CD16 receptors, and improving persistence through IL-15 armoring.
  • Novel approaches include disrupting immune checkpoints, preventing fratricide through targeted gene deletion, and incorporating safety switches for selective cell elimination in case of adverse events.

CRISPR Gene Editing Advances from Laboratory to Clinic with 250+ Active Trials Across Multiple Therapeutic Areas

Gene EditingCAR-TDrug Approval
  • CRISPR Medicine News tracks approximately 250 clinical trials involving gene-editing therapeutic candidates as of February 2025, with more than 150 trials currently active across diverse therapeutic areas.
  • CASGEVY became the first approved CRISPR-based therapy in 2023, receiving regulatory clearance in multiple regions for treating sickle cell disease and beta thalassemia through foetal haemoglobin induction.
  • Gene editing for blood disorders continues to lead the clinical landscape, with the majority of Phase 3 trials targeting sickle cell disease and beta thalassemia, while Phase 3 trials are also underway in hereditary amyloidosis and immunodeficiencies.
  • Clinical applications now span 15+ therapeutic areas including blood cancers, viral diseases, metabolic disorders, autoimmune diseases, inherited eye diseases, cardiovascular disease, and neurological conditions.

London Biotechnology Show 2025 Returns with Expanded Platform for Industry Innovation and Collaboration

Gene Editing
  • The second annual London Biotechnology Show (#LBS25) builds on the success of its inaugural edition, which attracted over 3,000 attendees from 1,500 companies and featured 70+ sponsors and exhibitors.
  • This year's expanded event features a 4,000 square meter floor plan showcasing cutting-edge products and services from leading companies including Discovery Park, Merck, Microsoft, and AstraZeneca.
  • The show introduces a Hosted Buyer Programme offering pre-arranged one-to-one meetings to facilitate strategic partnerships and business growth opportunities.
  • An enhanced conference program will feature speakers from major organizations including Novo Nordisk, Pfizer, Bayer Pharmaceuticals, and the Medicines and Healthcare products Regulatory Agency discussing gene editing, regenerative medicine, and digital health innovations.

Prime Medicine Advances Gene Editing Program for Alpha-1 Antitrypsin Deficiency with 72% Correction Rate

Gene EditingRare Disease
  • Prime Medicine unveiled a preclinical program using Prime Editing technology to treat alpha-1 antitrypsin deficiency (AATD), targeting the prevalent E342K mutation in the SERPINA1 gene.
  • Preclinical studies demonstrated up to 72% precise gene correction in hepatocytes of humanized mice, restoring over 95% of serum AAT to the corrected isoform with levels above 20µM.
  • The company plans to file an investigational new drug application in mid-2026, with clinical data expected in 2027 for this genetic liver disease affecting approximately 200,000 people.

Bayer and CRISPR Therapeutics Form $300 Million Joint Venture to Advance Gene-Editing Therapies

Gene EditingRare Disease
  • Bayer is investing $300 million over five years in a new joint venture with CRISPR Therapeutics, plus $35 million for a minority stake in the gene-editing pioneer.
  • The collaboration will focus on developing potential curative treatments for hemophilia, congenital heart disease, and Stargardt disease using CRISPR-Cas9 gene-editing technology.
  • This partnership represents Bayer's first investment through its newly established Bayer LifeScience Centre and joins other major pharmaceutical companies including Vertex, Novartis, and Celgene in backing CRISPR-Cas9 technology.

NHS England Approves First CRISPR Gene-Editing Therapy for Beta Thalassaemia Treatment

Gene EditingDrug ApprovalRare Disease
  • NHS England has approved Casgevy, the first CRISPR gene-editing therapy, for treating severe beta thalassaemia in patients aged 12 and older, making it among the first healthcare systems globally to offer this treatment.
  • Clinical trials demonstrated that 93% of beta thalassaemia patients did not require blood transfusions for at least a year after receiving the one-time gene therapy, offering potential lifetime cure for a condition that typically requires transfusions every 3-5 weeks.
  • The therapy will be available at seven specialist NHS centres within weeks, with an estimated 460 patients in England potentially eligible for the treatment that uses Nobel Prize-winning CRISPR technology to edit bone marrow stem cells.

CAR-T Cell Therapy Advances: Overcoming Challenges in Solid Tumors and Enhancing Safety Through Innovative Engineering

CAR-TGene Editing
  • CAR-T cell therapy has achieved remarkable success in hematological malignancies with complete remission rates of 70-90% in B-cell acute lymphoblastic leukemia, but faces significant challenges in solid tumors due to poor infiltration and immunosuppressive microenvironments.
  • Major toxicities including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) affect nearly 100% of patients in some studies, prompting development of safety mechanisms like suicide genes and reversible inhibition systems.
  • Next-generation CAR-T innovations include multi-targeted designs, armored CAR-T cells with enhanced durability, Boolean logic gates for improved selectivity, and CAR-NK cells that demonstrate superior safety profiles with reduced CRS risk.
  • Novel engineering approaches such as CRISPR/Cas9 gene editing for fifth-generation universal CARs and combination therapies with checkpoint inhibitors show promise for expanding therapeutic applications beyond oncology to autoimmune diseases.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.