MedPath

Tagged News

CRISPR Therapeutics Expands into siRNA Therapies with $95M Sirius Partnership for Thromboembolic Disorders

Gene Editing
  • CRISPR Therapeutics is diversifying beyond gene editing through a strategic $95 million partnership with Sirius Therapeutics to develop SRSD107, a long-acting siRNA therapy targeting Factor XI for thromboembolic disorders.
  • In Phase 1 trials, SRSD107 demonstrated impressive efficacy with over 93% reduction in Factor XI levels and doubled blood clotting time, with effects lasting up to six months from a single dose.
  • The collaboration includes a 50-50 cost and profit-sharing structure for SRSD107 development, with CRISPR leading U.S. commercialization and retaining rights to license two additional siRNA targets.

Prime Medicine Restructures: CEO Replaced, 25% Staff Cut, and Lead Program Halted

Gene Editing
• Prime Medicine announced a major restructuring on Monday, replacing its CEO, laying off 25% of staff, and discontinuing its only clinical program despite previous scientific milestones.
• The company's decision reflects broader challenges in the gene editing sector, where numerous firms have reduced workforce or terminated programs during an extended industry downturn.
• This reorganization highlights the disconnect between promising early scientific data and commercial viability in the advanced therapeutic space, particularly for CRISPR-based technologies.

Eascra Biotech Secures $100,000 MassVentures Grant for Novel Nanoparticle Drug Delivery Platform

Gene EditingOncology
  • Eascra Biotech has received a $100,000 Stage I SBIR Targeted Technologies grant to advance their Janus Base Nanoparticles (JBNps) delivery platform for hard-to-reach tissues.
  • The company has leveraged microgravity research, completing five missions on the International Space Station, becoming the first commercial entity to produce medical nanoparticles in space.
  • Eascra's proprietary technology can deliver RNA therapeutics to challenging tissues like cartilage and solid tumors while maintaining mRNA stability at room temperature, eliminating cold chain requirements.

Tessera Therapeutics Reports Breakthrough Gene Editing Results for Multiple Genetic Diseases

Rare DiseaseGene EditingCAR-T
  • Tessera's RNA Gene Writer technology achieved 76% and 70% editing efficiency in hepatocytes for alpha-1 antitrypsin deficiency and phenylketonuria respectively, with high specificity and durability in non-human primate studies.
  • Preclinical data for sickle cell disease demonstrated greater than 20% editing in long-term hematopoietic stem cells across multiple species, potentially reaching curative thresholds without requiring stem cell transplantation.
  • The company's proprietary lipid nanoparticle delivery system showed high liver specificity with no off-target activity detected, advancing the potential for in vivo gene editing therapies for multiple genetic disorders.

Acuitas Therapeutics and CHOP Pioneer Personalized CRISPR Therapy for Infant with Urea Cycle Disorder

Gene EditingRare Disease
• Acuitas Therapeutics, Children's Hospital of Philadelphia, and University of Pennsylvania successfully delivered the world's first personalized LNP-delivered CRISPR gene-editing therapy to an infant with urea cycle disorder, with no adverse events reported.
• The groundbreaking therapy was developed, manufactured, and delivered in just six months, establishing a new model for rapid development of personalized gene therapies through cross-functional partnerships.
• Acuitas also presented advances in targeted LNP delivery, including DARPin-conjugated formulations achieving up to 98% binding and 90% expression in human CD8+ T cells, expanding therapeutic applications beyond the liver.

World's First Patient Receives Personalized CRISPR Gene Editing Therapy at Children's Hospital of Philadelphia

Gene Editing
  • The world's first patient has been successfully treated with personalized CRISPR gene editing therapy at Children's Hospital of Philadelphia, marking a historic milestone in precision medicine.
  • Dr. Kiran Musunuru from Penn's Perelman School of Medicine expressed hope that this breakthrough method can be replicated for many rare diseases to give patients a fair shot at healthy living.
  • The achievement represents a significant advancement in gene editing technology with potential applications across multiple rare disease conditions.
  • This pioneering treatment demonstrates the clinical viability of personalized CRISPR approaches and could pave the way for broader therapeutic applications in genetic disorders.

Breakthrough Gene Editing Therapy Successfully Treats Infant with Rare Metabolic Disorder

Rare DiseaseGene Editing
  • Researchers at Children's Hospital of Philadelphia have successfully treated an infant with a severe metabolic disorder using a customized gene editing therapy, as reported at ASGCT 2025.
  • The groundbreaking case study, presented by Dr. Kiran Musunuru from the University of Pennsylvania, was simultaneously published in The New England Journal of Medicine.
  • This bespoke gene editing approach targets the underlying genetic causes rather than just managing symptoms, potentially transforming treatment paradigms for metabolic disorders.

Beam Therapeutics to Present Updated BEAM-101 Data from 17 Sickle Cell Disease Patients at EHA 2025

Gene EditingRare Disease
  • Beam Therapeutics will present updated safety and efficacy data from 17 sickle cell disease patients treated with BEAM-101 in the ongoing BEACON Phase 1/2 clinical trial at the European Hematology Association 2025 Congress.
  • BEAM-101 is an investigational one-time base-edited cell therapy that increases fetal hemoglobin production by targeting the HBG1/2 gene promoter regions to address the root cause of sickle cell disease.
  • The company will showcase multiple presentations including red blood cell health biomarkers and manufacturing process data, demonstrating the potential of base editing technology for precision genetic medicines.
  • Sickle cell disease affects approximately 100,000 individuals in the United States and eight million people worldwide, representing a significant unmet medical need for transformative treatments.

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

NCT05456880RecruitingPhase 1
Beam Therapeutics Inc.
Posted 8/30/2022

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 Participants With Transfusion-Dependent β-Thalassemia

NCT03655678Active, Not RecruitingPhase 2
Vertex Pharmaceuticals Incorporated
Posted 9/14/2018

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

NCT03745287CompletedPhase 2
Vertex Pharmaceuticals Incorporated
Posted 11/27/2018

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.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.