Tagged News
Applied StemCell Launches Hypoimmunogenic hiPSC Products to Advance Allogeneic Cell Therapy Development
• Applied StemCell has introduced two new hypoimmunogenic human induced pluripotent stem cell (hiPSC) products designed to overcome immune rejection challenges in allogeneic cell therapy development.
• The products feature B2M/CIITA double knock-out modifications that eliminate HLA class I and II expression, significantly reducing the risk of T cell-mediated immune rejection in transplanted cells.
• Derived from CD34+ umbilical cord blood cells with low mutational burden, these research-use-only tools are isogenic matches to forthcoming GMP-grade versions, offering researchers a seamless transition from early research to clinical development.
CRISPR Therapeutics Expands into siRNA Therapies with $95M Sirius Partnership for Thromboembolic Disorders
• 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
• 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
• 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
• 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
• 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.
Breakthrough Gene Editing Therapy Successfully Treats Infant with Rare Metabolic Disorder
• 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.
Synthego Launches GMP SpCas9 to Streamline CRISPR Therapeutic Development
• 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.
Bayer and CRISPR Therapeutics Form $300 Million Joint Venture to Advance Gene-Editing Therapies
• 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.
Intellia's CRISPR Gene Therapy Shows Potential as Functional Cure for Hereditary Angioedema in Long-Term Study
• Intellia Therapeutics' NTLA-2002, an in vivo CRISPR-based gene editing therapy, demonstrated a 98% mean reduction in monthly hereditary angioedema attack rates across all patients, with follow-up extending beyond two years.
• Eight of ten patients remained completely attack-free following the initial 16-week observation period, with the longest attack-free duration reaching over 26 months and continuing.
• The single-dose treatment showed a favorable safety profile across all dose levels, with no serious adverse events reported, positioning NTLA-2002 as a potential functional cure for this rare genetic disease.
Related Clinical Trials:
Intellia Therapeutics
Posted 12/10/2021