Genome Editing– tag –
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Synthetic Biology Firm Syntax Bio Expands Series A to $14.4M, Boosting Preclinical Research for CRISPR-Based Type 1 Diabetes Beta-Cell Therapy
Fierce Biotech USA Overview Synthetic biology company Syntax Bio announced an expansion of its Series A funding round to $14.4 million, pushing total capital raised over $25 million. This capital injection will primarily drive the develo... -
AATD Treatment Race Intensifies: Beam Therapeutics Plans Pivotal Cohort for Accelerated Approval of Base Editor BEAM-302
CRISPR Medicine News USA Overview The field of Alpha-1 Antitrypsin Deficiency (AATD) treatment is witnessing heightened competition, with five gene editing companies reporting new data and Sanofi introducing a non-edited recombinant prot... -
Prime Medicine’s Autologous Prime Editing Hematopoietic Stem Cell Therapy PM359 for p47phox-Deficient Chronic Granulomatous Disease Receives FDA RMAT Designation
BiopharmaWatch (Prime Medicine Press Release) USA Overview Prime Medicine announced its investigational autologous prime editing hematopoietic stem cell therapy, PM359, for the treatment of p47phox-deficient Chronic Granulomatous Disease... -
Prime Medicine Secures NZ Clinical Trial Clearance for Prime Editing Therapy PM577a in H1069Q-Mutated Wilson Disease, Initiating Global Phase 1/2 Study
GlobeNewswire (Prime Medicine Press Release) USA Overview Prime Medicine has announced the approval of its Clinical Trial Application (CTA) in New Zealand for PM577a, an investigational prime editing therapy for H1069Q-mutated Wilson dis... -
Engineered Exosomes Breach Blood-Brain Barrier, Unlocking New Vistas in Neuro-Oncology
MDPI (論文) Global Overview The blood-brain barrier (BBB) critically impedes drug delivery to the brain, posing a major challenge for treating central nervous system diseases, particularly brain tumors. Engineered exosomes are emerging a... -
CRISPR Medicine News Roundup: Sickle Cell CRISPR Drug Shows Potent Results in Young Children, Caribou’s Off-the-Shelf CAR-T Delivers Positive Phase 1 Data
CRISPR Medicine News USA Overview A CRISPR Medicine News summary article reports that the first approved CRISPR gene therapy for sickle cell disease demonstrated equally potent results in children as young as 5 years old. Additionally, C... -
Beam Therapeutics Gains FDA IND Approval for In Vivo Base Editing Therapy BEAM-304 for PKU
CRISPR Medicine News USA Overview Beam Therapeutics has received U.S. FDA Investigational New Drug (IND) approval for BEAM-304, an in vivo base editing therapy targeting phenylketonuria (PKU). This therapy utilizes adenine base editing d... -
Genomic Engineering Strategies and Alternative Cell Types in Allogeneic T-Cell Immunotherapy Platforms Enable Scalable Cancer Treatment
Academic Publication Global Overview Allogeneic T-cell therapy is emerging as a highly promising strategy in cancer immunotherapy due to its scalability and 'off-the-shelf' availability, overcoming logistical and manufacturing constraint... -
Allogeneic CAR-T Cell Therapies Promise to Overcome Autologous Challenges and Expand Therapeutic Potential in Solid Tumors
PubMed (Review Article) Global Overview This review article details how allogeneic CAR-T cell therapies demonstrate clinical benefits in B-cell malignancies and offer the potential to overcome key limitations of autologous CAR-T, such as... -
First Report of CRISPR-Derived Base Editing in Human Embryos Achieves Precise DNA Changes While Avoiding Large Chromosomal Aberrations
CMN Weekly (CRISPR Medicine News) Unknown Overview CRISPR-derived base editing has been successfully applied to human embryos for the first time, achieving precise DNA changes while circumventing the large chromosomal aberrations typical...
