September 2026– date –
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FDA Expands CASGEVY Pediatric Indication: CRISPR Gene-Editing Therapy Evaluated for Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia in 5-11 Year Olds
HCA Healthcare Today USA Overview The FDA expanded approval for CASGEVY (exagamglogene autotemcel), a CRISPR-based gene-editing therapy for sickle cell disease and transfusion-dependent beta-thalassemia, to eligible patients aged 2 years... -
Fate Therapeutics to Present on iPSC-Derived CAR T Cell Candidate FT819 for Autoimmune Diseases at 2026 Cantor Global Healthcare Conference
Stock Titan USA Overview Fate Therapeutics announced its participation in a fireside chat at the 2026 Cantor Global Healthcare Conference on September 9th, where it will discuss its off-the-shelf iPSC-derived CAR T cell candidate FT819 f... -
International Conference ‘3D Bioprinting & Regenerative Medicine’ in Berlin Highlights Advances in Personalized Medicine and Organ Regeneration
Coalesce Research Group Germany Overview The International Conference on '3D Bioprinting & Regenerative Medicine,' held in Berlin from August 27-28, 2026, showcased how 3D bioprinting is revolutionizing regenerative medicine by enabl... -
ESC Congress 2026 Reveals Breakthrough Gene Therapies for Cardiovascular Disease: AAV8 Reduces LDL-C by 80-90%, siRNA Kylo-11 Achieves 97% Lp(a) Reduction
HCPLive (YouTube) USA Overview Groundbreaking data on multiple gene and molecular therapies for cardiovascular disease were presented at ESC Congress 2026. An AAV8-mediated gene therapy for homozygous familial hypercholesterolemia (HoFH)... -
University of Toronto Develops Next-Gen RNA Therapy Using Engineered tRNA to Bypass Premature Stop Codons, Offering Hope for Thousands of Untreatable Genetic Diseases
University of Toronto Canada Overview Researchers at the University of Toronto have developed a groundbreaking next-generation RNA therapeutic approach that enables cells to 'read through' disease-causing premature termination codons (PT... -
Ocugen Doses First Patient in Phase 3 Registrational Trial for OCU410 Modifier Gene Therapy for Geographic Atrophy, Following RMAT Designation
GlobeNewswire USA Overview Ocugen has initiated patient dosing in its Phase 3 registrational trial for OCU410, a modifier gene therapy targeting geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). OCU410 rece... -
Fate Therapeutics Initiates Phase 1/2 Trial for Novel Cell Therapy FT839 in Autoimmune Diseases, Expanding Pipeline
TipRanks.com USA Overview Fate Therapeutics has launched a Phase 1/2 dose-escalation and expansion trial for its novel cell therapy candidate, FT839, in patients with autoimmune diseases. This intravenously administered therapy is design... -
ARPA-H Invests $125M in On-Demand RNA Medicine Manufacturing; ArsenalBio Pivots to In Vivo CAR T Cell Therapies for Solid Tumors
The Pharma Letter USA Overview ARPA-H has committed $125 million to five teams developing on-demand manufacturing capabilities for RNA medicines, a strategic investment to enhance rapid pharmaceutical response to future public health cri... -
Fate Therapeutics Initiates Phase 2 RECLAIM-LN Trial for Lupus Nephritis with Off-the-Shelf iPSC-Derived CD19 CAR-T Cell Therapy FT819
Seeking Alpha USA Overview Fate Therapeutics has commenced its Phase 2 RECLAIM-LN trial for lupus nephritis, administering the first patient with its iPSC-derived CD19 CAR-T cell therapy, FT819, in an outpatient setting. This marks a sig... -
August Sees Mixed Fortunes in Gene Therapy: Ultragenyx’s GSDIa Therapy Gets Accelerated Approval, Epicrispr Raises $90M, While REGENXBIO’s RGX-121 Faces Clinical Hold
Vertex AI Search USA Overview August 2026 brought both significant advancements and setbacks in the next-generation therapeutics space. Ultragenyx secured FDA accelerated approval for GENGLYCOS, a gene therapy for GSDIa, offering a new o...
