U.S. FDA AWARDS BOTH RARE PEDIATRIC DISEASE AND ORPHAN DRUG DESIGNATIONS TO HG204, A CRISPR RNA-EDITING THERAPY, FOR THE TREATMENT OF MECP2 DUPLICATION SYNDROME

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HUIDAGENE THERAPEUTICS ANNOUNCES FIRST PATIENT DOSED IN MULTINATIONAL PHASE 1/2 TRIAL OF HG004 FOR INHERITED BLINDNESS

The parable of the lost sheep mirrors our journey to find a solution for Duchenne Muscular Dystrophy (DMD), a rare disease. Our global interdisciplinary team is fortunate to have been gifted with Neu REFIX, whose hidden potentials of beneficial immune modulation, we unravelled by extensive preclinical and clinical studies. This gives hope to DMD patients for its potentials in improving their quality of life by halting the disease progress. Like the shepherd when he found the lost sheep, our joy knew no bounds, when we got encouraging outcome in DMD research. Now on receiving orphan drug and rare paediatric disease designations from US FDA, while it’s time to rejoice, we have miles to go until it reaches every deserving patient after an approval, the final goal. We are open to collaborate with likeminded institutes to reach the goal. Further insights have revealed the potentials of Neu REFIX in Psoriasis, auto-immune diseases and in Multiple Sclerosis by gut microbiome reconstitution. (Graphic: Business Wire)

Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy

Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Ellipses EP0031/A400 granted Orphan Drug Designation from the US Food and Drug Administration

European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

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The Department of Health - Abu Dhabi provides gene therapy for Duchenne Muscular Dystrophy for the first time in Abu Dhabi and outside the US

Vertex Announces Approval of First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™, for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) in Kingdom of Saudi Arabia