The FDA’s review of Regenxbio’s gene therapy for Hunter syndrome intensifies focus on biomarker validation in accelerated approvals, balancing innovation…
How FDA’s CRISPR approval improves sickle cell disease treatment outcomes
In 2025, medical breakthroughs like CRISPR gene therapies and new cancer immunotherapies show high efficacy in clinical trials, driving regulatory…
8.2% HbF increase with pociredir shows oral sickle cell therapy potential
Fulcrum Therapeutics presented Phase 2 data at ASH 2023 showing pociredir increased fetal hemoglobin by 8.2% in sickle cell patients,…
What FDA’s CRISPR approvals mean for rare disease economics
The FDA’s 2023 approval of CRISPR therapies for sickle cell disease and beta-thalassemia highlights clinical benefits like reduced crises and…
FDA Unveils New Pathway for Personalized Gene-Editing Therapies
The FDA’s new regulatory framework for personalized gene-editing therapies, leveraging AI and data analytics, aims to scale treatments for ultra-rare…
MeiraGTx and Hologen partner to integrate generative AI in Parkinson’s gene therapy trial
MeiraGTx and Hologen have announced a $230 million partnership to use generative AI in optimizing clinical trials and manufacturing for…