XTalks

APRIL 24, 2023

Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals. Last year, the FDA approved five novel cellular and gene therapy products with orphan drug designation.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

The Pharma Data

DECEMBER 9, 2020

Vigil’s pipeline strategy is to target these diseases by combining a high-level understanding of microglia physiology, disease genotyping, and patient phenotyping to identify specific genetic variations associated with microglial dysfunction. which will remain a key shareholder. SciNeuro Pharmaceuticals. in a statement. . Libra Therapeutics.

Marketing 52

Marketing In-Vivo Protein In-Vitro 52

Pharmaceutical Technology

AUGUST 18, 2022

0 genotypes. An ex-vivo lentiviral vector (LVV) gene therapy, Zynteglo’s every dose is made by genetically modifying the bone marrow stem cells of the patient to produce functional beta-globin. thalassemia in adult and paediatric patients. Findings showed that 89% of evaluable subjects attained transfusion independence (TI).

Gene Therapy 246

Gene Therapy Gene Genotype Genetics 246

Drug Discovery World

DECEMBER 12, 2023

KRRO-110 is designed to co-opt an endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to repair a pathogenic single nucleotide variant (SNV) on RNA and restore production of normal A1AT. I look forward to having access to this therapy as quickly as possible.”

RNA 52

RNA In-Vivo Genotype Genetics 52