XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Drug Discovery World

DECEMBER 12, 2023

Context and treatment potential AATD is an inherited, autosomal recessive genetic disorder that is most frequently caused by a single nucleotide variant (SNV) mutation in the SERPINA1 gene, the most common of which is the ‘PiZ’ mutation. I look forward to having access to this therapy as quickly as possible.”

RNA 52

RNA In-Vivo Genotype Genetics 52

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