XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting. They are due to enrol 45 apiece, and are scheduled to readout next year.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Delveinsight

FEBRUARY 11, 2021

The first Fabry disease patient treated with Avrobio’s Plato gene therapy platform in a phase 2 trial has undergone the complete clearance of toxic kidney substrate. Late in 2019, a Fabry patient in phase 2 clinical trial of ex vivo lentiviral gene therapy AVR-RD-01 became the first patient dosed using Plato.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

The Pharma Data

AUGUST 3, 2021

While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Gene 52

XTalks

OCTOBER 1, 2020

Leaving academia for industry is a route that academic scientists and professors did not traditionally pursue in the past due to perceived differences in skillsets, experiences and even associated stigmas. However, this has since changed, and now, transitioning between academia and industry has become more commonplace. Key Moments.

Scientist 52

Scientist In-Vitro Research Life Science 52