Pharmaceutical Technology

SEPTEMBER 30, 2022

Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

Gene Therapy 289

Gene Therapy FDA Approval Gene Genetic Disease 289

XTalks

SEPTEMBER 27, 2022

CALD is a rare, progressive neurodegenerative disease that mainly affects young boys. The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. Skysona was granted Orphan Drug designation, Rare Pediatric Disease designation and Breakthrough Therapy designation. Related: Bluebird’s $2.8M

FDA Approval 52

FDA Approval Gene Therapy Drugs Gene 52

Delveinsight

FEBRUARY 23, 2021

The company develops transformative biologics including vaccines and therapeutics for the world’s most debilitating diseases. The company plans to use the capital to expand its pipeline of protein-based vaccines and biologic cancer therapies utilizing its innovative and proprietary Trimer-Tag technology platform.

In-Vitro 52

In-Vitro Vaccination Vaccine Licensing 52

XTalks

OCTOBER 12, 2022

Features of blood perfusion in the brain detected by MRI now have clinical applications in central nervous system (CNS) cancers, neurodegenerative disease, cerebrovascular disease and demyelinating disease. An overview of the ASL MR perfusion technique to obtain cerebral blood flow (CBF) values.

Clinical Trials 93

Clinical Trials Clinical Trials Trials Drugs 93