Contract Manufacturing, FDA Approval and Genetic Disease

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

JULY 29, 2022

In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). In order to find the specific RNA sequences for a genetic disease, the biotech is using an AI- and machine learning-based approach.

RNA

RNA Gene Expression Gene Gene Therapy

Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

XTalks

MAY 17, 2023

In 2022, the FDA approved four new gene therapies, and more than a handful are looking to get approval this year for diseases including Duchenne muscular dystrophy (DMD), sickle cell disease (SCD) and hemophilia A.

Gene Therapy

Gene Therapy Clinical Trials Clinical Trials Gene

Clinical Research Informer

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Bespoke Gene Therapy Consortium Selects 8 Rare Diseases for Clinical Trial Portfolio

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