logo
  

Ultragenyx Reports Additional Positive Durability Data For DTX401, DTX301 Gene Therapies

Ultragenyx Pharmaceutical Inc. (RARE) reported new long-term durability data from the phase 1/2 studies of DTX401 for Glycogen Storage Disease Type Ia and DTX301 for Ornithine Transcarbamylase deficiency.

The additional longer-term phase 1/2 data with DTX401 for Glycogen Storage Disease Type Ia showed durability of response, with sustained responses lasting up to 3 years since treatment. Across all 12 patients, the mean reduction in daily cornstarch intake was 69.9% ranging from 19-100% when comparing baseline to the most recent visit. DTX401 is an investigational adeno-associated virus type 8 gene therapy.

The additional longer-term phase 1/2 data with DTX301 for Ornithine Transcarbamylase Deficiency showed durable metabolic control and sustained responses. The six patients who previously demonstrated a response remain clinically and metabolically stable, including all three treated at the highest dose. DTX301 is an investigational AAV type 8 gene therapy.

For comments and feedback contact: editorial@rttnews.com

Business News

Editors Pick
MTD Products Inc. is recalling 1,774 units of walk-behind self-propelled lawn mowers for potential risk of fire hazard, a statement by the U.S. Consumer Product Safety Commission (CPSC) showed. The company said the lawn mower can leak fuel when it is stored in the upright (vertical) storage position to pose a fire hazard. Nike Inc. announced its plans to exit the Russian market completely following Russia's ongoing war against Ukraine, reports said. The sportswear major, with more than 100 stores in the country, had suspended operations there in March. Earlier in March, while suspending operations temporarily, the company had said its customers that it couldn't guarantee product shipments. The U.S. Food and Drug Administration unveiled its action plan to advance drug development for rare neurodegenerative diseases, including Amyotrophic Lateral Sclerosis or ALS, in five years. The FDA plans specific actions such as regulatory science initiatives, enhancements to existing programs and new policy initiatives for the purpose. It will use public-private partnerships...
RELATED NEWS
Follow RTT