ToolGen, ANLBIO to jointly develop progeria treatments

South Korea's two biotech startups ToolGen Incorporated and ANLBIO on Tuesday signed a joint research and development agreement for developing progeria treatments.

Progeria, or Hutchinson-Gilford progeria syndrome (HGPS), is a rare autosomal dominant genetic disease affecting 1 in 8 million.

 At approximately nine to 24 months of age, affected children begin to experience profound growth delay and rapid aging. 

It results from a single-letter misspelling in a gene on chromosome 1 that codes for lamin A, a protein that is an essential component of the membrane surrounding the cell’s nucleus. The abnormal lamin A protein produced in HGPS is called progerin. 

Currently, Eiger BioPharmaceuticals’ Zokinvy (Lonafarnib) is the only progeria cure approved by the US Food and Drug Administration (FDA). 

Recently, a study has been published to extend the life of a model mouse with progeria using genetic scissors, a technology that edits a specific gene in DNA, raising the hope of the birth of a progeria treatment.

Through the latest agreement, the two companies will create a treatment by combining ToolGen's gene scissors platform and ANLBIO's adeno-associated virus (AAV) vector platform and evaluate its efficacy in animal models.

"Genetic scissors can cure many diseases, but we will face financial and human resource limits when we try to develop many treatments at the same time. With a good partner, ToolGen wants to expand the pipeline by jointly developing a treatment for progeria, which has been impossible to develop fundamental treatments," said Kim Young-ho, CEO of ToolGen.

ToolGen is a biotech company that conducts research and business in the field of human treatment, animal/plant development, and breeding based on its original patent for CRISPR genetic scissors. 

ANLBIO is a new drug developer with multiple gene therapy drug pipelines targeting degenerative CNS diseases and intractable rare diseases.

Write to Jeong Min Nam at peux@hankyung.com