What is a Gene?

Gene | Zegen Therapeutics

Genes are units of DNA inherited from one’s parents that store information and instructions on how to make specific proteins. These proteins express an  individual’s hereditary characteristics and control the growth and development of the body’s cells. Across the species, humans share about 99% of the same genes  with only about 1% variance. While genetic variance is normal, genetic mutation can be harmful, leading to serious health impacts. When a gene is mutated, it may  not produce normal protein levels, it may produce a mutated protein lacking normal function, or no protein at all.

What is a Genetic Disease?

A genetic disease is caused by one or more mutations in the DNA. Some diseases can be identified by a single mutated gene and others are associated with multiple gene mutations. Genetic diseases are usually inherited, but can also be acquired through genes mutating randomly or triggered by lifestyle and environmental fac

What is Gene Therapy?

Current efficacious gene therapies target diseases with a single gene mutation.  Many of these therapies use a specialized carrier (vector) to deliver a functioning gene to targeted cells to replace the mutated gene. Once united with the targeted cells, the replacement gene will gradually start to produce the missing or malfunctioning protein.

Asian Scientists Working on Vaccine Creation | Zegen Therapeutics

Clinical

Treatment | Zegen Therapeutics

Treatment and results

What is The Gene-Therapy Drug Development Process?

To develop a gene therapy treatment, scientists discover what gene(s) are missing, mutated or defective, and if corrected could cure disease. Next, researchers find the most effective delivery vehicle to carry and incorporate the new gene into the patient and to the target cells. Before any gene therapy is used to treat humans, the therapy undergoes rigorous testing and must satisfy strict safety, quality, efficacy and ethical requirements.

Production | Zegen Therapeutics

Production

Preclinical | Zegen Therapeutics

Preclinical

What is the gene-therapy drug development process?

To develop a gene therapy treatment, scientists discover what gene(s) are missing, mutated or defective, and if corrected could cure disease. Next, researchers find the most effective delivery vehicle to carry and incorporate the new gene into the patient and to the target cells. Before any gene therapy is used to treat humans, the therapy undergoes rigorous testing and must satisfy strict safety, quality, efficacy and ethical requirements.

Asian Scientists Working on Vaccine Creation | Zegen Therapeutics

Clinical

Treatment | Zegen Therapeutics

Treatment and results

Production | Zegen Therapeutics

Production

Preclinical | Zegen Therapeutics

Preclinical

Benefits of Gene Therapy

The patient benefits of gene therapy are specific to the genetic disease it is treating. Although we must demonstrate the safety and effectiveness, we believe there are numerous potential benefits of our AAV gene therapy treatment for rare epilepsies:

  • The potential for earlier intervention to stop disease progression before clinical damage
  • A potential reduction in treatment burden.  A onetime treatment as opposed to continuous life-long treatments.

What is Developmental and Epileptic Encephalopathy (DEE)?

DEE are a heterogeneous group of rare neurodevelopmental disorders characterized by

  • Early-onset seizures that are often intractable

  • Electroencephalographic abnormalities

  • Developmental delay or regression
  • In some cases, early death