The Institute for Gene Therapies is Working to Improve Policy and Access for Charcot-Marie-Tooth Disease and Others

The Institute for Gene Therapies (IGT) is a new development, aimed at improving the speed of development and accessibility of gene therapies.

Gene Therapy

Many rare diseases are caused by a singular gene mutation, making it feasible to effectively cure the condition with gene therapy. This involves the modifying of genes that aren’t currently functioning or inserting genes that don’t exist where they should. This treatment has curative potential. As a one-time procedure, it could significantly improve the quality of life of patients.

Until now, many rare disease patients have relied on symptomatic treatment.

Gene therapy is no longer a far off dream. Some therapies have already received FDA approval. Numerous trials are underway to continue this trend. These include studies for hemophilia A, hemophilia B, neuromuscular diseases, various forms of cancer, Charcot-Marie-Tooth disease, and others. 


Charcot-Marie-Tooth (CMT) disease has no cure, but researchers are hopeful that gene therapy could be an option for this condition.

CMT has various types and subtypes, meaning different genes could be administered to patients depending on type. These genes are administered using a virus which is harmless.

Preclinical programs and clinical programs are already underway.


The IGT is made up of a Patient Advisory Council, scientists, and leaders in the gene therapy industry. It’s primary goal is to improve the process of how gene therapy is regulated. The problem is that healthcare regulations were formed without gene therapy in mind because it didn’t exist. That means for gene therapy approval is slow and reimbursement is difficult. IGT hopes to improve this by educating stakeholders.

By changing policy, we will able to accelerate development. However, we don’t just need to educate stakeholders; we need to encourage them to collaborate with one another so that novel discoveries can come about faster. Further, the IGT wants to raise awareness for the method, encouraging investors to keep their eye on gene therapy. 

You can read more about the IGT here.

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