Gene therapy: Making Rare Disease Therapies Less Rare

While individually uncommon, rare diseases collectively impact the lives of over 300 million people worldwide. Many rare conditions are chronic, progressive, and life-threatening with limited treatment options available. Advances in gene therapy provide hope to actual cures and could revolutionize treatment prospects for patients with rare genetic diseases. 

This infographic shows how we can unlock the full potential of gene therapies for patients with rare diseases.

More than 70% of rare diseases are known to be caused by a single mutation, which makes them good candidates for gene therapy. Several gene therapies for rare diseases have been approved already and many more are in the pipeline. 

Examples of approved gene therapies include Luxturna^® for the treatment of a type of inherited vision loss known as Leber congenital amaurosis; Zolgensma^® for the neuromuscular disorder spinal muscular atrophy; and Zynteglo^® for the treatment of the blood disorder beta-thalassemia.

However, to really make gene therapy a more widely available treatment option, several challenges need to be overcome.

Due to the novelty of this field, standardized manufacturing protocols are not available and regulatory guidelines are continuously evolving. At the same time, the growing demand for gene therapies has resulted in a lack of manufacturing capacity. 

Moreover, gene therapies are extremely costly, hampering patient access and causing reimbursement difficulties with healthcare providers.

This infographic, created in collaboration with Merck, gives a detailed overview of the challenges facing gene therapy development for rare diseases. It shows how biomanufacturing experts like Merck can reduce costs and accelerate the development of more accessible gene therapies.

This will have an immense impact on the future of medicine and the millions of patients suffering from rare diseases. 

You can download a free version of the infographic here!

To learn how Merck is bringing gene therapy to life, visit MerckMillipore.com/genetherapy!

Author & Designer: Lidwien Veugen