InGeNA welcomes our new associate member Bespoke Genetic Medicines Inc

InGeNA is delighted to welcome our new Associate Member: Bespoke Genetic Medicines Inc.

Where Traditional pharmaceutical companies develop one drug for millions of patients, at Bespoke Genetic Medicines, therapies are designed and tailored to each patient’s unique genetic error, one at a time, using modular gene therapy platforms.

Bespoke Genetic Medicines Inc., believes no disease is too rare and no patient group is too few to deserve a cure. There are up to 1,700 distinct rare genetic neurological conditions with no available treatment. Individuals with ultra-rare diseases and their families are too often left behind because there is no profitable market opportunity for conditions affecting fewer than 100 patients globally.

A priority research focus  is a group of corticospinal motor neuron diseases called spastic paraplegias. Over 90 genes have been identified to drive the neurodegenerative conditions, which affect vision, speech, swallowing, gross and fine motor skills, motor function, balance, gait and mobility.