Imagine a baby boy who suddenly starts to loose vision.He does not feel any pain only a cloudy spot in the visual field.The spots grow with time leading to complete blindness. Deficit in knowledge delays the diagnosis of this rare disease.There is currently no cure for his condition.He is confused and scared.Would you help.
Since January I have been successfully building and strengthening collaboration between global stakeholders from National Institutes of Health (NIH), FDA, EMA, National Organization for Rare Disorders(NORD), industry and research institutions to accelerate translation from discovery to health benefits of patients with rare disorders.
To support development of biotechnology startup companies with a pipeline of drug products characterized by the highest probability of success relevant to the rare eye diseases, I have identified and initiated relationships with investors, market analysts,CEOs and founders, National Association of Corporate Directors(NACD), and a number of chapters of Women in Bio(WIB) organizations.
To integrate patient-centred research with scientific expertise, I lead in the proactive establishment of communication with Leber’s Hereditary Optic Neuropathy(LHON) society, Leber Congenital Amaurosis(LCA), Foundation Fighting Blindness, advocacy groups,100000 Genome Project representatives, and NCATS.
I am involved in raising public awareness and education on the use of cutting-edge technologies to develop treatments for diseases associated with mutations in genes.By the use of oligonucleotide therapeutic sequences, we help to replace malfunctioning genes with normal functional genes, allowing a patient’s own body to produce proteins to treat the debilitating illness.
Apart from the above I volunteer my time to participate in public meetings with international community regarding the underlying principles of governance for human gene editing and provide a support to fundraising bodies as well as charity events.