Job ID 84176
n-Lorem is a non-profit foundation founded in 2020 committed to discovering and providing experimental treatments for free for life for patients who have genetic diseases that affect only one person or at most 30 patients worldwide. The rarity of the mutations identified in these patients, identified as nano-rare diseases, resulting in a difficult multi-year diagnostic journey, which often abruptly ends with the fact that no treatments are available, leaving them feeling hopeless. Though it seems an impossible task we are changing that! The reason we can do the seemingly impossible, to create mutation-directed therapies for our patients, is due not only to the efficiency and versatility of antisense oligonucleotide (ASO) technology as a drug discovery platform but also a strong network of partners. Our unique ability to meet the needs of our patients is grounded in 30 years of prior experience including extensive molecular mechanism screening, and safety expertise. Importantly, our work is aligned with four draft guidance documents released by the FDA over the past year on individualized antisense oligonucleotides- the only technology for which there is specific FDA guidance. We are in an exciting time of significant growth while we establish and scale our infrastructure- and know that this endeavor is only possible with a strong team!