Alnylam's Nobel-prize-winning technology and massive intellectual property estate could allow the firm to create an entirely new class of therapeutics for tough-to-treat diseases.
RNAi therapeutics have the potential to gain access to well-studied targets in the cell that are currently deemed "undruggable," and Alnylam could capture this low-hanging fruit in a wide range of diseases.
Alnylam's most advanced drug candidate, patisiran, is now in Phase III trials, and the firm's GalNAc-conjugate delivery technology appears to allow for safe, targeted, subcutaneous administration. Read more
No RNAi therapeutics have been approved by the FDA, and given the novelty of this field of drug development, the regulatory road ahead could be a long one.
While Alnylam is targeting several rare genetic disorders--including amyloidosis, hemophilia, and complement-mediated diseases--entrenched competitors are also producing next-generation treatments.
Alnylam's clinical data is heavily centered on its amyloidosis and PCSK9 programs, and while preclinical data is intriguing, other promising candidates are just entering development. Read more
Despite Alnylam's history of diluting shareholders and the early-stage nature of the firm's pipeline, we think its highly qualified management and board bring the firm to a standard level of stewardship. John Maraganore had senior management-level experience Read more
Alnylam Pharmaceuticals is a leader in the study of RNA interference and is attempting to harness this mechanism to create a new class of drugs. Alnylam has partnered several Read more