Year: 2014-15

Company: Alnylam

Liaison(s): Chris Larsen Alfica Sehgal

Alnylam Pharmaceuticals is a leading RNAi therapeutic
innovator whose goal, announced in 2011, and has
been the “Alnylam 5 x 15.” The “Alnylam 5×15”
goal is to develop five RNAi products for genetically
defined diseases into advanced clinical phases by
2015. Currently, Alnylam is focused on the continuing
development of these investigational drugs. In early
2015, Alnylam announced the evolution of its strategy
with a focus on development and commercialization
of investigational RNAi therapeutics in 3 strategic
therapeutic areas – genetic medicine, cardio-metabolic
medicine, and hepatic infectious disease.
Alnylam is interested in applying its platform
technology to a rare, often misdiagnosed, liver disease
project independent of its “Alnylam 5×15” initiative.
The Alnylam TMP team was tasked with investigating
disease demographics, while at the same time
increasing awareness among patients and physicians.
By understanding patient demographics, including
prevalence of this disease, the team will contribute to
a feasibility study for the development of Alnylam’s
potential drug.
To do this, the Alnylam TMP team first created an
independent patient advocacy non-profit organization
(NPO) to help raise disease awareness, collect
epidemiological data, and present any opportunities
for patients to participate in clinical trials and basic
research. As a “face” for the NPO, the team created
a website which serves as both an information portal
and a database for patients who choose to participate.
Included in this website are informational videos and
patient stories. The NPO is an entity independent of
Alnylam and KGI. To gain an understanding of the
disease demographics, the team surveyed nephrologists,
reached out to all authors who have published on the
disease, and interviewed several patients. To increase
disease awareness, the team created brochures for
patients and physicians, and maintained accounts
on social media to update interested parties. Upon
completion of the project, Alnylam will be able
to determine whether it will continue pursuing
development of an investigational agent for the
potential treatment of this disease. The NPO will
continue to serve as a space to support the community
of patients, physicians, and researchers who are
interested in furthering research for this currently
untreatable disease.