Global Medical Director LC FAOD and MPS
UltraGenix PharmaceuticalUnited States - RemotePosted 13 March 2026
Job Description
Why Join Us?
Be a hero for our rare disease patients
At Ultragenyx, we fundamentally believe that taking real impactful action to care for the needs of patients and our people is always the right thing to do. To achieve this goal, our vision is to lead the future of rare disease medicine. For us, this means going where other biopharma companies won’t go – challenging the status quo and creating a new model that advances our field so more patients and caregivers can benefit from life-changing treatments. We do this by following the science, applying a novel rapid development approach, making innovative medicines at fair and reasonable prices, and creating a collaborative ecosystem to reach patients in ways that are most meaningful for them.
Our commitment and care for patients extends to our people, so culture is an essential cornerstone for Ultragenyx. We remain continuously focused on creating a supportive and inclusive environment of profound learning and growth – so employees can thrive in all areas of their lives, in and outside of work. Ultimately, we want to be an organization where we would be proud for our family, friends and children to work.
If you want to have a meaningful impact, do the best work of your career, and grow a lot, both professionally and personally, come join our team .
Position Summary:
ultra
focused – Work together to fearlessly uncover new possibilities
Ultragenyx is seeking a n experienced
physician leader with
a
passion
for rare and ultra - rare
diseases . Reporting to the
Global Medical Lead , the Director will be a key contributor to shaping
the
strategy and
executing
the medical affairs plans for
LC-FAOD, MPS
VII
and MPS IIIA .
This role
offers
a
unique opportunity to be part of
highly collaborative
cross- functional
teams
working on
both
approved therapies ( Dojolvi
and
Mepsevii ) ,
a nd an
investigational
AAV gene therapy (UX111)
as we prepare for future potential launches.
Work Model:
Remote: Officially documented as working full-time from home, with travel to Ultragenyx's offices or other locations on occasion as needed.
Responsibilities:
Provide strategic medical and scientific
leadership
to
LC‑FAOD ( Dojolvi ) and the MPS programs (MPS VII
and
MPS IIIA)
to support the development and execution of the global medical plans.
Contribute to the development
and execution
of
the annual
Integrated Evidence Generation Plans.
Provide medical
expertise
and leadership to
Real‑World Evidence initiatives
(e.g .,
ISTs, collaborative research) and the
Disease Monitoring Program s
for LC
‑
FAOD and MPS
focused on data
generation and
publication
activities.
Act as a subject matter expert in assigned therapeutic areas, guiding
and contributing to
the execution of
key
global
medical affairs activities including advisory boards, medical education
initiatives
and
congress activities.
Lead robust
scientific engagement
and education,
maintaining
s trong relationships with external stakeholders to generate insight s
and advance
scientific understanding of
assigned programs .
Provide scientific and medical leadership as needed for publication
and medical
communication
material
development .
S erve as
a
medical reviewer for promotional materials and non-promotional scientific materia ls
ensuring
accuracy,
balance,
strategic
alignment
and
compliance
with regulatory
requirement s .
Provide
s ubject matter
expertise
to develop and
deliver
training
across the organization.
C ollaborate
cross‑functionally with Clinical Development, Commercial, Regulatory, HEOR,
Biostatistics,
Patient Advocacy , Regional Medical
Teams
and others
to achieve program goals.
Requirements:
Medical Doctor (MD
or
DO )
required .
Clinical
expertise
in
the management of patients with
inherited metabolic
and/ or neurodegenerative rare diseases
strongly
preferred .
At least 2 years of experie ... (truncated, view full listing at source)
Apply Now
Direct link to company career page
More jobs at UltraGenix Pharmaceutical
See all →Sr. Director, Global Regulatory Affairs Labeling
United States - Remote · 13 March 2026
Sr Director, Business Operations, EMEA
Basel, Switzerland; Greater London, United Kingdom · 12 March 2026
Executive Director, Business Development
Somerville, MA · 12 March 2026
Co-op, GMP Facilities
Bedford, MA · 12 March 2026
More Express jobs
See all →Senior Product Designer - Post Purchase
Stubhub Inc · Los Angeles, California, United States
Senior Product Designer - Post Purchase
Stubhub Inc · New York, New York, United States
Banking Research - Associate Director (NY)
Kroll Bond Rating Agency · New York, New York
CMBS Surveillance - Analyst (PA)
Kroll Bond Rating Agency · Dresher, Pennsylvania