Medical Director, Translational Medicine

Working from home
Sep 29, 2020
Required Education
Bachelors Degree
Position Type
Full time


Ready to join a team committed to developing and commercializing gene therapies for patients and families devastated by rare neurological genetic diseases? Since 2013, Novartis Gene Therapies (formerly AveXis) has had one focus: bringing change to those devastated by genetic diseases.

The Novartis Gene Therapies culture embraces this mission. As a compassionate and dedicated team, we are enthusiastic about the science behind our work and finding answers to difficult questions. We are dedicated to communities affected by rare diseases, and these patients and families are the motivation for everything we do.

We have built a team with exceptional depth of experience, unified by a common vision; to develop gene therapies with the potential to positively impact the lives of the patients and families devastated by rare and life-threatening neurological genetic diseases. Though we are proud of what we have achieved to date, we remain relentlessly focused on making that vision a reality.

Novartis Gene Therapies is seeking a Medical Director who will, as part of the Translational Medicine team be responsible for the successful translation of gene therapy products from the pre-clinical proof of concept stage in R&D to first-in human trials. This includes ensuring trial readiness with regards to the optimization of natural history data-set, bio-markers and clinical outcomes. This also includes stakeholder engagement with clinicians, researchers and patient groups, as well as the design of potentially label-enabling first-inhumantrials. Building on Novartis Gene Therapies gene replacement therapy technology platform, the Translational Medicine team drives the company's platform to accelerate the development of novel treatments for patients suffering from rare and life-threatening neurological genetic diseases.The current focus of the role is on the company's pipeline programs including AVXS-201 for Rett syndrome and AVXS-301 for genetic amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene. It is anticipated that there will be 2 - 5 gene therapy programs in the clinic each year.



  • Ensure milestone-gated Investigational New Drug (IND) submissions are planned and executed across the different modules building on FDA guidance relevant to gene therapy and rare diseases.
  • Work as part of cross-functional teams and serve as the primary Translational Medicine interface to peers in research and development, clinical development, clinical operations, regulatory, CMC, project management, patient advocacy, commercial and business development functions.
  • Engage early with patients, families, patient advocacy groups, researchers and clinicians to better understand disease burden, unmet needs, meaningful endpoints, acceptable and feasible clinical development strategies, and opportunities to partner towards the shared goal of finding better treatments.
  • Optimize trial readiness in any given indication by pro-actively identifying and evaluating available tools and data-sets, and by strategically fostering research to fill critical gapsin natural history datasets, registries, clinical outcomes, biomarkers, pathways to rapid genetic diagnosis, observational study protocols and standard-of-care guidelines.
  • Provide strategic input to maximize platform efficiency by building a platform that shares approaches between programs, including optimized delivery, formulation, monitoring and managing immune responses, IND submission processes, and innovative trial designs for rare genetic disease.
  • Maintain scientific awareness regarding advances in indications of interest, regulatory awareness regarding new guidance and rules, and industry awareness regarding trends and competition through literature, conferences and stakeholder interactions.
  • Serve as scientific point of reference, ensuring that assigned Translational Medicine programs are firmly grounded in the latest research, and that internal and external communications are consistent with this standard.




  • Proven ability to thrive in a fast-paced, innovative environment while remaining flexible, proactive, resourceful and efficient.
  • Ability to excel in a multidisciplinary environment as an integral leader and team member across several project teams.
  • Ability to analyze complex issues to develop relevant and realistic plans, programs and recommendations.
  • Medical degree and 2-5 years of translational or clinical research experience are required.
  • Background and interest in relevant science and technology, as well bio/pharmaceutical industry or IND experience are preferred.
  • Knowledge of neurology or pediatric neurology, genetics and/or rare diseases is strongly preferred.
  • Demonstrated excellence in translational, clinical research, drug development, and/or regulatory issues are desirable.
  • True care and passion for improving the lives of people living with rare diseases.
  • Diplomacy and professionalism, able to command respect from peers and subordinates alike, capable of highly-independent work as well as being a team player and role model.
  • Excellent verbal and written communication skills, allowing for an open and effective dialogue throughout the company, and for effectively representing the company externally.
  • Strong problem-solving ability, and attention to timelines, detail and quality are critical to success.
  • Highly motivated and results-oriented individual with the flexibility and creativity to excel in and contribute to multiple projects simultaneously in a rapidly growing company.
  • Must have the passion and ability to forge and nurture key partnerships and collaborations with the external scientific community, including academia, governmental organizations, nonprofit organizations and CROs.
  • Travel is estimated to be 30%, most being domestic and some international.


The level of this position will be based on the final candidate's qualifications.

Please note this job description is not designed to cover or contain a comprehensive listing of activities, duties or responsibilities that are required of the employee for this job. Duties, responsibilities and activities may change at any time with or without notice.

Novartis Gene Therapies is committed to creating a diverse environment and is proud to be an equal opportunity employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, national origin, genetics, disability, age, sexual orientation or veteran status.