Medical Director, Translational Medicine

Working from home
Nov 27, 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.

The Medical Director, Translational Medicine is responsible for the successful translation of gene therapy products from the pre-clinical proof of concept stage in Research and Early Development to first-in human trials.

  • Works as a study lead on cross-functional teams on specific pipeline programs to evaluate and ensure trial readiness with regards to natural history data-sets, bio-markers and clinical outcomes.
  • Engages with key stakeholders including clinicians, researchers and patient groups to better understand disease burden, unmet needs, meaningful endpoints, acceptable and feasible clinical development strategies, pathways to rapid diagnosis, as well as opportunities to partner towards the shared goal of finding better treatment.
  • Collaborates with cross functional teams, designs and maintains responsibility for innovative and potentially label-enabling first-in human trials as well as phase 1 and 2 clinical trials, natural history studies and observational studies.
  • Ensures that translational medicine contributions to Investigational New Drug (IND) submissions are planned and executed in a timely manner.
  • Serves as the primary Translational Medicine interface to peers in research, clinical development, clinical operations, regulatory, CMC, project management, and business development.
  • Provides strategic input to the gene therapy platform such as optimized delivery, formulation, monitoring and managing immune responses, IND submission processes, and innovative trial designs for rare genetic disease (e.g., adaptive, seamless Phase 1/2 approaches).
  • Works with research, commercial and business development teams to evaluate internal and external technologies and programs that may be suitable for development, partnership or acquisition by Novartis Gene Therapies.
  • Maintains 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.
  • Serves as a medical and scientific point of reference, ensuring that programs as well as internal and external communications are firmly grounded in the latest research and medical knowledge.
  • Other related job duties as assigned.

  • Medical degree and 3 years of translational or clinical research experience.
  • 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.
  • Background and interest in relevant science and technology, as well bio/pharmaceutical industry or IND experience are preferred.
  • Knowledge of neurology or pediatric neurology, 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 with 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 contract research organizations.
  • Travel estimated at 30%, most 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.