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Executive / Senior Director, Gene Therapy, Research

Location:San Rafael, California

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Overview

With accelerated review underway in the United States and Europe for our Hemophilia A drug, a second gene therapy program entering the clinic for PKU patients, and a third program in late research for Hereditary Angioedema, BioMarin continues to pioneer innovative approaches to treating rare diseases. Our commitment to gene therapy is further underscored by having developed the first and largest gene therapy manufacturing facility in the world. The scientific possibilities and the hope we offer to patients with rare diseases is enormous.

BioMarin is a midsize biotechnology company with a market cap of approximately 18 billion. Over the course of our 22 year history we have successfully launched 7 drugs for the treatment of rare diseases. Our success is due to our scientific rigor and our ability to leverage multiple therapeutic modalities. BioMarin is based in the San Francisco Bay Area and offers a comprehensive relocation program.

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Success Profile

Check out the traits we’re looking for and see if you have the right mix.

  • Decision Making
  • Influence
  • Collaboration
  • Agility
  • Leadership
  • Intellectual Curiosity

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Our CULTURE

  • Patient Focused
  • Results Oriented
  • Pioneering Science
  • Cross Functional Collaboration
  • Willing to try new ways to look at problems

Benefits

  • Healthcare

  • Medical

  • Dental

  • Vision

  • Health & Wellness Programs

  • 401(k)/Retirement plans

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" BioMarin has this, in their own DNA, an approach: they only work on projects for which they know the underlying biology."
Lon C., Chief Scientific Officer
"We’ve got to understand: What are the problems other people are trying to solve? And do we have a better way of fundamentally doing that?"
Brinda B, GVP, Corporate and Business Development

Responsibilities

BioMarin is the world leader in delivering therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We target diseases that lack effective therapies and affect relatively small numbers of patients, many of whom are children. These conditions are often inherited, difficult to diagnose, progressively debilitating and have few, if any, treatment options. BioMarin will continue to focus on advancing therapies that are the first or best of their kind.

BioMarin’s Research & Development group is responsible for everything from research and discovery to post-market clinical development. Research & Development involves all bench and clinical research and the associated groups that support those endeavors. Our teams work on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with rare diseases. Come join our team and make a meaningful impact on patients’ lives.

SUMMARY:

Reporting to the Vice President, Gene Therapy, the Executive Director will build and lead dynamic teams of scientists in the Genetics/Gene Therapy department to design, evaluate, and develop a portfolio of transformative genetic medicines for serious, rare diseases. The Executive Director, Gene Therapy will have demonstrated thought leadership, strong team-building skills, technical proficiency, scientific creativity, multiple successful regulatory interactions, and a history of bringing exciting, value-creating programs from preclinical POC to human clinical trials. The successful candidate will have the leadership and operational skills to execute against company goals, while also growing and developing others within the department, and cultivating innovative, “out-of-the-box” ideas that build on the core competencies of the organization.

 KEY RESPONSIBILITIES:

  • Manage, develop, and grow the gene therapy scientific team to identify new and support existing non-clinical development programs
  • Work with other departments to develop study plans and timelines; mentor team to ensure all projects are appropriately prioritized and key goals are met on time
  • Assist due diligence efforts, audits, monitors, landscape analyses, and site visits
  • Lead team to write, review and/or finalize study reports, SOPs, and appropriate sections of regulatory filings
  • Work effectively within the senior, cross-functional leadership team, to achieve corporate and program goals; collaborate and build strong internal network to leverage technical leadership
  • Efficiently communicate project status and risks; present data and strategies adequately to internal and external stakeholders, including investors and regulatory agencies
  • Work collaboratively with senior management, clinical, external advisors and professional advocacy groups, IP counsel, and others to maximize the potential of the organization
  • Contribute to publication efforts around the company’s novel vector candidates, technologies, tools and scientific insights

PROFESSIONAL EXPERIENCE / QUALIFICATIONS:

  • A PhD in pertinent life sciences or equivalent is required with a demonstrated track record of building and leading teams
  • 10+ years’ relevant industry experience, with biotechnology and/or pharmaceutical companies
  • Experience and direct responsibility with viral vectors (AAV or other vector systems) is strongly preferred
  • Excellent understanding of both scientific and regulatory requirements related to pharmaceutical gene therapy development, manufacturing and specifications is required
  • Experience working closely with process development/manufacturing.
  • Extensive experience with animal models used in biology research and development
  • Understands Good Laboratory Practices; proficient in use of ELN and safety/quality systems
  • Ability to work in interdisciplinary teams and interact efficiently at the highest levels of senior management
  • Excellent presentation skills are required; ability to convey scientific excitement to multiple stakeholders
  • Experience in a cross-functional, global environment, where interactions with other, equally talented individuals requires technical expertise, negotiating skills and diplomacy

We are an equal opportunity employer and all qualified applicants will receive consideration for employment without regard to race, color, religion, sex, national origin, disability status, protected veteran status, or any other characteristic protected by law.

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About our location

San Rafael

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Our Benefits

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Meet Our Team

From internationally renowned scientists to patient advocates, BioMarin Hemophilia has brought together the right people.

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In the News

BioMarin has entered into a preclinical collaboration and license agreement with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies.

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