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Sr Director, Core Team Leader - PKU, Gene Therapy

Location:San Rafael, California



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


  • 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


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.

About the Team:

The Core Team Leader Group is part of BioMarin’s Corporate Product Portfolio Development function which reports to the Chief Scientific Strategy Officer.  Product Portfolio Development is responsible for BioMarin’s corporate and product governance, business operating model (core teams and partner teams), portfolio strategy and business financial planning, strategic business insights and Competitive Intelligence. 

About the Program:

BMN 307 is BioMarin’s 2nd gene therapy asset being developed for the treatment of PKU. BioMarin is a world leader in PKU with two approved medicines (Kuvan and Palynziq) and is excited to strengthen its leadership position in PKU by developing an innovative treatment option for patients. Building on its gene therapy experience in Hemophilia A, BioMarin recently initiated the first in human study for BMN 307. BMN 307 has the potential to replace the missing PAH enzyme and reduce elevated Phenylalanine levels in patients which consequently has meaningful clinical impact on their lives.

Core Team Leader (CTL) Position:

CTL is the product champion and leads a team of functional leaders that together develop product strategy which is reviewed and approved by BioMarin governance.  CTL is the primary liaison with executive management and has overall accountability to executive management for the integrated product strategy. In collaboration with core team (CT) members, the CTL ensures progress and execution against plans and as the leader of the CT is the primary contact for internal stakeholders. As a senior leader in the core team leader group, is a role model, mentors peers and contributes to key core team processes and initiatives.


10+ years of experience in the biotechnology/ pharmaceutical industry. Clinical development experience is highly preferred. Rare disease experience is a plus.

2+ years of experience leading a core/cross functional strategy team for an investigational therapy.

The successful candidate will also demonstrate strong matrix leadership ability and professional credibility to influence a highly intelligent and sophisticated audience of colleagues, partners, and external constituents.

This individual must be a self-starter and possess excellent leadership, influencing and communication skills, as they will be applied at all levels, including at the core team, interactions with the executive team, and presentations to internal and external audiences.

General CTL Responsibilities:

  1. Program Strategy

Lead the CT in the generation of a Target Product Profile to maximize the molecule’s potential for patients and for the business

Ensure integrated stage-based product plans that are aligned with program strategy and ensures assumptions and drivers are aligned with plans to inform financial planning

Establish strong partnerships with all functional stakeholders, specifically Medical Affairs, Regulatory and Commercial to ensure approval and launch success.

Ensure potential risks/trade-offs are identified and contingency planning with a solutions’ focused mindset.

  1. Team Effectiveness

Ensure cross functional partnerships and resources are leveraged effectively, and with accountability, to achieve program objectives.

Drive collaboration and transparency across all functions on the CT and challenge team members to be broad and inclusive in their strategy planning.  

Resolve conflicts at the Core Team, provide and receive feedback to develop and maintain a high performing team

III.  Governance and Approvals

Ensure that team recommendations are brought to appropriate governance for decision

Ensure alignment of program strategy/plan/execution with management expectations & priorities

  1. Communication

Single point of contact on program for executive and functional management

Ensures communication of rationale and context for decisions made to team

Leads team in developing a comprehensive internal and external communication plan with team input for important program milestones, key data, etc.

Reviews and agrees to publication plan, reviews publication concepts, presentations and manuscripts

Primary source for information as needed for external communications (press releases, investor related, etc.)

Reviews health authority related documents and ensures consistency with strategic objectives for the program


The successful candidate will ideally have an advanced degree in a scientific discipline (e.g. Pharm D, Ph.D. or M.D.)

B.S. is Life Sciences is a plus.


About our location

San Rafael


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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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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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