VP, Global Rare Disease Franchise

Since our founding in 2009, the company has pioneered what’s possible in gene therapy. Today, we are advancing a pipeline of gene therapies designed to improve the lives of patients facing serious rare and retinal diseases and preparing to bring innovative new medicines to patients.

Our work is fueled by purpose and urgency because every day matters to patients and their families. At the company, you’ll find meaningful opportunities to grow, collaborate, and contribute to the advancement of potentially transformative gene therapies. Together, our talented team is working to make a meaningful difference for patients and their families.

What we do matters.

The opportunity

As VP, Global Rare Disease Franchise, you’ll lead the overall strategy and commercialization of the company’s rare disease portfolio. With full accountability for business performance and P&L, you’ll lead the commercial strategy and launch of RGX-202 while shaping the growth and evolution of our rare disease franchise.  Reporting to the Chief Commercial Officer, you'll lead the Rare Disease Franchise and Marketing organization, partnering cross-functionally to turn strategy into execution and deliver meaningful outcomes for patients, providers, and the company. As a member of the commercial leadership team, you’ll also help shape the company’s global growth strategy and strengthen our leadership in rare disease.

What you’ll be doing

• Own the overall rare disease franchise strategy, business performance, and P&L, with full accountability for revenue, profitability, forecasting, budget, resource allocation, and achievement of commercial objectives, including:

• Lead the development and execution of the overall rare disease franchise strategy and commercialization roadmap for RGX-202, and all future programs and products

• Establish clear business objectives, performance metrics, and operating plans to maximize franchise growth and long-term value

• Own the annual operating plan, revenue forecast, expense budget, and resource allocation for the rare disease franchise, ensuring disciplined investment and accountability for financial performance

• Regularly assess market, competitive, customer, and business performance and adjust franchise strategy and investments to maximize growth and P&L performance

• Partner closely with Finance and executive leadership to ensure financial rigor, transparency, and accountability across the franchise

• Lead the commercial strategy for rare disease product launches, including:

• Develop and oversee the integrated U.S. launch strategy, roadmap, and market entry plan for RGX-202 in partnership with key functional leaders.

• Establish launch objectives, readiness milestones, performance measures, and operating plans, ensuring organizational alignment and accountability for execution.

• Identify and address critical launch barriers across market development, patient identification and referral, treatment-center readiness, access and reimbursement, and patient onboarding, in partnership with functional leaders.

• Manage the the company rare disease franchise’s performance and growth, with full P&L responsibility, including accountability for:

• Revenue and demand generation

• Commercial operating expenses and resource allocation

• Annual business planning, forecasting, and performance management

• Investment decisions and prioritization of resources across the franchise

• Identifying and addressing risks and opportunities that impact financial and business performance

• Delivering sustainable growth and maximizing the long-term value of the rare disease franchise

• Partner with the the company executive and senior leadership team to align on strategy and plans for global commercialization, including:

• Translate enterprise strategy into clear franchise priorities, operating plans, and measurable business outcomes

• Provide senior leadership with a clear view of market dynamics, business performance, risks, opportunities, and resource requirements

• Drive cross-functional alignment and accountability for critical commercial and franchise initiatives

• Serve as a senior member of the commercial and management teams of the company and contribute to its overall growth strategy, including:

• Bring an enterprise perspective to strategic decisions regarding commercialization, portfolio prioritization, business development, and resource allocation

• Represent the voice of the rare disease community within broader company strategy and decision-making

• Partner with executive leadership to identify opportunities to strengthen the company’s commercial capabilities and long-term growth

• Collaborate with partners in R&D to be responsible for the rare neuromuscular and muscle Franchise and lead the cross-functional Leadership and Strategy Plans for this therapeutic area for the company, including:

• Align commercial, clinical, medical, regulatory, market access, and operational priorities across the franchise

• Inform pipeline strategy and development priorities based on evolving market, customer, patient, and competitive insights

• Ensure appropriate commercial and market development input into development plans and lifecycle strategies

• Elevate the company’s external leadership in Rare diseases through industry thought leadership; symposia and key associations.

• Ability to travel as required.

• It is imperative that the company employees embody our core values by working collaboratively, building strong relationships and using clear communication to meet shared objectives.

What we’re looking for

We set our employees up for success.  To be successful in this role and help us achieve our goals, we are looking for someone with the following skills and qualifications:

• Advanced degree in relevant life sciences or finance (Ph.D., MBA, MS) is required

• 15+ years progressive commercial leadership experience in biotechnology or pharmaceuticals, including leadership of substantial commercial teams and/or businesses.

• Demonstrated experience successfully leading a product launch and/or holding significant leadership accountability for launch strategy, readiness, and execution.

• Demonstrated ability to translate commercial and franchise strategy into clear business priorities and performance expectations, and partner with field and functional leaders to drive execution.

• Strong business and financial acumen, including meaningful responsibility for revenue, operating budgets, forecasting, resource allocation, and investment decisions.

• Demonstrated experience operating effectively in highly cross-functional environments and influencing senior leaders across Commercial, Medical, R&D, Market Access, Finance, and other functions.

• Demonstrated ability to build organizations and capabilities, develop high-performing teams, and lead effectively through ambiguity and change.

Preferred experience includes

• Significant rare disease commercial leadership experience.

• Experience commercializing therapies with complex patient journeys, concentrated treatment-center models, and significant institutional access and reimbursement requirements.

• Experience with gene therapy, one-time therapies, biologics, and/or other highly complex specialty treatment models.

• Experience contributing to lifecycle, pipeline, portfolio, or business-development decisions from a commercial perspective.

Why should you apply?

By joining the company, you will have the opportunity to be a part of a growing company, with an incredible team, who is passionate about developing novel AAV gene therapies for patients in need.

We offer a comprehensive and competitive benefit & compensation package which includes market-competitive salaries, an annual bonus program, education assistance, retirement plan with employer match, stock options at all levels, summer hours and more! The estimated compensation range for this role is $289,000 to $410,000 annually. The actual salary offered to the final candidate depends on a number of factors such as relevant work experience, skills, education, and years of experience.

In addition, professional development is important to us.  By joining our team, you’ll have the opportunity to be exposed to challenging projects and have access to development resources to help you grow personally and professionally.