Director / Senior Director, Rare Disease Clinical Lead

About Maze Therapeutics

Maze is a clinical-stage biopharmaceutical company harnessing the power of human genetics to transform the lives of patients through the development of a pipeline of novel precision medicines for patients with genetically defined diseases. Maze is leveraging Compass™, a proprietary, end-to-end purpose built platform to advance a pipeline of precision medicines across four disease areas - renal, cardiac, neurological and metabolic diseases. Our flexible business model aims to translate our genetic insights into new therapies for patients as possible.

Our People

Maze is comprised of a team of passionate and creative professionals committed to discovering and delivering transformative medicines to patients suffering from both rare and common genetic diseases. We are fostering a culture that encourages vision, initiative and the development of talent. Our supportive work environment inspires creative thinking and freedom of expression, resulting in a stimulating atmosphere where people enjoy coming to work. While we have a passion for advanced science and pride ourselves on excellence in execution, ultimately, everything we do is about patients. 

Our Core Values

Further Together – Our path is paved with challenges, but with resilience and a team-first mentality, we’ll achieve our mission.  

Impact Obsessed – We embrace the bold, take calculated risks, and learn from our mistakes to improve the lives of others. 

Stand True – Our integrity is foundational; it guides us no matter the obstacle. 

Role Description:

Maze is seeking an experienced Director/Senior Medical Director who will provide strategic and tactical medical leadership and oversight of clinical trials in rare diseases.  This position will serve as the clinical lead for the GYS1 program in Pompe disease as well as support broader clinical development efforts for rare metabolic diseases across the pipeline.  This individual will be accountable for setting the mid and late-stage development strategy and driving clinical execution of a small molecule targeting GYS1 to support the first oral therapy for Pompe disease and transforming the treatment landscape in glycogen storage diseases.   This position will also support execution of early phase studies of other small molecules for rare metabolic diseases and play a role in business development and licensing decisions. This individual must have the ability to work in a fast-paced, team-oriented, start-up environment to oversee all aspects strategic planning as well as oversight of nonclinical activities. The successful candidate will serve in a highly visible, cross-functional leadership role requiring an ability to be a liaison with senior management, functional leaders, and technical leaders in drug discovery, translational science, and clinical development.

The position will report to the Chief Medical Officer and will be a key member of the development leadership team.

Your Impact Includes:

• Lead clinical development of all rare metabolic disease assets by defining the clinical and scientific development strategy to clinical proof of concept and global registration
• Serve as medical monitor and/or oversee junior level medical monitors in early development studies, and support medical oversight of other clinical stage assets at Maze
• Partner with Clinical Development Operations on study feasibility evaluations to identify and select study investigators and sites and build strong professional relationships with study investigators.
• Interact with patient advocacy groups and community engagement leaders to build awareness and enthusiasm for Maze pipeline assets
• Serve as internal and external project champion through interactions with Maze senior leadership, Maze board of directors, academic experts, steering committees, and patient groups
• Be primary clinical author on key clinical-regulatory documents including study protocols, investigator brochures, general investigational plans, annual safety reports, regulatory briefing packages, INDs, and NDAs
• Stay up to date with advances in literature in therapeutic/disease area including mechanism of action, diagnostic tests, treatment, drug development trends, and regulatory requirements
• Develop program-specific publication plans and work with internal and external colleagues to prepare study results for timely publication
• Attend and contribute to relevant scientific conferences, seminars or presentations
• Over time, build, mentor, and manage a clinical team of medical directors to support Maze’s pipeline including a broad range of therapeutic indications and modalities
• Partner with Clinical Development Operations to identify, select, and oversee vendors and CROs to support program needs
• Participate in due diligence activities for potential in-license assets and support intelligence activities for competitor assets
• Develop and integrate timelines and budgets for nonclinical and clinical activities with overall company timelines and goals, in close coordination with project management, CMC, nonclinical development, regulatory affairs, and other stakeholders
• Proactively identify nonclinical risks to Maze programs and develop risk mitigation plans
• Collaborate closely with key cross-functional colleagues to ensure preclinical and clinical studies are conducted with the rigor and documentation required to support global marketing approvals
• Contribute to the development and maintenance of a positive team-focused company culture and champion Maze values through sharp and thoughtful leadership

Qualifications:

• MD with a minimum of 5-10+ years of relevant industry experience, including experience as a clinical study medical monitor
• Board eligibility/certification and clinical experience and/or fellowship in neurology, neuromuscular diseases, nephrology, pediatrics, or clinical genetics is highly desired
• Experience with the preparation and submission of study protocols, INDs, statistical analysis plans, and Investigator Brochures
• Track record of early development leadership on multiple programs resulting in important go/no go decision-making
• Experience in the oversight of early-stage and/or registration-enabling clinical global clinical development programs including CRO, site, and vendor selection and relationship building with investigators/site study staff, advocacy groups, and other third parties
• Direct experience setting patient advocacy strategy and leading successful interactions with patient advocacy groups and patient leaders
• Management experience, particularly in areas of team building, professional development, and mentorship of more junior staff is desired
• Strong leadership and communication skills to translate corporate objectives into action plans that effectively and consistently deliver high quality data on time and on budget
• Ability to adapt quickly to changing environments and priorities
• Positive and enthusiastic disposition and high personal and ethical standards in approaching corporate goals
• Ability to travel domestically and internationally for vendor visits, industry conferences, regulatory meetings, advisory board, and other ad hoc meetings as required