Head of Clinical Development (Vice President)

At Krystal Biotech, we bring together the brightest and most eager minds to relentlessly pursue the discovery, development, manufacturing, and commercialization of genetic medicines to treat diseases with high unmet medical needs. Founded in 2016, Krystal is distinguished by its science and technology using a patented gene therapy platform, innovative manufacturing supported by commercial-scale facilities, and a unique commercialization model focused on the patient’s end-to-end experience. Krystal received U.S. FDA approval for the first and only redosable gene therapy treatment, VYJUVEK®, for Dystrophic Epidermolysis Bullosa (DEB). The company continues to leverage its proprietary platform to advance a robust pipeline of investigational genetic medicines in respiratory, oncology, dermatology, and ophthalmology. Krystal is headquartered in Pittsburgh, PA, with state-of-the-art CGMP manufacturing facilities and teams worldwide.

About the role

The Vice President of Clinical Development will lead parallel development programs across multiple therapeutic areas from the clinic through product approval. This high-profile, high-impact role will be integral to the clinical team, contributing significantly to advancing programs into and through the clinic as part of a fast-paced, cross-functional team. Responsibilities span Phase 1 through Phase 3 and post-approval activities, including expanding and advancing the company’s programs from early clinical trial development to proof-of-concept and commercialization. The role is based in Pittsburgh, PA.

Responsibilities

• Lead clinical development programs from the late preclinical stage to registration and completion of post-approval commitments.
• Work cross-functionally with Clinical Operations and/or vendors to successfully execute clinical trials and natural history studies, including coordination, collection, and analysis of clinical data, reporting, and ongoing medical monitoring (e.g., eligibility criteria, toxicity management, drug safety surveillance).
• Manage the preparation and/or review of data listings, summary tables, study results, study reports, clinical/regulatory/safety documents, investigator brochures, and clinical development plans.
• Interact with global regulatory agencies, authoring and/or reviewing relevant Pre-IND, IND, and BLA sections, and generating responses.
• Collaborate closely with academic institutions associated with the clinical development program.
• Engage with thought leaders, investigators, cooperative groups, and other experts in scientific and clinical dialog around study design, conduct, and interpretation of results.
• Support patient recruitment on assigned clinical programs, including strategy and execution in tandem with clinical sites.
• Critically evaluate unmet medical needs in various therapeutic areas, outcome measures, and treatment options.
• Partner with internal and external stakeholders to develop manuscripts for publication in peer-reviewed journals and prepare presentations for scientific conferences, investigator meetings, and expert clinical advisory meetings.
• Collaborate with Medical Affairs in developing medical slide decks, educational materials, and publication strategies.
• Represent Krystal Biotech externally through publications and presentations at scientific meetings and congresses, both domestically and internationally.
• Assist with business development and pipeline activities.
• Ensure adherence to regulatory requirements of study conduct and industry standards of Good Clinical Practice.

Requirements

• Ten (10) years of clinical development experience, including designing and managing clinical trials, authoring clinical trial protocols and study reports, and creating clinical development strategies.
• MD with biopharmaceutical industry experience and translational medicine experience; rare disease and clinical pharmacology experience preferred.
• Demonstrated leadership in a clinical development capacity, team-building skills, and the ability to perform effectively in a dynamic, evolving environment.
• Excellent working knowledge of FDA and EMA regulations, Good Clinical Practice, ICH guidelines, and clinical drug development.
• Experience representing the sponsor in front of regulatory agencies and obtaining regulatory approval is preferred.
• Successful submission of INDs and marketing approval-directed filings (BLAs, NDAs, MAAs) is preferred.
• Ability to create an active network with KOLs, key research centers, patient advocacy groups, and patient care organizations.
• Understanding of statistics and data management considerations for clinical trial design and scientific communications.
• Knowledge of medical monitoring of clinical trials (e.g., responding to questions about patient eligibility and protocol waivers; review of safety parameters; receiving and processing SAEs, including SAE narratives; and IND expedited safety reports).
• Strong communication (oral, written) skills to present internally and at scientific meetings, and to author medical documents and scientific publications.
• Ability to collaborate with internal and external stakeholders and a diverse workforce within a cross-functional matrix environment.
• Exceptional organizational, analytical, critical thinking, problem-solving abilities, and strong attention to detail while working on multiple projects in a fast-paced, dynamic start-up and scaling environment.