Job Overview: Krystal Biotech, Inc., a leader in innovative gene therapy solutions, is seeking a dynamic Regional Gene Therapy Liaison specializing in ultra-rare diseases for the East Coast region, based in Philadelphia, PA. This pivotal role involves serving as the primary point of contact between Krystal's cutting-edge gene therapies and key stakeholders, including healthcare professionals, patient advocacy groups, and regional partners, to accelerate access and adoption of transformative treatments for patients with ultra-rare conditions. Key Responsibilities: Act as a scientific expert and liaison, delivering tailored education on Krystal's gene therapy portfolio to physicians, nurses, and treatment centers across the East Coast. Build and maintain strategic relationships with key opinion leaders (KOLs), advocacy organizations, and payers to support therapy launches and reimbursement strategies. Identify regional needs, gather insights on ultra-rare disease landscapes, and relay actionable feedback to internal teams to refine commercialization efforts. Organize and lead regional advisory boards, webinars, and field training sessions to ensure compliant and impactful dissemination of clinical data. Collaborate cross-functionally with medical affairs, sales, and market access teams to drive patient identification and therapy uptake. Monitor competitive activities and emerging trends in gene therapy for ultra-rare diseases, providing timely reports to leadership. Ensure all activities adhere to regulatory guidelines, including FDA requirements and industry codes of conduct. Required Qualifications: Bachelor's degree in life sciences, biotechnology, or related field; advanced degree (MD, PharmD, PhD) strongly preferred. Minimum 5-7 years of experience in biotech/pharma, with at least 3 years in rare diseases, gene therapy, or medical affairs roles. Proven track record in stakeholder engagement and field-based medical liaison positions. Deep knowledge of ultra-rare disease pathophysiology, gene therapy mechanisms, and the U.S. healthcare ecosystem, particularly on the East Coast. Skills: Exceptional communication and presentation skills, with the ability to translate complex scientific concepts into accessible language. Strong relationship-building acumen and networking prowess. Proficiency in data analysis, CRM tools (e.g., Veeva), and Microsoft Office suite. Strategic thinking, problem-solving, and adaptability in a fast-paced, field-oriented environment. Travel readiness (50-70% regionally). Commitment to patient-centricity and ethical standards. Company Info: Krystal Biotech, Inc. (NASDAQ: KRYS) is a Philadelphia-headquartered biotechnology company pioneering herpes simplex virus (HSV)-based gene therapies for serious diseases. With FDA-approved Vyjuvek as the first gene therapy for dystrophic epidermolysis bullosa (DEB), Krystal is expanding its pipeline to address unmet needs in ultra-rare conditions through innovative manufacturing and delivery platforms. Benefits: Competitive compensation package including base salary, performance bonuses, and equity. Comprehensive health, dental, and vision insurance; 401(k) matching; generous PTO and paid parental leave. Professional development stipend, wellness programs, and employee stock purchase plan. Growth Opportunities: Join a high-growth biotech at the forefront of gene therapy innovation. Advance to senior liaison, director-level roles, or transition into medical affairs leadership. Ongoing training in emerging therapies, leadership development programs, and opportunities to contribute to global launches.