Senior Director, Biostats

At Neurogene, we focus on bringing life-changing genetic medicines to patients and families affected by rare, devastating neurological diseases. Our clinical-stage company is developing NGN-401 for the treatment of Rett syndrome and NGN-101 for the treatment of CLN5 Batten disease. We are using our proprietary EXACT transgene regulation technology and internal expertise to develop novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. Our wholly owned and fully operational GMP-capable manufacturing facility supports vector production from research through clinical grade and is designed for commercial-grade production. In addition, Neurogene is now trading on the NASDAQ Global Market. This transformative work is driven by our highly collaborative people and comes together to bring us closer to achieving our mission of turning devastating diseases into treatable conditions, to improve the lives of patients and their families. As an employee of Neurogene, you will be part of building a culture that honors patient and caregiver mindsets and nurtures innovation, creative problem solving and a strong sense of purpose.

Position Overview

Neurogene is seeking a Senior Director, Biostatistics to lead the data and analytics work across Development in support of advancing our programs focused on neurologic genetic disorders into and through the clinic. This individual will be part of a fast-paced cross-functional team, providing expertise in biostatistics and programming, working across research, preclinical and development to bring therapies through development. In this highly collaborative role, the direct responsibilities will be leading statistical efforts for clinical and nonclinical studies, leveraging skills in biostatistics to support regulatory review on the safety and efficacy of our gene therapies, and executing and delivering on critical clinical milestones. The candidate should manifest keen ability to develop novel statistical approaches for small sample sizes, including potentially Bayesian approaches, and to perform multivariate modeling of biomarker and clinical data from animal models to inform human clinical trial design is desired. Additionally, the expertise should be developed from real experiences in challenging the normal approaches to data analysis of normal populations and have experience in rare disease drug and therapeutic development. The candidate should be comfortable with developing insights into novel data analyses supporting clinical evidence of effectiveness for rare disease populations.

Accountabilities and Responsibilities

• Serve as a lead statistician and manage statistical efforts for clinical studies
• Contribute to clinical protocol development, including authoring sections on statistical methods and reviewing/editing other sections, by applying statistical principles
• Author statistical analysis plans for studies and/or ISS/ISEs, and author/edit tables, figures, and listings
• Review CRF designs to ensure data collection meets the study objectives and the requirements of statistical analyses
• Provide statistical input to independent data monitoring board charters, project management plans, and other study-level documents
• Work with statistical programmers or CROs to generate tables, figures, and listings
• Support ongoing safety review and data monitoring review of the clinical development program/study teams
• Perform ad hoc and exploratory statistical analyses as needed
• Contribute to clinical study reports, including authoring of statistical sections and interpretation of the study results
• Support regulatory submissions as needed
• Support the preparation of publications, including manuscripts, posters, and oral presentations
• Provide oversight of CROs for outsourced statistical activities and QC key results generated by CROs

Education

• PhD in biostatistics, mathematics, statistics, public health, or related discipline, with at least 7 years of relevant clinical trial experience
• MS in biostatistics, mathematics, statistics, public health, or related discipline, with at least 9 years of relevant clinical trial experience

Experience

• With an advanced degree, 7-9 years of substantive experience in the pharmaceutical/biotechnology industry
• In-depth understanding of clinical study designs, drafting protocols, authoring statistical methods sections in Regulatory documents
• Experience in pediatrics, neurology, rare diseases and/or gene therapy and small trial designs would be ideal

Skills, Knowledge & Attributes

• Deep knowledge of statistical methods for clinical trials
• Knowledge of FDA, EMA and ICH regulations and guidelines
• Proficient in statistical programming (SAS is required and R is a plus)
• Experience with trial design software
• Effective communication skills and ability to work with cross-functional study teams
• Excellent analytical and problem-solving skills
• Positive, outgoing, and collaborative attitude
• Strong organizational skills and ability to deliver professional and quality work products, while working on multiple projects in an extremely fast-paced environment
• Continuous strong focus on meeting or exceeding expectations in executing deliverables, performance and results.
• Ability to track and measure performance against defined metrics.
• Self-starter with high degree of energy, independence, initiative and self-motivation, as illustrated by the ability to instill a sense of pace and urgency into the team
• Someone who moves fast and decisively in a balanced manner, with a passion for the growing company
• Passionate, collaborative, creative, willing to take prudent risks, and motivated by the creation of a company focused on transformative treatments for patients and their families