Sr. Director - Biostatistics, Neurology

About GondolaBio

GondolaBio is a clinical-stage biopharmaceutical company focused on developing breakthrough therapeutics for genetic diseases. GondolaBio was originally carved out in 2024 after securing $300M in private financing from external investors. GondolaBio aims to leverage cutting-edge biological research to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases.

Who You Are

You are an experienced biostatistician with deep expertise in rare disease clinical development. You thrive in lean, cross-functional settings and bring both methodological rigor and pragmatic judgment to study design and analysis. You are energized by the challenge of extracting meaningful, credible signal from small, heterogeneous patient populations, and you understand that in rare disease, thoughtful statistical thinking is itself a competitive advantage.

Key Responsibilities

• Statistical leadership for ASO programs (Phase 1/2 to PoC readiness): Serve as the lead statistician for GondolaBio's ASO assets, owning statistical strategy from early FIH through proof-of-concept. Develop and defend fit-for-purpose statistical analysis plans (SAPs) that account for small sample sizes, placebo and open-label designs, and high inter-patient variability.

• Seizure endpoint strategy: Design robust statistical approaches for seizure frequency and responder analyses in Developmental and Epileptic Encephalopathy populations, where within-patient variability, seizure clustering, and non-normality require non-standard methods. Partner with clinical colleagues to define scientifically credible and regulatorily acceptable primary and secondary endpoints.

• Rare disease-adapted trial design: Apply specialized methods appropriate for small N studies including Bayesian frameworks, adaptive designs, n-of-1 elements, and natural history benchmarking. Proactively identify and mitigate risks from high variability, missing data, and limited statistical power, communicating trade-offs clearly to program leadership.

• Regulatory statistical support: Prepare and contribute to statistical sections of INDs, briefing documents, and FDA/EMA meeting packages. Support pre-IND and Type B/C meetings, and respond to agency questions on statistical methodology.

• Biomarker and exploratory analysis: Collaborate with Pharmacology, Translational, and Clinical teams to develop analysis plans for PD biomarkers, target engagement endpoints, and patient stratification. Apply appropriate multiplicity controls and exploratory frameworks to preserve interpretability.

• Cross-functional collaboration: Partner closely with Clinical Development, Regulatory, Clinical Operations, and external CROs/vendors to ensure statistical considerations are embedded early in protocol development, CRF design, data management, and study conduct. Represent Statistics in cross-functional program team meetings.

Requirements

• Preferred Education & Experience:

  • M.S. or Ph.D. in Biostatistics, Statistics, or a closely related field.
  • 8+ years of pharmaceutical/biotech industry experience, with meaningful early-phase (Phase 1/2) rare disease exposure.
  • Demonstrated experience designing and analyzing seizure trials (e.g., DEE, TSC, Dravet, LGS) or other high-variability neurological endpoints.
  • Deep familiarity with statistical challenges specific to rare disease: small sample sizes, open-label designs, natural history controls, and high inter-patient variability.
  • Hands-on proficiency with SAS and/or R; ability to program and review analysis datasets and outputs independently.
  • Experience authoring and defending SAPs, study reports, and regulatory-facing statistical documents.
  • Comfort operating in lean, fast-paced biotech environments with significant autonomy and accountability.

• Nice-to-Haves:

  • Experience with Bayesian or adaptive design methodologies in rare or neurological disease trials.
  • Familiarity with PRO/ClinRO endpoint development and validation in pediatric or rare disease populations.
  • Experience with responder analyses, seizure diary data, and mixed-effects models for repeated measures (MMRM) in epilepsy studies.
  • Exposure to seamless Phase 1/2 designs or platform trials.
  • Experience interfacing directly with FDA or EMA on statistical methodology for rare disease.

What We Offer

• Patient Days, where we are fortunate to hear directly from individuals living with the conditions we are seeking to impact throughout the year and learn how we can improve our efforts.

• A culture inspired by our values: put patients first, think independently, be radically transparent, every minute counts, and let the science speak.

• An unyielding commitment to always putting patients first.

• A decentralized model that enables our program teams to focus on advancing science and helping patients.

• The base pay range for this position is $300,000 to $360,000 per year. The actual compensation offered will be determined based on a number of job-related factors, including experience, skills, qualifications, and location.

• A bonus plan and equity will also be offered.

• GondolaBio ServiceCo is an equal opportunity employer. All applicants will be considered for employment without attention to race, color, religion, sex, sexual orientation, gender identity, national origin, veteran or disability status.