GondolaBio

Director

• Identify, formulate, prioritize, and oversee chemical development strategies and activities to address complex technical and supply challenges.
• Collaborate with senior leadership, Research, Regulatory, Quality Assurance, Drug Product, Supply Chain, and Clinical Operations, and communicate chemical development risks and timeline impacts promptly.
• Lead CDMO process chemistry teams in advancing small molecule drug substances from candidate selection through NDA approval and launch.
• Lead route scouting, route selection, and process optimization to improve safety, robustness, yield, purity, scalability, cost, and raw material availability.
• Develop phase-appropriate impurity control strategies, including understanding impurity formation, fate, purge, and carryover, as well as mutagenic impurity and nitrosamine risks.
• Lead process scale-up, crystallization and isolation development, technology transfer, process characterization, validation, and GMP manufacturing activities.
• Design and execute detailed development and manufacturing work plans with CDMOs, providing active technical oversight and holding partners accountable for quality, timelines, budgets, and deliverables.
• Author and approve regulatory documents, development reports, manufacturing documentation, and responses to health authority questions, and participate in regulatory interactions.
• Review and approve protocols, reports, batch records, deviations, investigations, change controls, and corrective and preventive actions.
• Develop budgets, timelines, and resource plans, and manage and mentor internal scientists, consultants, and external technical teams while fostering a culture of urgency, ownership, and collaboration.

Senior Director, Chemical Development

• 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.

Senior Director – Biostatistics, Neurology

Vice President

• Provide scientific strategy and operational leadership across the development sciences functions that take genetic disease programs from candidate selection through first-in-human and early clinical development, including:
• Nonclinical Pharmacology & DMPK Sciences • Direct nonclinical PK/PD strategy spanning target engagement, pharmacodynamic response, and efficacy.
• Oversee in vitro and in vivo ADME and DMPK characterization to support candidate selection and IND-enabling packages.
• Biomarker Sciences & Development • Lead biomarker identification and the nonclinical-to-clinical biomarker strategy and execution.
• Drive clinical biomarker qualifications to support proof of mechanism and proof of concept.
• Clinical Pharmacology & Early Clinical Development • Own first-in-human through Phase 2 early clinical sciences and development strategy.
• Direct clinical pharmacology, including Phase 1 healthy-volunteer and special-population studies.
• Quantitative Sciences • Provide leadership across data analytics, pharmacometrics, biostatistics, and data management and data sciences to inform dose selection and program decisions.
• Research Operations • Oversee nonclinical and translational study execution and vendor management.
• Manage clinical pharmacology study execution and associated vendor relationships.
• Bioanalytical Sciences • Direct bioanalytical assay development for PK, biomarkers, and immunogenicity.
• Oversee nonclinical and clinical bioanalytical operations.
• Cross-Functional Leadership • Partner closely with Clinical Development, Global Regulatory Affairs, CMC, and Research to deliver integrated development plans and regulatory submissions (pre-IND, IND, FIH, Phase 1/2).
• Personally drive the technical work across these functions, staying close to the science and the data while leveraging CROs and external partners to deliver, consistent with a lean, hands-on early-stage culture.
• Represent development sciences to senior leadership, the board, and external advisors, and contribute to portfolio-level prioritization and resourcing decisions.

Vice President, Development

General

• Identify promising new preclinical or early clinical stage programs to treat genetically driven diseases through deep engagement with the literature, academic and industry experts, and internal development efforts
• Deeply evaluate all aspects of a potential therapeutic program—from genetic rationale and pre-clinical evidence all the way to the development plan and clinical trial design
• Advocate for those that fit our goal of targeting the genetic source of a disease by building a scientific and business case for each potential investment
• Interface with leading academics, patient advocacy groups, biotechs, and entrepreneurs
• Lead internal discussions with management and the Investment Committee
• Establish a focused investment strategy and follow through to identify new investment opportunities
• Coordinate and work with a talented team of internal subject-matter experts—including clinical, commercial, and disease experts—to dive as deeply as needed for each project

Associate, Asset Acquisition

Compliance

• Serve as the global regulatory lead for assigned development programs, defining stage-appropriate regulatory strategies and development pathways.
• Develop regulatory strategy for rare disease and accelerated development programs, including orphan designation applications and expedited development pathways.
• Partner closely with Clinical Development, Translational Sciences, Nonclinical, CMC, and other functional teams, including working with CROs as needed, to integrate regulatory considerations into development planning and key program decisions.
• Provide strategic regulatory input on study design, translational biomarkers, dose selection, and first-in-human safety considerations.
• Provide regulatory guidance to support efficient and informed decision-making while balancing development speed, scientific rigor, and long-term program optionality.
• Lead preparation for and interactions with global health authorities, including briefing documents, meeting strategy, and written correspondence.
• Lead and contribute directly to the planning, preparation, and submission of high-quality regulatory submissions (e.g., IND, CTA), amendments, annual reports, and other regulatory filings.
• Maintain awareness of evolving regulatory guidance and industry trends that may impact development strategies.
• Support global development efforts by evaluating regional regulatory requirements and opportunities.

Associate Director – Regulatory Affairs

Translator

• Lead or directly contribute to evaluation of oligonucleotide DMPK data: pharmacokinetics, biodistribution, tissue uptake, durability, and exposure-response relationships
• Collaborate with DMPK teams to assess PK/PD relationships and contextualize safety findings relative to systemic and tissue-level exposure
• Integrate DMPK insights into cohesive preclinical and translational narratives supporting candidate selection and IND filing
• Lead integration and interpretation of preclinical safety and pharmacology data, with a strong focus on off-target effects and risk mitigation
• Partner with toxicology and biology teams to distinguish target-related from off-target findings, assess dose-limiting toxicities, and evaluate therapeutic index
• Identify data gaps, recommend mechanistic or follow-up studies, and support risk mitigation strategy
• Contribute directly to IND-enabling study design and execution, including in vitro and in vivo pharmacology, ADME, and supporting safety packages
• Serve as a senior scientific leader on siRNA and/or ASO programs, providing expert review of oligonucleotide design strategies, chemistry, modification approaches, and conjugation strategies
• Critically evaluate complex preclinical datasets and synthesize data-driven recommendations for program decisions
• Provide scientific oversight of CRO-executed studies — preclinical safety, in vivo pharmacology, bioanalytical, and DMPK — including study design input, protocol review, data package review, and final reports
• Serve as primary scientific point of contact with external partners to ensure scientific rigor, clear communication, and timely delivery
• Collaborate closely with chemistry, DMPK, toxicology, translational, and clinical teams to support data-driven program decisions
• Clearly communicate complex preclinical datasets in team meetings and governance forums

Senior Director, Chemical Development

Senior Director – Biostatistics, Neurology

Vice President, Development

Associate, Asset Acquisition

Associate Director – Regulatory Affairs

Director/Sr. Director, Oligonucleotide Translational Science