Scientist II, Gene Therapy

At Insmed, every moment and every patient counts — and so does every person who joins in. As a global biopharmaceutical company dedicated to transforming the lives of patients with serious and rare diseases, you’ll be part of a community that prioritizes the human experience, celebrates curiosity, and values every person’s contributions to meaningful progress. That commitment has earned us recognition as Science magazine’s No. 1 Top Employer for five consecutive years, certification as a Great Place to Work® in the U.S., and a place on The Sunday Times Best Places to Work list in the UK. For patients, for each other, and for the future of science, we’re in. Are you? About the Role: We’re looking for a Scientist II on the Gene Therapy Research team to help us expand what’s possible for patients with serious diseases. Reporting to the Senior Director, Research – Gene Therapy, you’ll be a highly independent scientist spearheading critical gene replacement therapy projects, specifically focusing on Stargardt Disease. In this pivotal role, you will lead the design and execution of complex experimental workflows to assess therapeutic efficacy. Your primary focus will be developing specialized in vitro assays to confirm protein function and utilizing advanced histology (IHC, RNA scope) and in vivo mouse models to advance candidates toward clinical trials. What You'll Do: In this role, you’ll have sole responsibility for planning, designing, and executing complex experimental workflows. You’ll also be responsible for: Experimental Design & Project Leadership: Lead highly independent and critical projects aimed at the functional assessment of therapeutic candidates, specifically regarding gene replacement therapies for conditions such as Stargardt Disease. Assay Development & Validation: Spearhead the development of critical in-vitro assays to demonstrate therapeutic efficacy, such as confirming enzymatic or structural function. Establish and confirm that gene therapy products result in fully functional proteins, a pivotal milestone for advancing candidates to clinical trials. Laboratory Management & Supervision: Supervise, manage, and train junior scientific staff (e.g., Senior Research Associates) to support laboratory operations and experimental execution. Manage project-specific expenditures and allocate resources effectively to ensure funds are used to advance the program. Data Analysis & Histology: Analyze complex experimental data and oversee advanced histology workflows, including immunohistochemistry (IHC), RNA in situ hybridization (RNA and Base scope), and quantitative microscopy. Communication & Reporting: Prepare scientific reports and author manuscripts for peer-reviewed journals. Present research findings to cross-functional internal teams and external stakeholders (such as at scientific conferences) to facilitate project progress and support regulatory submissions. Advanced Assay Development: Specialized expertise in designing and executing complex in vitro assays for assessing the functional activity of gene therapy products. Ability to research and develop a deep understanding of complex biological systems (e.g., ABCA4 protein function). In Vivo & Surgical Techniques: Proficiency in conducting in vivo studies, including mouse handling, surgeries, and tissue collection for efficacy and functional analysis. Histology & Microscopy: Competence in implementing and optimizing histology workflows, specifically IHC and RNA in situ hybridization, as well as performing quantitative/qualitative analysis of histological samples. Molecular Biology: Expertise molecular biology, mechanisms, and evaluating the yield, potency, and functionality of gene therapy systems. Resource Management: Ability to manage project-level budgets and oversee the allocation of laboratory resources. Who You Are: You have a Ph.D in Molecular Biology or related field along with relevant post-doctoral or industrial experience. You are or you also have: Prov ... (truncated, view full listing at source)