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We follow the science that will lead us to the best possible outcomes for patients.

Mission

We are a mutation-agnostic in vivo gene editing company striving to cure liver disorders with high unmet needs by harnessing new advancements in genetic medicine. Our in vivo insertion methods knock in healthy copies of disease-causing genes, offering long-term, stable expression of those genes in patients.

Culture

Our science is at the leading edge, and so are we. Our team of experts is here to transform lives and bring potentially curative treatments to patients in need. Great work takes a deep level of collaboration at all levels, and we strive every day to foster an environment that inspires innovation, execution and an unrelenting commitment to patients.

We are built on the world-class translational engine at the University of Pennsylvania’s Gene Therapy Program (UPenn GTP). The work executed by this group of world‑renowned gene therapy experts on our pipeline provides a substantial developmental head start – meaning treatments in the hands of patients as quickly as possible.

Work With Us

Interested in joining our team of genetic medicine experts?

Please email careers@iecure.com to learn more, and browse our current job openings below.

No current openings.

Position Summary:

The Lead Field Medical Director will report directly to the VP, Therapeutic Area Lead and be responsible for developing and maintaining professional relationships with external healthcare professionals (HCPs) to provide comprehensive medical and scientific support for targeted therapeutic areas.

This position will support the VP, Therapeutic Area Lead in successful execution of clinical trials including engaging appropriately in scientific exchanges around product information, disease education, and research ideas/activities with KOLS, investigators, physicians, and ancillary HCPs regarding current and future therapies in development.

The selected candidate will work closely to ensure adequate training, access to educational resources and alignment with medical affairs plans are communicated to address the unmet needs of the patients and HCPs. The Lead Field Medical Director will be responsible for setting Medical Affairs strategy as well as plan execution. This position will require at least 40-60% domestic and international travel and requires the candidate to live within driving distance of the iECURE Blue Bell, PA office and attend meetings in the Blue Bell office when not traveling.

Characteristics sought for a good cultural fit to the company include the ability to work collaboratively, strong project management skills, excellent planning and budgeting skills, ability to think strategically and act independently, a “hands on” approach in the implementation of an agreed-upon plan, nimble problem-solving skills, an innate sense of urgency, creative thinking about what is possible in execution, and general positivity.

Specific Responsibilities

  • Primary role will be to provide medical support, education, training, respond to questions for ongoing clinical trials.
  • Identify Medical Experts, consultants, investigators, and HCPs in targeted therapeutic areas.
  • Develop and maintain professional relationships to ensure timely, effective, and appropriate communication of current and emerging medical and scientific information on therapeutic disease areas in support of clinical trials.
  • Present data on products to HCPs including physicians, academic institutions, researchers, and allied health care professionals, consistent with all company policies and procedures.
  • Recruit and develop an Advisory Board and leverage in scientific forums to solicit critical KOL insights and feedback. Foster the development of scientific publications in peer reviewed journals.
  • Conduct in person scientific exchanges related to standards of care and iECURE clinical trials at Centers of Excellence, potential referring centers and Academic Medical Centers.
  • Field therapeutic area expert demonstrating high scientific knowledge and competency of therapeutic disease states, products, and competitor data.
  • Coordinate and attend activities and external engagements at appropriate meetings and conferences.
  • Support research and development programs and may include trial site identification, ongoing recruitment motivation, and issue/problem solving.
  • Serve as a local medical and scientific resource for clinical trial planning, clinical insights, and intelligence. Capture, synthesize and deliver HCP derived scientific insights to cross functional teams.
  • Provide leadership for publication planning development and execution. Deliver and execute effective product and disease-state training and mentoring to internal colleagues.
  • Provide full support and execution of Medical Affairs plans, Clinical Development projects, programs, and initiatives.

Education & Qualifications

  • An advanced degree in the life sciences field (Pharm D, PhD, MD, NP/PA, and CGC) or relevant field medical experience with a strong background in molecular biology, genetics, and genomics.
  • A minimum of 5 years of experience as a MSL in rare/orphan pediatric diseases required, unless the candidate has strong related experience.
  • Other Pharmaceutical experience – Clinical management/trial experience and 2 years clinical or research experience (non-pharma) specifically in pediatrics is preferred.
  • An excellent command of the English language. Effective presentation skills and advanced proficiency in interpersonal skills (listening, probing, persuasiveness, negotiation) are essential for engaging successfully and productively with HCPs, external customers, field colleagues, and internal stakeholders.
  • Skilled in conflict resolution and problem solving.
  • Knowledge of the PhRMA code of conduct, IHI GCP, the Sunshine Act, HIPAA regulations, Promotional Regulations, and all laws and regulations that are applicable to the role.
  • Uncompromising ethical standard and conduct.
  • Excels at building relationships, networking with others and working cross-functionally.
  • Ideal candidate will be within driving distance of the Blue Bell, PA office.

Department

Medical Affairs

Updated

April 2024

Location

Fulltime, based in Blue Bell, PA

EEO

iECURE is an Equal Opportunity employer and will consider all qualified applicants for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, protected veteran status, or disability status.

Position Summary:

The Senior Clinical Research Associate (CRA) will report directly to the Senior Clinical Trials Manager, Clinical Operations. This position has the responsibility of supporting the company’s clinical operations function in close collaboration with research, development, and other functions. This includes working directly with the study team, vendors, and clinical study sites to ensure that clinical trials are conducted in a manner compliant with SOPs, ICH/GCP/CFR regulatory guidelines, as well as in line with company goals, timelines, and budgets. This position provides input and necessary support and oversight for high quality execution of global clinical trials. This position also provides support for documentation of important clinical trial data and necessary operational information to prepare for data base lock and study closure.

This individual will be accountable for executing on the operational plan for clinical studies and leading site training, patient finding/recruitment, data collection and source data verification activities at the site level according to program timelines and quality targets. The Senior Clinical Research Associate will be a seasoned professional with a background in life sciences and end-to-end clinical operations experience from working within the biotech or pharmaceutical industry. Experience supporting clinical trials for rare disease indications is preferred.

Characteristics sought for a good cultural fit to the company include the ability to work collaboratively, strong organizational skills, excellent planning and communication and interpersonal skills, ability to think strategically and act independently, a “hands on” approach in the implementation of an agreed-upon plan, nimble problem-solving skills, an innate sense of urgency, creative thinking about what is possible in execution, and general positivity.

Responsibilities

  • All aspects of study site monitoring and management including routine monitoring and close-out of clinical sites, maintenance of study files, conduct of pre-study and initiation visits; liaise with vendors; and other duties, as assigned
  • Organize and conduct presentations at Investigator Meetings
  • Participate in the development of protocols and Case Report Forms and clinical trial reports as assigned
  • Recruitment of potential investigators, preparation of IRB/EC submissions, notifications to regulatory authorities, translation of study-related documentation, organization of meetings and other tasks as instructed by supervisor as assigned
  • Negotiate study budgets with potential investigators and assist the iECURE/CRO legal departments with statements of agreements as assigned
  • Independently perform CRF review; query generation and resolution against established data review guidelines per SOPs or data management systems as assigned by management
  • Domestic and international travel may be required and is an essential function of the job; prepare accurate and timely trip reports; review progress of projects and initiate appropriate actions to achieve target objectives
  • Conduct, report and follow-up on Shadow Monitoring Visits (Quality Spot Checks) when requested for CRO or iECURE monitors
  • Manage the operational, quality, and financial aspects of clinical trial sites
  • Support the study project plan, including timeline, budget, and resources
  • Participate in protocol, CRF, and study plans strategy development as appropriate
  • Develop patient recruitment planning and strategy at the site levels
  • Proactively identify potential study issues/risks and recommend solutions
  • Maintain systems (eTMF, CTMS, EDC, etc.) that track progress throughout the duration of the program
  • Manage all site activities ensuring compliance with Good Clinical Practices (GCP) and applicable regulations
  • Ensure appropriate clinical trial supplies are planned and ordered in time for all assigned sites
  • Develop and conduct site training materials for disease state and clinical protocol objectives
  • Hold clinical sites accountable to study timelines for start-up, patient recruitment strategies, enrollment, and quality data deliverables
  • Report progress of key study milestones and data deliverables to supervisor on a weekly basis
  • Collaborate effectively with vendor partners and internal functions to develop and execute strategies to expedite enrollment of clinical studies
  • Ensure supporting documentation (e.g., case report forms, monitoring plans, data management plans, safety plans) protocol objectives and operational efficiencies are followed
  • Accountable for the ongoing review of EDC data and issuance of queries in collaboration with CROs/vendors
  • Establish, grow, and maintain strong relationships with study sites and investigators
  • Review monitoring reports, conduct co-monitoring visits and support resolution of issues related to sites as needed

Qualifications

  • A BA/BS degree required; advanced degree with scientific or health-care training preferred
  • COVID-19 vaccination required
  • Minimum of at least 3-4 years of Onsite Clinical Monitoring experience. At least 1-year additional experience in related field
  • Advanced site monitoring; study site management; and registry administration skills
  • Ability to train and supervise junior staff; resolve project related processes
  • Thorough knowledge of monitoring procedures and strong understanding of the drug accountability process
  • Valid driver’s license and current passport
  • Have a full understanding of the Serious Adverse Event (SAE) reporting, process production of reports, narratives and follow up of SAEs
  • Strong knowledge of rare disease clinical research highly desired
  • Knowledge and experience in gene therapy and gene editing, preferred
  • Experience with pediatric, in-patient / hospital-based studies, highly desired
  • Experience monitoring global clinical trials for a product through all clinical phases of clinical development
  • Demonstrated career success as a seasoned CRA, effective organizational skills, excellent verbal and written communication skills and high attention to detail
  • Excellent working knowledge of FDA & ICH/GCP/CFR regulations and guidelines
  • Ability to work in a team environment, meet deadlines, and prioritize and balance work from multiple individuals
  • A professional who inspires trust and confidence in others. A resourceful, energetic self-starter who can shift between collaboration and execution
  • Possess unquestionable integrity with the highest ethical standards
  • Willingness and ability to participate in domestic and international travel, as required, up to 30%.

Department

Operations

Updated

January 2023

Location

Fulltime, Onsite, East Coast

EEO

iECURE is an Equal Opportunity employer and will consider all qualified applicants for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, protected veteran status, or disability status.

Position Summary:

Reporting directly to the Analytical Development (AD) and Testing Lead, the Analytical Development (AD) and Testing Senior Analyst will support the management of the life cycle of analytical development for in-process, release and stability program testing of raw materials, plasmids, cell/bacterial banks, drug substance and drug products, in support of discovery, development, and manufacturing of iECURE’s product portfolio from the clinic through commercial launch.

This individual will be active, technical, collaborative, a critical thinker with project management skills, who can work effectively with the founding scientific team at Penn’s Gene Therapy Program (GTP) and contract partners. This individual will provide input and support global regulatory submissions for the Chemistry, Manufacturing and Controls (CMC) package into the clinic through commercial under the stewardship of iECURE.

The Senior Analyst will be a seasoned professional who has supported, executed, and helped define analytical development and testing strategy associated with all phases of drug development, including the coordination and preparation of document packages for regulatory submissions. This individual will have strong experience dealing with the U.S. Food and Drug Administration (FDA) and other regulatory agencies worldwide.

Characteristics sought for a good cultural fit to the company include the ability to work collaboratively, strong project management skills, excellent planning and budgeting skills, ability to think strategically and act independently, a “hands on” approach in the implementation of an agreed-upon plan, nimble problem-solving skills, an innate sense of urgency, creative thinking about what is possible in execution, and general positivity.

Specific Responsibilities

  • Manage the development, verification, qualification, and validation of analytical methods inclusive of assay relevance, reproducibility, reliability, robustness, and optimization.
  • Provide strong scientific background in implementation of molecular biology assays, microbiological safety assays, compendial methods and have deep understanding of phase appropriate method verification, qualification, validation, sample and data analysis, GLP, GMP requirements, ICH and FDA guidance.
  • Work with CDMOs in technical transfer, design, method development, bridging, comparability, and validation employing phase appropriate approaches at the different stages of development for assay test articles.
  • Support the development and execute an approach for characterization to support identification of CPP/CQAs and define the control strategy. Support the development of process analytical technology (PAT) to design, define, analyze, and control the manufacturing process.
  • Be responsible for CDMO scheduling, sample plans and management, test articles/procedures, personnel training, test execution, results and troubleshooting analytical method issues and lab investigation/deviations during QC testing.
  • Provide in-process manufacturing as person-in-plant and QA batch release.
  • Provide input, draft and review of analytical related technical studies, gap analyses, risk assessments, methods/articles, protocols, memos, data, impact assessments, and reports.
  • Support clinical assay development and help manage the operations and execution of clinical assays at CROs.
  • Support the oversight of the stability program and ensure it supports regulatory objectives and timelines.
  • Help prepare the CMC sections for regulatory filings. Participate in the preparation, assembly and filing of CMC regulatory submissions to support development stage and new product approvals, including interactions between the company and health authority representatives to facilitate submissions. Assist in the submission and resolution of agency related questions.
  • Remain at the forefront of the relevant science and competitive landscape including new analytical technologies.
  • Build a collaborative working relationship with Penn GTP’s CMC organization and provide guidance on analytical development and testing activities to produce the most efficient and effective outcomes possible for iECURE.
  • Collaborate with Technical Operations and other internal functions to ensure the management of all financial aspects of manufacturing activities including budgeting/forecasting, long-range planning, quantitative decision making, and overall cost management and accountability of product supply, in relation to CMC activities.

Education & Qualifications

  • BS/BA degree in Science.
  • An advanced degree is desired, such as a PhD or MS, in chemistry, biochemistry, or a relevant discipline.
  • 7+ years of experience in the biotech industry, and experience in all stages of biological CMC development.
  • Knowledge and experience in gene therapy and gene editing in AAV manufacturing are preferred.
  • Knowledgeable in analytical techniques such as NGS, AUC, PCR, cell-based potency, ELISA, HPLC, MS, DLS and compendial methods.
  • Experience with clinical assays is desired.
  • Strategic/forward-thinker and CMC technical experience. Experience in a successful project participant role supporting the development, supply, and registration of candidate drugs through market approval.
  • Experience interacting with regulatory agencies, specifically with the FDA and international regulatory authorities.
  • Proven track record of successfully participating in technology transfer of projects.
  • Experience in developing and scaling manufacturing, with a stellar track record interacting with third parties and CDMOs to produce the highest quality drug products for patients.
  • Familiarity with line management and project management, embracing budgets and timelines to deliver on commitments made.
  • Demonstrable experience of working hands-on with limited resources.
  • Ability to understand the strategic, big-picture level to be effective when delving into details to successfully deliver project goals.
  • Positive attitude and change agent capable of working with others in a matrix environment.
  • Proficient at approaching problems/risks/issues with professionalism, confidence, and decisiveness with the ability to provide resolutions.
  • Possess outstanding presentation, oral and written communication skills, as well as an enthusiastic and open attitude toward learning and continuing professional development.
  • A commitment to excellence and continuous quality & efficiency improvements.
  • Able to manage local and national travel, with up to 20% travel to vendor sites.
  • Ability to work and support holidays, weekends, and off hours, as needed.

Department

Operations

Updated

April 2024

Location

Fulltime, on-site, based in Blue Bell, PA

EEO

iECURE is an Equal Opportunity employer and will consider all qualified applicants for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, protected veteran status, or disability status.