PUSH THE ENVELOPE.
JOIN OUR TEAM.
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.
VP, Head of Regulatory Affairs
Location: Plymouth Meeting, PA
Type: Full time
Company Overview
iECURE (pronounced EE-ah-cure) is a preclinical stage gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, therapy for the treatment of monogenic liver disorders with significant unmet need. iECURE’s approach has the potential to replace and restore the function of a dysfunctional gene by knocking-in a healthy copy, regardless of mutation, to offer durable gene expression and long-term, potentially curative, therapeutic benefit.
The company is collaborating with the University of Pennsylvania’s (Penn) Gene Therapy Program (GTP), led by Dr. James Wilson, to utilize the GTP’s world-class translational expertise and infrastructure, which has helped generate our initial pipeline of product candidates. The Penn collaboration provides iECURE with the exclusive right to develop up to 15 product candidates each focused on a liver indication; we have nominated three product candidates to date, which comprise our initial pipeline.
Position overview
With preparations underway to translate pre-clinical work from Penn’s GTP to the clinic, as well as ambitions to build out a manufacturing capability, iECURE is seeking a Head of Regulatory Affairs (RA) to join its senior leadership team.
The successful candidate will be strategic-minded, diplomatic and a highly experienced regulatory affairs leader who can work effectively with the founding scientific team at Penn to provide strategic guidance on IND submissions and support on translating the pre-clinical package into the clinic under the stewardship of iECURE. This individual will also play a key role in defining the company’s regulatory strategy across its pipeline and providing regulatory CMC support.
Key Responsibilities
- Provide overall leadership and regulatory guidance that shapes the direction of iECURE’s programs
- Lead regulatory strategy with pediatric division of global regulatory agencies
- Develop strategy to enable expedited submission of BLA and minimize agency’s review timelines for clinical candidates (e.g., Breakthrough Designation, Prime Designation, Orphan Drug Designation, Pediatric Designation)
- Serve as an influential leader and develop strong and productive working relationships with key stakeholders
- Conduct a process to select the appropriate regulatory consulting firm to support global regulatory IND/CTA submissions
- Represent the regulatory function in each of the clinical program’s Project Team meetings, informing the Project Team as to the status of key tasks
- Collaborate with Project Management Team members to develop and execute global regulatory strategy that accelerates timelines for approval
- Participate in the Quality Board ensuring iECURE’s quality programs are maintaining GxP standards
- Oversee and lead the preparation of effective, persuasive presentations to regulatory authorities
- Create clear and concise documents regarding regulatory strategy for senior management
- Provide strategic direction regarding dossier content to ensure that knowledge of the drug, disease, regulatory requirements, and idiosyncrasies of regulatory authorities are incorporated
- Review protocols, study reports, and all types of regulatory documentation (e.g., quality, safety, efficacy)
- Hire, develop, and lead a strategic team to build the organization as it evolves through approval, prelaunch, and launch activities
- Ensure representation as needed in governance or strategic teams or committees
- Remain at the forefront of the relevant science and competitive landscape including detailed competitive intelligence and be viewed as an expert in its application to the regulatory process
- Partner and provide guidance on Penn’s regulatory activities to produce the most efficient and effective outcomes possible for iECURE
Requirements
iECURE is seeking a seasoned professional who has held the lead role in a company defining the regulatory strategy associated with all phases of drug development, including the coordination and preparation of document packages for regulatory submissions. This individual will have a strong rapport and collaborative relationship with the U.S. Food and Drug Administration (FDA) and other regulatory agencies worldwide.
Aside from technical capabilities, this individual will have outstanding presentation, oral and written communication skills, as well as an enthusiastic and open attitude toward learning and continuing professional development. Evidence of the ability to achieve results, both independently and in a team, is required in addition to being able to navigate a dynamic and fast-paced work environment.
Qualifications
- BA/BS required, advanced degree in a life science or related discipline preferred (e.g., PhD, PharmD, MSc, MBA)
- A minimum of 10 years of regulatory affairs experience, ideally gained from a mix of both large and small companies
- Experience working with cell and gene therapies and/or rare diseases is preferred. Other therapeutic areas will be considered if the individual has experience defining and designing novel regulatory pathways
- Experience with global regulatory agencies on submission for pediatric indications
- A prior track-record of successful regulatory delivery, with IND/CTA and NDA/BLA submission experience
- Deep knowledge which reflects current regulatory guidelines and practice, including electronic submissions
- Ability to balance corporate goals with the current regulatory requirements
- Possess a high level of credibility and relationships with the FDA and other international regulatory authorities
- Comfortable brainstorming and collaborating with executives and members of the team, and encompassing the ability to translate ideas into more detailed strategy
- Outstanding written and communication skills to include experience in authoring both internal and externally facing regulatory related documents
- Demonstrable experience of working hands-on with limited resources
- Ability to think at a strategic, big-picture level and delve into details when appropriate
- Positive attitude and change agent capable of working and leading others in a matrix environment
- Proficient at approaching problems/risks/issues with professionalism, confidence, and decisiveness with the ability to provide resolutions
iECURE, Inc. 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.
Senior Clinical Trials Manager, Clinical Operations
Location: Plymouth Meeting, PA
Type: Full time
Company Overview
iECURE (pronounced EE-ah-cure) is a preclinical stage gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, therapy for the treatment of monogenic liver disorders with significant unmet need. iECURE’s approach has the potential to replace and restore the function of a dysfunctional gene by knocking-in a healthy copy, regardless of mutation, to offer durable gene expression and long-term, potentially curative, therapeutic benefit.
The company is collaborating with the University of Pennsylvania’s (Penn) Gene Therapy Program (GTP), led by Dr. James Wilson, to utilize the GTP’s world-class translational expertise and infrastructure, which has helped generate our initial pipeline of product candidates. The Penn collaboration provides iECURE with the exclusive right to develop up to 15 product candidates each focused on a liver indication; we have nominated three product candidates to date, which comprise our initial pipeline.
Position overview
The Clinical Trial Manager (CTM)/Senior CTM will report directly to the VP of 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 clinical trials and provision of necessary operational information to prepare for the company’s regulatory product submissions.
This individual will be accountable for executing on the operational plan for clinical studies and leading the delivery of these studies according to time, cost and quality targets. The Senior Clinical Trial Manager 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 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.
Key Responsibilities
- Manage the operational aspects of clinical trials
- Manage vendors enlisted in clinical trials to ensure timely and quality delivery of services, including necessary documents such as site manuals
- Manage CRO interactions
- Facilitate CRO/vendor selection process for outsourced activities
- 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 global and site levels
- Proactively identify potential study issues/risks and recommend/implement solutions
- Maintain systems (eTMF, CTMS, EDC, etc.) that track progress throughout the duration of the program
- Manage clinical monitoring activities ensuring compliance with Good Clinical Practices (GCP) and applicable regulations
- Ensure appropriate clinical trial supplies are planned and ordered in time and supply plans are implemented and managed
- Develop and conduct vendor and site training materials for disease state and clinical protocol objectives
- Work with CRO to develop and revise scope of service agreements, budgets, plans and detailed timelines, and ensure that performance expectations are met
- Hold CROs accountable to clinical study timelines for start-up, patient recruitment strategies, enrollment, and ensuring quality data deliverables
- Report progress of key study milestones in clinical program’s Project Team meetings
- Collaborate with vendor partners and internal functions to develop and execute strategies to expedite enrollment of clinical studies
- Oversee study operational excellence, monitoring of clinical trial sites to ensure quality data deliverables
- Ensure supporting documentation (e.g., case report forms, monitoring plans, data management plans, safety plans, clinical study reports), protocol objectives and operational efficiencies are followed
- Work collaboratively with the Finance team on clinical trial accruals and budget updates on a quarterly or as needed basis
- Accountable for the ongoing review of EDC data and issuance of queries in collaboration with CROs/vendors
- Ensure completion of accurate and timely clinical study reports for use by internal and external customers
- 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 external sites as needed
- Maintain a Clinical Operations Dashboard to inform management of weekly progress of each phase of the clinical operation process including: site initiation, IRB/EC approval, patient identification, enrollment and dosing
Qualifications
- A BA/BS degree required; advanced degree with scientific or health-care training preferred
- 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
- Minimum 5 – 8 years of related clinical trial management experience
- Experience in managing outside vendors and CROs
- Ability to manage global clinical operations for a product through all clinical phases of clinical development
- Experience developing, implementing, and building clinical operations infrastructure, including SOPs, and managing vendors
- Demonstrated leadership in a clinical operations role, 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
- An effective leader 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 25%.
iECURE, Inc. 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.
Senior Director / Director, Manufacturing Sciences & Technology Lead
Location: Plymouth Meeting, PA
Type: Full time
Company Overview
iECURE (pronounced EE-ah-cure) is a preclinical stage gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, therapy for the treatment of monogenic liver disorders with significant unmet need. iECURE’s approach has the potential to replace and restore the function of a dysfunctional gene by knocking-in a healthy copy, regardless of mutation, to offer durable gene expression and long-term, potentially curative, therapeutic benefit.
The company is collaborating with the University of Pennsylvania’s (Penn) Gene Therapy Program (GTP), led by Dr. James Wilson, to utilize the GTP’s world-class translational expertise and infrastructure, which has helped generate our initial pipeline of product candidates. The Penn collaboration provides iECURE with the exclusive right to develop up to 15 product candidates each focused on a liver indication; we have nominated three product candidates to date, which comprise our initial pipeline.
Position Summary
Reporting directly to the Head of Technical Operations, the Manufacturing Sciences & Technology (MS&T) Lead will build out capability to manage the life cycle of the manufacturing process in support of discovery, development, and manufacturing of iECURE’s product portfolio from the clinic through commercial launch. This individual will manage the development, transfer, validation and control of raw materials, plasmids, and cell/bacterial banks in support of the manufacturing of drug substance and drug products.
This individual will be hands-on, strategic-minded, diplomatic and a highly experienced technical leader, who can work effectively with the founding scientific team at Penn’s Gene Therapy Program (GTP). This leader will provide strategic guidance on global regulatory submissions and support the CMC package into the clinic under the stewardship of iECURE.
The MS&T Lead will be a seasoned professional who has held the lead role in a company defining the material and process 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
- Lead the technical development, validation, and continued verification of the manufacturing process inclusive of optimization, reproducibility, reliability, robustness, and improvements related to yield and product quality.
- Provide strong scientific background and have deep understanding of phase appropriate process development studies, data analysis, GMP requirements, ICH, and FDA guidance.
- Work with CDMOs in technical transfer, design of experiments, implementation, bridging, comparability, and validation employing phase appropriate approaches at the different stages of process development.
- Develop an approach for characterization to support identification of CPP/CQAs. Develop process analytical technology (PAT) to design, define, analyze, and control the manufacturing process.
- Lead CDMO activities: new product introduction, planning and scheduling, bill of material (BOM) and material management, sample plans, process descriptions and flow diagrams, master batch records, operational and general procedures, facility/equipment fit, training, right-first time (RFT) batch execution, process monitoring, data analysis, on-the-floor troubleshooting, investigation/deviations, impact assessments, corrective and preventative actions (CAPA), change controls, effectiveness checks, and overall continuous improvements and operational excellence.
- Provide aseptic and sterile processing expertise.
- Support in-process manufacturing as person-in-plant and QA batch release.
- Provide input, draft and review of studies, gap analyses, risk assessments, master records, protocols, memos, data, impact assessments, and reports.
- Support the stability program.
- Help prepare the CMC sections for regulatory filings. Help manage, direct, and monitor 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 process technologies.
- Build a collaborative working relationship with Penn GTP’s CMC organization and provide guidance on process 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 and Qualifications
- An advanced degree is preferred, such as a PhD or MS, in chemistry, biochemistry, or a relevant discipline.
- 10+ 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.
- Strategic/forward-thinker and CMC technical expert. Experience in a successful project leadership role supporting the developing, supplying, and registering of candidate drugs through market approval.
- Outstanding accomplishments interacting with regulatory agencies, specifically with the FDA and international regulatory authorities.
- Proven track record of successfully leading technology transfer projects.
- Experience in developing and scaling manufacturing, with a stellar track record managing 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 think at a strategic, big-picture level and delve into details when appropriate.
- Positive attitude and change agent capable of working and leading 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.
iECURE, Inc. 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.
Senior Director / Director, Analytical Development and Testing Lead
Location: Plymouth Meeting, PA
Type: Full time
Company Overview
iECURE (pronounced EE-ah-cure) is a preclinical stage gene editing company focused on developing therapies that utilize mutation-agnostic in vivo gene insertion, or knock-in, therapy for the treatment of monogenic liver disorders with significant unmet need. iECURE’s approach has the potential to replace and restore the function of a dysfunctional gene by knocking-in a healthy copy, regardless of mutation, to offer durable gene expression and long-term, potentially curative, therapeutic benefit.
The company is collaborating with the University of Pennsylvania’s (Penn) Gene Therapy Program (GTP), led by Dr. James Wilson, to utilize the GTP’s world-class translational expertise and infrastructure, which has helped generate our initial pipeline of product candidates. The Penn collaboration provides iECURE with the exclusive right to develop up to 15 product candidates each focused on a liver indication; we have nominated three product candidates to date, which comprise our initial pipeline.
Position Summary
Reporting directly to the Head of Technical Operations, the Analytical Development (AD) and Testing Lead will build out capability to manage 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 hands-on, strategic-minded, diplomatic and a highly experienced technical leader, who can work effectively with the founding scientific team at Penn’s Gene Therapy Program (GTP). This leader will provide strategic guidance on global regulatory submissions and support the Chemistry, Manufacturing and Controls (CMC) package into the clinic under the stewardship of iECURE.
The AD and Testing Lead will be a seasoned professional who has held the lead role in a company defining the analytical 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
- Lead 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.
- Develop 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.
- Review 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.
- Support 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.
- Oversee the stability program and ensure it supports clinical development and regulatory objectives and timelines.
- Help prepare the CMC sections for regulatory filings. Help manage, direct, and monitor 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
- An advanced degree is preferred, such as a PhD or MS, in chemistry, biochemistry, or a relevant discipline.
- 10+ 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
- Strategic/forward-thinker and CMC technical expert. Experience in a successful project leadership role supporting the developing, supplying, and registering of candidate drugs through market approval.
- Outstanding accomplishments interacting with regulatory agencies, specifically with the FDA and international regulatory authorities.
- Proven track record of successfully leading technology transfer projects.
- Experience in developing and scaling manufacturing, with a stellar track record managing 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 think at a strategic, big-picture level and delve into details when appropriate.
- Positive attitude and change agent capable of working and leading 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.
iECURE, Inc. 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.