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.
Senior Clinical Research Associate, Onsite, East, US Region
Location: On-site, Blue Bell, 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
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.
Specific 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
Education & 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%.
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 Research Associate, Remote, West, US Region
Location: Remote, West Coast
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
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.
Specific 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
Education & 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%.
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, Supply Chain Lead
Location: On-site, Blue Bell, 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 Supply Chain Lead will manage supporting the life cycle of discovery, development, manufacturing, and distribution of iECURE’s product portfolio from the clinic through commercial launch. This individual will manage and oversee planning, procurement, inventory, and logistics and distribution of materials, supplies and product at a global scale.
This individual will be hands-on, strategic-minded, diplomatic and a highly experienced leader, who can work effectively with senior management, functional leaders, vendors and external suppliers. This leader will provide strategic guidance on how to operate in a global market providing collaborative relationship management that enables, accelerated right-first-time operational success.
The Supply Chain Lead will be a seasoned professional who has held the lead role in a company defining and executing planning, procurement, inventory management, logistics and distribution in support of all phases of drug development, manufacturing, product storage and distribution. 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 advancement of proactive, robust, cost-effective material planning and procurement strategies to ensure compliant supply of materials and patient product.
- Lead the oversight of CDMOs/CROs and fulfillment vendor execution of planning, procurement, inventory management, storage, logistics and distribution inclusive of couriers, specialty couriers, VAT, and customs on a global scale.
- Build and manage strong relationships with vendors and external suppliers in support of technical and clinical operations.
- Facilitate all supply chain processes, collaborating with functional leaders in Operations, Quality and Finance and senior management to ensure availability of real-time, comprehensive data, information, analysis, risk assessments and reports.
- Provide strong background and have deep understanding of phase appropriate supply chain policies and procedures in accordance with GMP requirements, ICH and FDA guidance, and other laws and regulations.
- Support the preparation of regulatory filings and assist in the submission and resolution of agency related questions.
- Remain at the forefront of relevant global supply chain and competitive landscape best practices.
- Build a collaborative working relationship with Penn GTP’s organization and provide guidance on supply chain activities to produce the most efficient and effective outcomes possible for iECURE.
- Collaborate with operations and other internal functions to ensure the management of all financial aspects including budgeting/forecasting, long-range planning, quantitative decision making, and overall cost management and accountability of vendors and suppliers of materials and product.
Education & Qualifications
- BS/BA degree in Business, Science or Engineering.
- An advanced degree, such as an MBA, is preferred.
- Professional qualification in supply chain management is desired.
- 10+ years of experience in the biotech industry with experience in all stages supporting development, clinical and commercial manufacturing.
- Knowledge and experience in cell and gene therapy is desired.
- Strategic/forward-thinker supply chain expert. Experience in a successful project leadership role supporting the development, supply, and registration 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 projects.
- Experience in developing and scaling manufacturing, with a stellar track record managing third parties and CDMOs/CROs, couriers and specialty couriers, and dealing with customs 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 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.