Open Positions

Come join our team of talented and passionate individuals to help the nano-rare patient community.

n-Lorem Foundation

The mission of the foundation is to take advantage of the efficiency, versatility and specificity of antisense technology to provide charitably investigational antisense oligonucleotide (ASO) drugs to treat patients with nano-rare diseases (n1-30 patients). We plan a collaboration between the Foundation, the Undiagnosed Disease Network (UDN), investigators and institutions that comprise the UDN and Ionis Pharmaceuticals. Although each partner is fully independent, to maximize the efficiency with which efficacious therapies may be delivered to patients with rare diseases, there will be substantial interaction among the three partners.


Research Associate

RESEARCH ASSOCIATE

Reporting to: Director of Research and Development

Location: El Cajon, CA

Job Overview

  • Perform tissue culture of common human cell lines as well as reprogrammed cells and ipsc derived cells.
  • Perform drug treatment assays including transfection and free uptake.
  • Maintain a well-organized, clean, and safe lab working environment.
  • Maintain inventory, ordering supplies, and ensuring laboratory equipment is performing within specifications.
  • Plan, execute and analyze experiments under supervision.
  • Summarize and report experimental data and relevant documents to supervisors.
  • May present data in group meetings.
  • Other responsibilities as assigned.

Requirements

  • US work authorization.
  • >1 year experience establishing/performing molecular/cellular/biochemical assays.
  • Hold a B.A/B.S. degree in Biology or related field.
  • Have strong attention to detail.
  • Possess excellent written and verbal communication skills.
  • Demonstrate an ability to work as part of team and possess an excellent work ethic.
  • Have the ability to be successful in a fast-paced, intense work environment.

Preferred Qualifications

  • Experience troubleshooting molecular/cellular biology techniques and/or biochemical assays
  • Experience with high throughput gene transfection, high throughput RNA isolation, and high throughput real-time PCR.
  • Experience with liquid handling automated workstations.
  • Industry experience with ASO/RNAi technology.
  • Knowledge of biostatistics.
  • Work experience with CROs.

To apply for this position please click the link below.

Senior Research Associate

SENIOR RESEARCH ASSOCIATE

Reporting to: Director of Research and Development
Location: El Cajon, CA

Job Overview

The successful candidate will work within a fast-paced, interactive, and innovative research team to answer key mutation-dependent questions using outstanding molecular/cellular/biochemical techniques to discover and validate novel antisense drugs for nano-rare diseases. Responsibilities will include assisting with strategic planning, design, execution, trouble-shooting, and analysis of in vitro experiments to evaluate potential clinical development compounds in addition to disease relevant biology in human cell culture systems. We are looking for a highly motivated individual with strong attention to detail, capable of multi-tasking and who would succeed in a fast-paced atmosphere.

Requirements

  • US work authorization.
  • PhD degree in Biology, or a related field.
  • > 2 years of postdoctoral or professional experience in an academic or industry research setting, with a proven track record of accomplishments, including publications in top tier scientific journals.
  • Demonstrated experience with culturing human cell lines, fibroblasts, and ips-derived cells is required.
  • Demonstrated experience performing screening assays and analysis.
  • Essential laboratory skills including RNA and protein analysis such as qPCR, Western blot, and ELISA are required. Additional in vitro skill sets are highly desired including automation.
  • Translational biology experience developing biomarkers are a plus.
  • Have strong interpersonal skills including a proven track record of building and maintaining effective working relationships with teams and partners.
  • Have the ability to manage multiple assignments effectively in a quickly evolving environment.
  • Outstanding communication and team skills.
  • Self-starter who is highly motivated and not afraid to take risks

To apply for this position please click the link below.

Associate Director/Director, Drug Substance and Drug Product Development, CMC

Associate Director/Director, Drug Substance and Drug Product Development, CMC

Reporting to: Director of Preclinical Development
Location: El Cajon, CA – Decentralized

Job Overview

n-Lorem is seeking an experienced, organized, and dedicated individual to join our team as an Associate Director/Director, Drug Substance and Drug Product Development, CMC.

This critically important person will be the n-Lorem leader for all aspects related to CMC. A key accountability will be to maintain alignment with the CMOs as together we forge the most efficient and effective regulatory path to enabling individualized treatment of patients in need. This person will lead strategic planning and execution of all aspects related to CMC. A successful candidate will preferably have experience with development of antisense oligonucleotides (ASO) and submission of research INDs and/or other regulatory submissions, experience with US and/or international regulatory interactions, and strong knowledge of GMPs and state of the art quality systems.

Job Duties

  • Develop and lead the strategic CMC Development Plan for all products in the pipeline
  • Set priorities, establish team goals, and plan activities to deliver the CMC strategy for all programs
  • Effectively manage early-stage drug substance development as well as drug product activities working in a virtual environment.
  • Draft RFPs and tech transfer packages, review and provide feedback of batch records and development reports, manage internal timeline, and monitor development and manufacturing progress.
  • Efficiently support cross functional project teams. Coordinate drug substance activities with analytical and drug product development activities within CMC. Understand and support the needs and requirements from other functional groups including toxicology and regulatory. Provide updates to project and management teams.
  • Drive decision making and the associated deliverables across all functions on the CMC team
  • Identify, and escalate key issues and risks to ensure prompt resolution and optimal development path
  • Participate in budget planning and forecasting for CMC expenditures (including expenditures with CDMOs and consultants)
  • Draft drug substance and drug product sections for regulatory filings (including INDs, briefing packages or other regulatory dossiers)

Requirements

  • US work authorization
  • Ph.D. in chemistry or closely related field with a minimum of 5 years’ experience, or equivalent combination of degree and related ASO research and development experience.
  • Prior experience in technical management of domestic and international CDMOs is strongly desired
  • Good understanding of CGMP requirements and ICH Quality guidances
  • Well versed with the latest ASO technologies and the outsourcing industry
  • Excellent writing skills as they relate to preparation of RFPs, protocols, reports and regulatory filings
  • Excellent interpersonal and communication skills (written and oral). This includes the ability to present data effectively and accurately to peers, management, and external partners.
  • Excellent ability to work in a goal and team-oriented setting and handle competing priorities. Flexibility within a rapidly changing environment and high attention to details.
  • Well-developed organizational skills and the ability to thrive under pressure
  • Intermediate to advanced software skills (e.g., Office 365, Microsoft Excel, PowerPoint).
  • Experience with chemistry data base software (i.e., SciFinder, ChemDraw)
  • Some travel may be required

References

  • Initial FDA guidance for ASO for patients with diseases caused by ultra-ultra-rare mutations: Jan. 4, 2021
  • Pre-clinical requirements: Detailed guidance April 2021
  • CMC guidance Dec 2021
  • Clinical guidance Dec 2021

To apply for this position please click the link below.

Director of Regulatory Affairs

DIRECTOR OF REGULATORY AFFAIRS

Reporting to: Chief Development Officer
Location: El Cajon, CA Decentralized

Job Overview

n-Lorem is seeking an experienced, organized, and dedicated individual to join our team as a Director of Regulatory Affairs. 

n-Lorem is a non-profit foundation developing individualized treatments for patients who will be treated under investigator-initiated research INDs in alignment with the Individualized Antisense Oligonucleotide guidance documents (4) released in 2021. This critically important role will be the n-Lorem regulatory leader.  A key accountability will be to maintain alignment with the regulatory agencies as together we forge the most efficient and effective regulatory path to enabling individualized treatment of patients in need.  This position will lead strategic planning and execution of all aspects related to regulatory affairs.  A successful candidate will preferably have experience with preparation and submission of research INDs and/or other regulatory submissions; and experience with US and international regulatory interactions.

Responsibilities in Regulatory Strategy and Leadership

  • Lead and execute on regulatory strategies for individualized treatment programs for single patients and their lifecycle management
  • Represent Regulatory expertise across the organization and to external partners
  • Provide strategic guidance to teams on the regulatory requirements, precedent, and risk to support clinical development programs
  • Monitors, analyzes, and disseminates intelligence on regulatory matters that may affect ongoing development programs

Responsibilities in Regulatory Regulatory Operations

  • Lead interdepartmental and cross-organizational teams in the authoring of high-quality regulatory communications such as regulatory meeting requests, briefing packages, responses to regulatory health authority requests, and IND components.
  • Lead preparations for, and participate in, meetings with regulatory agencies to ensure efficient drug development, product license approvals and their maintenance
  • Plans, prepares and reviews submissions to regulatory authorities including FDA, Health Canada, EMA, MHRA and other global health authorities to support the conduct of investigator-initiated studies of individualized antisense oligonucleotides, in partnership with the investigator and treating institution
  • Able and willing to participate in writing of the regulatory communications and submissions to support the functional area experts when needed
  • Provide technical review of data or reports that will be incorporated into regulatory submissions to assure scientific rigor, accuracy, and clarity of presentation
  • Ensure regulatory documentation is maintained appropriately
  • Prepares and revises internal regulatory procedures for continuous improvement

Responsibilities in Partnering with Key Stakeholders

  • Establishes robust partnerships with investigators and shepherds them through the process of drafting, submitting and holding a research IND related to treating their patient with an individualized ASO
  • Collaborates closely with the n-Lorem team and clinical research partner to execute on all regulatory strategies efficiently land effectively
  • Other duties as assigned

Requirements

  • US work authorization
  • PhD degree in a scientific discipline required; advanced degree preferred, or a related field
  • Minimum of 10 years of experience working in the pharmaceutical/biotechnology industry with direct experience in Regulatory Affairs and demonstrated ability to present and articulate requirements and influence teams
  • Demonstrated Experience in preparation and submissions of INDs and/or other regulatory applications
  • Familiarity with the interpretation of applicable FDA, Canadian, EU, UK and ICH guidelines related to clinical trial and marketing applications
  • Exercise outstanding judgment in all areas of responsibility
  • History of successful interactions with regulatory authorities
  • Proven ability to effectively work collaboratively in cross-functional teams and lead team discussions
  • Highly organized and able to project manage the regulatory components of an ASO program to meet agreed upon milestones
  • Energetic, self-motivated and a hands-on professional with a strong work ethic
  • Excellent communication and strong interpersonal skills including a proven track record of building and maintaining effective working relationships with teams and partners
  • Have the ability to manage multiple assignments effectively in a quickly evolving environment
  • Willing to travel domestically and internationally

References

  • Initial FDA guidance for ASO for patients with diseases caused by ultra-ultra-rare mutations: Jan. 4, 2021
  • Pre-clinical requirements: Detailed guidance April 2021

To apply for this position please click the link below.

Medical Geneticist

MEDICAL GENETICIST

Reporting to: Chief Development Officer
Location: El Cajon, CA Decentralized, or peripheral to leading academic institution

Job Overview

n-Lorem is seeking a trained, experienced clinician (MD) with scientist training (PhD preferred) in human genetics. In this role, you will partner closely with the Chief Executive Officer, Chief Medical Officer, Chief Technical Officer, Senior Director of Research, and Chief Development Officer to evaluate patients’ genetic and clinical characteristics of determine if n-Lorem’s technology has a high likelihood of success when targeting the patients’ mutation and the benefit/risk ratio is favorable. This candidate will establish an intimate knowledge of the patients’ clinical characteristics to enable monitoring the benefit/risk ratio over the course of the drug discovery and development period to ensure appropriate decisions in partnership with the treating physician.

The successful candidate will exemplify through a deep understanding of the n-Lorem mission as it applies to the existing n-Lorem patients and their ASO Programs, as well as to the many more patients n-Lorem will aim to help in the future. You will provide the day-to-day medical expertise

to the n-Lorem team and a primary clinical point of contact for the treating physicians to ensure their needs are met during both the pre-treatment and treatment period.

Key Responsibilities

  • Provide core clinical expertise in rare diseases
  • Ensure a deep understanding of a patient’s clinical manifestations during the application process to drive benefit/risk decisions related to acceptance into the program
  • Maintain close relationships with treating physicians before and during patient treatment to continually assess the patient status as it relates to the ASO treatment
  • Assess patients’ genetic mutations, confirm causation and assess evidence of mechanism of the mutation to frame an ASO strategy
  • Assess clinical data collected for the treatment goals and support physician in driving treatment decision related to benefit/risk ratio
  • Shepherd physicians through the n-Lorem process from the point of considering a patient for application through to long-term follow-up.
  • Act as the primary n-Lorem clinical contact and support for all physicians
  • Collaborate closely with internal and external experts to support recommendations for treatment outcomes
  • Contribute to authoring clinical research documents including study protocols, informed consent, and regulatory documents (pre-IND briefing document and IND)

Requirements

  • US work authorization
  • Education & Experience: MD required, PhD or scientific training and experience in genetics or a related discipline
  • Minimum of 1-3 years of experience working as an MD required with scientific training and/or experience in human genetics.
  • Experience and/or knowledge of RNA therapeutics a plus
  • Responsible for upholding the highest standards of leadership, integrity and patient-centricity in their day-to-day behaviors
  • Exercise outstanding judgment in all areas of responsibility
  • Proven ability to effectively work collaboratively in cross-functional teams and lead team discussions
  • Energetic, self-motivated and a hands-on professional with a strong work ethic
  • Excellent communication and strong interpersonal skills including a proven track record of building and maintaining effective working relationships with teams and partners
  • Have the ability to manage multiple assignments effectively in a quickly evolving environment
  • Willing to travel domestically and internationally
  • Completed residency and fellowship in fields relevant to the mission of n-Lorem
  • Solid knowledge of genetic underpinning of disease, inheritance patterns, and natural history assessments
  • Experience working as part of a dynamic team, with shifting priorities, and complex medical situations
  • Excellent publication record
  • Extensive knowledge of genetic databases including OMIM, ClinVar, GeneTests, GnomAD, ClinicalTrials.gov, and interpretation of various mechanisms by which mutations can lead to altered cellular function
  • Extensive knowledge in interpretation of whole exome and whole genome clinical sequencing
  • Knowledge of molecular and cellular biology, disease pathogenesis, and medical statistics
  • Board certified or board eligible in specialty

References

  • Initial FDA guidance for ASO for patients with diseases caused by ultra-ultra-rare mutations: Jan. 4, 2021
  • Pre-clinical requirements: Detailed guidance April 2021

To apply for this position please click the link below.

Director,  Development, Clinical and Corporate

DIRECTOR, DEVELOPMENT, CLINICAL AND CORPORATE OPERATIONS

Reporting to: Chief Development Officer
Location: Carlsbad, CA, or Decentralized / El Cajon, CA Hybrid

Job Overview

n-Lorem is seeking a passionate, organized and action-oriented individual to join our team as a Director of Development, Clinical and Corporate Operations. In this role, you will partner closely with the Chief Development Officer, Senior Director of Operations, and Senior Clinical Research Specialist and operational partner CRO.   As a strategist and implementer, the successful candidate will exemplify simplicity and organization through a deep understanding of the n-Lorem process and portfolio of patients’ ASO Programs.  You will ensure activities are streamlined within the organization based rapidly evolving development and operational priorities. In addition, you will act as a catalyst by bringing expertise and dedicated focus on process improvement and efficiency initiatives. In your day-to-day interactions, you will advance all n-Lorem Patient ASO programs and business operations activities to meet the needs of our patients and internal team.

  

This position will also support executive team members in their roles by taking over some tactical responsibilities or handling other day-to-day items. For example, those duties might include leading and implementing various strategic planning processes, preparing for upcoming meetings or presentations, calendaring, tracking, reporting activities and deliverables for objectives and specific initiatives, facilitating meetings and workshops and supporting a variety of operational and organizational matters.   In general, you will assist the development and operations leadership in ensuring that their business runs smoothly. This may include leading processes like objective setting, results and milestone tracking, budget/headcount planning, and required communications. 

Key Responsibilities

  • Synthesize all elements of project management and leadership across the n-Lorem process and portfolio by leveraging experience in efficiencies into a single streamlined solution that is easily accessible and usable for all staff
  • Create operating principles and processes to align the organization towards the highest priority work supporting critical department and corporate goals. 
  • Spearhead process establishment, improvement, and efficiency projects. Define where to focus time, attention, and energy to achieve quarterly/annual goals. Help define an approach to tackle complex, multi-layered questions, striking a delicate balance between responsiveness, action orientation, and the need for delicate interpersonal problem-solving.
  • Build relationships at every level within the organization. A critical part of your role will be to establish and maintain a web of stakeholder interactions within n-Lorem and with all of our partners, factoring in nuanced viewpoints, histories, and personalities. You will be frequently called upon to lead and shape facilitation approaches for these stakeholders and guide leaders in interactions and strategies to influence and get things done. Think strategically while providing tactical support to implement ideas. 
  • Develop and maintain a centralized location for all Corporate, Clinical and Development Operations dashboards, metrics reports, tools, and databases.
  • Collaborate with team members to determine key performance indicators, measure performance, and incorporate them into a centralized location. Track decisions and follow up on critical actions. 
  • Proactively address pressing business issues that need immediate attention. Proactively escalate and resolve potential issues by calling them to the department head’s attention and identifying possible solutions. 
  • Analyze and report performance based on strategies and business goals and recommend areas needing improvement. Improve overall business acumen and governance (performance metrics), operational, organizational, and financials.  Assess the need to build an operating system — the goal-setting processes, meeting structure, and analytics — that would enable our executives and team leaders to be as focused and successful as possible. 
  • Expand and extend the leverage of senior management on mission-critical projects that don’t fit into any team’s responsibility or that would otherwise fall through the cracks. 

Skills & Qualifications 

A successful Director of Corporate, Clinical & Development Operations will have specific prerequisite skills and qualifications, which include: 

  • Communication: regularly works with team members of all levels, so strong written and verbal communication skills are crucial.  
  • Leadership: acts as a leader, planning meetings and determining the needs of the business. A strong team player who supports their team and embraces ownership, accountability, and responsibility for the team’s work.  The ability to lead in an effective and trustworthy way is essential. Ability to influence decision making with or without authority, facilitate groups with diverse perspectives, across geographies, and bring teams to agreement. Demonstrated ability to build credible professional relations with SMEs, functional leaders, and Key Opinion Leaders .  
  • Time management: responsible for improving the efficiency of the business and managing the time of the executive leaders. Use skills in organization and planning to prioritize tasks, set meetings, and diffuse work conflict without bringing it to the attention of the executive leadership. 
  • Problem-solving: Whether it is conflict within the workplace or a problem with a vendor, customer, or partner, it is the role of the Director, Corporate & Development Operations to take the necessary steps to overcome problems their organization faces. Experience in influencing and building strong relationships with stakeholders and senior leaders across functions. Ability to act as a liaison between business stakeholders and subject matter experts. Excellent customer service skills. Able to deal with ambiguity: can effectively cope with change; can shift gears comfortably; can decide and act without having the total picture; can comfortably handle risk and uncertainty. Exceptional influence without authority and excellent collaboration skills. 

Education & Experience:

  • PMP and / or  MBA, preferred
  • clinical background with relevant experience (10yrs) in academia, consulting, and biotech/pharmaceutical industry. 
  • Experience and knowledge of end-to-end management of clinical trial conduct, knowledge of the pharmaceutical industry and an understanding of clinical drug development, clinical trials operations and regulatory components is essential.
  • Experience with Investigator Initiated Studies is a plus; A thorough understanding of drug development from IND to NDA – is a plus

To apply for this position please click the link below.

n-Lorem offers a competitive benefits package including medical, dental, vision, 403(b) and 4 weeks paid vacation. n-Lorem is a small foundation with an extraordinary mission, to provide hope and potentially help to nano-rare patients today.  Every employee in our organization is a significant contributor to this mission. We know that our work could have a profound impact on the life of a patient today. 

COVID-19 update:

We  are  pleased  that  100%  of  n-Lorem  employees/temps  who  have  reported  their vaccination status are vaccinated against the COVID-19 virus.

Additional COVID-19 precautions

All   employees,   and   contractors/consultants   regularly   working   on-site   are   strongly encouraged to be fully vaccinated against the COVID-19 virus.

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