Careers
Come join our team of talented and passionate individuals to help the nano-rare patient community.
Careers at n-Lorem
Join our team at n-Lorem Foundation
The mission of the foundation is to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental ASO medicines to treat patients with nano-rare diseases.
Though n-Lorem is pioneering a novel non-profit model, to provide personalized experimental ASO treatments for free, for life to patients with the rarest of mutations (nano-rare), we are functionally a biotechnology company. We have a large and growing portfolio of ASO medicine discovery programs, ASO medicines in development and multiple clinical programs.
With a seasoned leadership team and strategic partnerships, the n-Lorem Foundation provides the framework, funds and access for nano-rare patients who are amendable to our technology to receive personalized ASO medicines for free, for life. We hope that you will consider joining us as we strive to change the world, one nano-rare patient at a time.
Open Positions at n-Lorem
Strategic Operations Manager / Sr. Strategic Operations Manager
n-Lorem is seeking a passionate, organized, detail- and action-oriented individual to join our team as Strategic Operations Manager, partnering directly with the Chief Operating Officer. This role will work across functional teams to support operational alignment, execution of key initiatives, and coordination of organizational priorities. This role will require the ability to multi-task, pivot and be flexible in a range of duties ranging from administrative, to project manager, to communication and PR-like activities. This role functions similarly to a junior Chief of Staff position, helping the COO stay one step ahead by tracking priorities, preparing materials for leadership discussions, coordinating cross-functional workstreams, and ensuring commitments translate into action.
n-Lorem offers a competitive benefits package including medical, dental, vision, 403(b) with a match 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 precautions
All employees, and contractors/consultants regularly working on-site are strongly encouraged to be fully vaccinated against the COVID-19 virus.
Reporting to: Executive Director of Clinical Development
Hours: Full Time
Location: On-site in Sorrento Valley, CA
Compensation: $70k – $110k / annual+ benefits
Job Overview
n-Lorem is seeking a passionate, organized, detail- and action-oriented individual to join our team as a Clinical Scientist. In this role, you will partner closely with the clinical development, regulatory and clinical operations/project management teams. You will help set up, manage and track n-Lorem’s clinical trials, including assisting in protocol and informed consent development, data monitoring, and the preparation of clinical data for internal and external presentations. You will also support the development of Investigational New Drug (IND) applications, and various regulatory activities. You will be expected to wear many hats and will have the opportunity to expand the scope of your work and responsibilities over time.
You will work across many functional teams which will require clear and timely communication, and the ability to quickly adjust to changing priorities. No two days will be the same, therefore flexibility and self-motivation will be key aspects for a candidate to be successful in our fast-paced biotech environment.
Job Duties
- Assist in the development of clinical materials, including protocols, informed consent documents, IND sections, and site initiation materials
- Assist in the development and implementation of natural history studies
- Ensure timely data entry and assist with facilitating data resolution queries from the sites.
- Track outcomes assessments used across all clinical trials
- Track upcoming clinical milestones
- Provide literature updates on areas relevant to clinical development such as collection of phenotypic data in clinical trials, clinical updates from ASO trials, new outcomes measures
- Assist in compiling important information on rare diseases, such as incidence rates, disease characteristics
- Work with clinical development team on preparing data reports from all n-Lorem programs for presentation to internal and external teams
- Develop, maintain, and improve templates for various documents including clinical protocols, informed consents, clinical trackers
- Proactively identify inefficiencies and propose possible solutions to enhance clinical development operations
- Provide general support for n-Lorem scientific and clinical publications
- Other duties as assigned to support our quickly growing multi-disciplinary team
Requirements
- US work authorization
- BSc, MS or PhD degree in a scientific discipline required, preferably rare disease
- Minimum of 3 years of experience working in the pharmaceutical/biotechnology industry / clinical site
- Familiarity with clinical trials and ICH guidelines related to clinical trials
- Understanding of working with rare disease populations
- Exemplify discipline in meeting deadlines and deliverable on a daily basis
- Demonstrated ability to rapidly acquire and apply new skills and concepts
- Excellent interpersonal and communication skills and effective cross-functional collaborations
- 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
- Proficient software skills (e.g., MS Office including Word, Excel, PowerPoint), with experience in Prism, PowerBI, and Electronic Data Capture systems preferred.
To apply for this position please click the link below.
Location: on-site in Sorrento Valley (San Diego, CA 92121)
Job Overview
We are seeking a highly motivated Scientist to join our dynamic and innovative Discovery and Research team focused on developing novel antisense therapies for nano-rare diseases. The successful candidate will play a critical role in advancing our ASO Discovery pipeline while answering key research questions using cutting-edge molecular, cellular, and biochemical techniques. This is an exciting opportunity for an individual with expertise in cell and molecular biology, oligonucleotide drug discovery, and high-throughput screening capabilities. The ideal candidate will have a strong attention to detail, the ability to manage multiple priorities and deliverables, and thrive in a fast-paced, collaborative environment.
Key Responsibilities
- Execute, troubleshoot, and analyze in vitro screening experiments within our Discovery pipeline to evaluate potential clinical compounds.
- Work closely with cross-functional teams to ensure smooth communication and alignment, contributing to the strategic advancement of our research goals.
- Plan and conduct cellular and molecular biology experiments to advance our understanding of antisense oligonucleotide
- Adapt quickly to evolving priorities and provide timely updates and clear communication on research progress.
- Collaborate with internal teams to design and execute experiments that will inform decision-making and drive progress in the development of novel therapeutics.
Requirements
- US work authorization is required.
- Bachelor (BSc) or Master’s degree (MSc) or equivalent experience in oligonucleotide research and discovery. 2+ years of industry experience is strongly preferred
- Drug discovery experience is strongly preferred.
- Expertise with neuronal differentiation is strongly preferred
- A proven track record of working in a highly collaborative, team- and mission- oriented environment. Track record of accomplishments, including publications in top-tier scientific journals is preferred.
- Demonstrated expertise in culturing human cell lines, fibroblasts, and iPSC-derived cells.
- Strong experience in performing high-throughput screening assays and data analysis.
- Essential laboratory skills, including RNA and protein analysis techniques such as qPCR, Western blot, and ELISA. Additional in vitro skills, especially in automation are highly desirable.
- Excellent interpersonal skills, with a proven ability to build and maintain effective working relationships within teams and with external partners.
- Ability to manage assignments in a fast-paced, rapidly changing environment.
- Strong ability to collaborate effectively within a team.
- Self-starter with a high level of motivation, and a willingness to take initiative and embrace new challenges.
To apply for this position please click the link below.
Job Overview
n-Lorem is seeking a passionate, organized, detail- and action-oriented individual to join our team as Strategic Operations Manager, partnering directly with the Chief Operating Officer. This role will work across functional teams to support operational alignment, execution of key initiatives, and coordination of organizational priorities. This role will require the ability to multi-task, pivot and be flexible in a range of duties ranging from administrative, to project manager, to communication and PR-like activities.
This role functions similarly to a junior Chief of Staff position, helping the COO stay one step ahead by tracking priorities, preparing materials for leadership discussions, coordinating cross-functional workstreams, and ensuring commitments translate into action.
The successful candidate will bring strong organizational skills, attention to detail, and a proactive mindset. You will help create clarity, maintain momentum across initiatives, and ensure that key operational priorities are progressing efficiently.
No two days will be the same, and flexibility, responsiveness, and a positive attitude will be essential as an important member of our fast-paced and growing team.
Key Responsibilities
- Partner closely with the COO to:
- Advance priorities, initiatives, and commitments across the organization.
- Prepare materials, summaries, and follow-up documentation for leadership meetings and key internal discussions.
- Coordinate cross-functional initiatives in the US and globally to ensure clear ownership, timelines, and accountability.
- Track progress on operational priorities and proactively follow up to ensure action items are completed.
- Help structure and maintain effective operating rhythms, including leadership meetings and cross-functional updates.
- Identify potential bottlenecks or capacity conflicts and escalate issues with proposed solutions.
- Ensure tracking of all COO actions and timely follow-through to completion of tasks
- Support preparation of materials for executive updates, board discussions, and external or internal presentations or meetings.
- Assist in the development and improvement of operational processes, tools, and templates that increase organizational efficiency and clarity.
- Support special projects and initiatives as needed to ensure forward progress of key organizational priorities.
- Maintain strong documentation, communication and organizational practices to ensure institutional knowledge and transparency across initiatives.
- Collaborate across functions to advance joint strategic initiatives and objectives, primarily with the VP of ASO Discovery and Development, Executive Director of Clinical Development, VP of Foundation Development and External Relations, Senior Director of Clinical Operations and Program Management
Requirements
- Bachelor’s degree required; degree in business, life sciences, public health, or a related field preferred
- 3–6 years of experience in operations, program management, administration, in biotechnology, healthcare or a related field
- Strong organizational, project management, and time management skills
- Exceptional attention to detail and ability to manage multiple priorities simultaneously
- Strong written and verbal communication skills with the ability to synthesize complex information clearly
- Ability to work effectively in a fast-paced, cross-functional environment with multiple stakeholders
- Proactive, solutions-oriented mindset with a strong sense of ownership and accountability
- High level of professionalism, judgment, and discretion when working with leadership
- Willingness to travel in support of COO or n-Lorem Executive Committee
- Proficiency with Microsoft Office applications and operational tracking tools
n-Lorem offers a competitive benefits package including medical, dental, vision, 403(b), four weeks paid vacation, paid sick time, life insurance, employee assistance program.
n-Lorem is a small foundation with an extraordinary mission, to provide hope and potentially help to nano-rare patients. 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.
n-Lorem is an equal opportunity employer. All applicants will be considered for employment without attention to race, color, religion, sex, sexual orientation, gender identity, national origin, veteran or disability status. n-Lorem is committed to providing reasonable accommodations for candidates with disabilities in our recruiting process. If you need any assistance or accommodations due to a disability, please let us know.
For more information on n-Lorem, please visit our website www.nlorem.org
To apply for this position please click the link below.
Position Title: Senior Scientist
Reporting to: Associate Director, ASO Discovery
Location: on-site in 9410 Carroll Park Dr, San Diego, CA 92121
Employment: Full-time
Compensation: $120 – $160K
Job Overview
We are seeking a highly motivated Senior Scientist to join our dynamic and innovative Discovery and Research team focused on developing novel antisense therapies for nano-rare diseases. The successful candidate will report to Associate Director, ASO Discovery and will play a critical role in advancing our ASO Discovery pipeline while answering key research questions using cutting-edge molecular, cellular, and biochemical techniques. This is an exciting opportunity for an individual with extensive expertise in oligonucleotide discovery, cell and molecular biology, and mechanistic antisense research. The ideal candidate will have a strong attention to detail, the ability to manage multiple priorities, and thrive in a fast-paced, collaborative environment.
Key Responsibilities
- Design, execute, troubleshoot, and analyze in vitro screening experiments within our Discovery pipeline to evaluate potential clinical compounds.
- Plan and conduct cellular and molecular biology experiments to advance our understanding of core antisense technologies.
- Work closely with cross-functional teams to ensure smooth communication and alignment, contributing to the strategic advancement of our research goals.
- Adapt quickly to evolving priorities and provide timely updates and clear communication on research progress.
- Collaborate with internal teams to design and execute experiments that will inform decision-making and drive progress in the development of novel therapeutics.
Requirements
- US work authorization is required.
- Master’s degree (MSc) or equivalent experience in oligonucleotide research and discovery. A PhD is preferred but not mandatory. 2+ years of industry experience is strongly preferred
- Drug discovery experience is strongly preferred.
- Expertise with neuronal differentiation is strongly preferred
- A proven track record of accomplishments, including publications in top-tier scientific journals. Supervisory experience is highly desirable.
- Demonstrated expertise in culturing human cell lines, fibroblasts, and iPSC-derived cells.
- Strong experience in performing high-throughput screening assays and data analysis.
- Essential laboratory skills, including RNA and protein analysis techniques such as qPCR, Western blot, and ELISA. Additional in vitro skills, especially in automation are highly desirable.
- Excellent interpersonal skills, with a proven ability to build and maintain effective working relationships within teams and with external partners.
- Ability to manage multiple assignments simultaneously in a fast-paced, rapidly changing environment.
- Outstanding communication skills and the ability to collaborate effectively within a team.
- Self-starter with a high level of motivation, and a willingness to take initiative and embrace new challenges.
To apply for this position please click the link below.
Position Title: Human Resources Senior Manager/ Human Resources Associate Director
Reporting to: Vice President, Finance
Location: On-site (San Diego- Mira Mesa area)
Employment: Full-Time Exempt
Salary range: $120,000 – $180,000
(Compensation may vary based on related skills, experience, and relevant key attributes)
n-Lorem Foundation
Though n-Lorem is pioneering a novel non-profit model, to provide personalized experimental ASO treatments for free, for life to patients with the rarest of mutations (nano-rare), we are functionally a biotechnology company. We have a large and growing portfolio of ASO medicine discovery programs, ASO medicines in development and multiple clinical programs.
With a seasoned leadership team and strategic partnerships, the n-Lorem Foundation provides the framework, funds and access for nano-rare patients who are amenable to our technology to receive personalized ASO medicines for free, for life. We hope that you will consider joining us as we strive to change the world, one nano-rare patient at a time.
If you are a human resources professional with strong experience in the biotech/ life sciences industry and would like to join a cohesive, experienced team committed to the belief that we can change the world one patient, one family at a time, we may have a position for you.
Job Overview
Reporting to the Vice President, Finance, the HR Senior Manager/ HR Associate Director leads the organization’s HR operations, ensuring alignment with business objectives. This role oversees employee relations, compliance, compensation and benefits, talent management, organizational development, and HR policy implementation while fostering a positive, inclusive workplace culture.
Key Responsibilities
- Employee Relations & Culture
- Foster a positive, inclusive, and high-performing workplace culture
- Address employee relations issues, investigations, and conflict resolution
- Advise leadership on employee performance, discipline, and terminations
- Compliance & Risk Management
- Ensure compliance with federal, state, and local employment laws and regulations
- Develop, update, and enforce HR policies and procedures
- Lead audits, reporting, and risk mitigation related to employment practices
- Partner with Legal and external advisors on employment matters.
- HR Operations & Systems
- Update and implement HR systems and data reporting
- Compensation & Benefits
- Oversee compensation and benefits programs
- Talent Management
- Oversee recruitment, onboarding, and retention for scientific and corporate hires
- Update and implement onboarding programs
- Implement performance management and professional development programs
- Support workforce planning and headcount growth in collaboration with leadership.
- Promote leadership development and employee engagement initiatives
- Leadership & Training
- Serve as a partner to executive leadership on workforce planning and organizational development
- Serve as a hands-on HR partner for employees and managers.
- Coach managers on performance management, feedback, and development.
Key Requirements
- Education & Experience
- Bachelor’s degree in Human Resources, Business Administration, or related field
- 8–12+ years of progressive experience in biotech / life sciences industry
- Strong working knowledge of employment law and HR fundamentals.
- Skills & Competencies
- Strong knowledge of employment law and HR best practices
- Strategic thinking with strong execution capabilities
- Excellent leadership, communication, and interpersonal skills
- Proven ability to handle sensitive and confidential matters
- Experience with implementing HRIS platforms
- Experience supporting scientific teams
- Preferred Certifications
- SHRM-SCP or SHRM-CP
- SPHR or PHR
- Additional Requirements
- U.S. work authorization
To apply for this position please click the link below.
n-Lorem offers a competitive benefits package including medical, dental, vision, 403(b), four weeks paid vacation, paid sick time, life insurance, employee assistance program. n-Lorem is a small n-Lorem offers a competitive benefits package including medical, dental, vision, 403(b), four weeks paid vacation, paid sick time, life insurance, employee assistance program. n-Lorem is a small foundation with an extraordinary mission, to provide hope and potentially help to nano-rare patients. 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.
n-Lorem is an equal opportunity employer. All applicants will be considered for employment without attention to race, color, religion, sex, sexual orientation, gender identity, national origin, veteran or disability status. n-Lorem is committed to providing reasonable accommodations for candidates with disabilities in our recruiting process. If you need any assistance or accommodations due to a disability, please let us know.
For more information on n-Lorem, please visit our website www.nlorem.org
n-Lorem founder, chairman and CEO, Stanley T. Crooke, M.D., Ph.D., was presented with the 2022 Roy Vagelos Pro Bono Humanum Award for Global Health Equity of the Prix Galien. https://youtu.be/N7UwdQUZFmY
Position Title: Digital Marketing Specialist
Reporting to: Executive Director, Communications and Donor Relations
Location: On-site (San Diego- Mira Mesa area)
Employment: Full-Time
Salary range: $31.25- $36.00/hour
(Compensation may vary based on related skills, experience, and relevant key attributes)
n-Lorem Foundation
Though n-Lorem is pioneering a novel non-profit model, to provide personalized experimental ASO treatments for free, for life to patients with the rarest of mutations (nano-rare), we are functionally a biotechnology company. We have a large and growing portfolio of ASO medicine discovery programs, ASO medicines in development and multiple clinical programs.
With a seasoned leadership team and strategic partnerships, the n-Lorem Foundation provides the framework, funds and access for nano-rare patients who are amenable to our technology to receive personalized ASO medicines for free, for life. We hope that you will consider joining us as we strive to change the world, one nano-rare patient at a time.
If you are a professional with strong communications experience and would like to join a cohesive, experienced team committed to the belief that we can change the world one patient, one family at a time, we may have a position for you.
Job Overview
Reporting to the Executive Director Communications and Donor Relations, the Digital Marketing Specialist will be responsible for supporting and advancing n-Lorem’s digital presence across social media, website, podcast, and partner channels. This role focuses on executing and optimizing digital content and campaigns, while contributing ideas and insights that strengthen audience engagement. The specialist will create multimedia assets, maintain and improve the foundation’s website, oversee day-to-day social activity, organize fundraisers, support podcast production and promotion, and develop digital sponsor materials.
Key Responsibilities
- Social Media & Digital Execution
- Manage daily activity across LinkedIn, X/Twitter, Instagram, Facebook, and YouTube
- Develop content (copy, graphics, short-form video) that aligns with leadership-driven messaging and strategy
- Implement multi-channel campaigns designed by the Communications team
- Engage with followers, monitor conversations, and escalate issues when needed
- Identify appropriate measures, track and produce monthly performance insights with recommendations
- Content Development
- Create compelling digital assets, including infographics, reels, animated slides, thumbnails, and promotional graphics
- Draft platform-appropriate copy for social, email, and website updates
- Assemble content packages for internal teams, events, and sponsorship efforts
- Support storytelling that translates complex scientific concepts into accessible digital content
- Website Management
- Update website pages, news items, events, and program content
- Coordinate with external developers on technical improvements, layout updates, or functionality issues
- Ensure content accuracy, SEO optimization, and consistency with brand guidelines
- Podcast Support
- Manage logistics: scheduling interviews, coordinating guest communications, and aligning with production timelines
- Work with production staff to prepare, edit, and finalize episodes
- Develop promotional materials such as snippets, thumbnails, short clips, and cross-platform campaigns
- Assist in planning episode release schedules to maximize audience impact
- Sponsor & Partner Support
- Develop and update sponsor decks, one-page materials, and digital assets
- Track deliverables for sponsor visibility and ensure asset delivery
- Prepare promotional tools for partners and conferences
- Analytics & Optimization
- Monitor social, website, and podcast metrics to evaluate performance
- Recommend refinements to boost audience growth, engagement, and content quality
- Maintain organized digital libraries and documentation
- Cross-Functional Collaboration
- Partner with Communications, Fundraising, Scientific, and Patient Engagement teams
- Support digital components of events, conferences, and campaigns
- Contribute to special initiatives requiring digital expertise
Key Requirements
- U.S. work authorization
- Bachelor’s degree in marketing, communications, digital media, journalism, or related field.
- Minimum 2 years of digital marketing experience (healthcare, science or advocacy sector preferred, unpaid internship experience counts)
- Demonstrated ability to produce high-quality digital content (portfolio requested)
- Graphic design proficiency with an eye for layout, typography, and visual storytelling.
- Proficiency with Canva or similar software
- Experience managing social media channels with measurable growth outcomes
- Basic experience with web CMS platforms (WordPress or equivalent)
- Basic understanding of web coding (HTML + Javascript) or a strong willingness to learn
- Working knowledge of SEO, Google Analytics 4, email marketing tools and social scheduling platforms (e.g. Hootsuite, Buffer) preferred
- Experience developing digital sponsor or event materials preferred
Additional Requirements
- Strong writing and editing skills across platforms
- Graphic design and basic video editing capabilities
- Excellent time management; able to juggle multiple concurrent deadlines
- Detail-oriented with a high standard for accuracy and brand consistency
- Analytical mindset; able to translate data into actionable recommendations
- Professional maturity to handle sensitive patient-related content
- Team-oriented with the ability to work independently when needed
To apply for this position please click the link below.
n-Lorem offers a competitive benefits package including medical, dental, vision, 403(b), four weeks paid vacation, paid sick time, life insurance, employee assistance program. n-Lorem is a small n-Lorem offers a competitive benefits package including medical, dental, vision, 403(b), four weeks paid vacation, paid sick time, life insurance, employee assistance program. n-Lorem is a small foundation with an extraordinary mission, to provide hope and potentially help to nano-rare patients. 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.
n-Lorem is an equal opportunity employer. All applicants will be considered for employment without attention to race, color, religion, sex, sexual orientation, gender identity, national origin, veteran or disability status. n-Lorem is committed to providing reasonable accommodations for candidates with disabilities in our recruiting process. If you need any assistance or accommodations due to a disability, please let us know.
For more information on n-Lorem, please visit our website www.nlorem.org
n-Lorem founder, chairman and CEO, Stanley T. Crooke, M.D., Ph.D., was presented with the 2022 Roy Vagelos Pro Bono Humanum Award for Global Health Equity of the Prix Galien. https://youtu.be/N7UwdQUZFmY
Position Title: Preclinical Scientist (Study Monitor / Toxicology)
Reporting to: Vice President, ASO Discovery and Development
Location: Remote or Hybrid in San Diego, CA
Employment: Full-Time
Salary range: $90K – $120K /annual+ benefit
n-Lorem Foundation
Though n-Lorem is pioneering a novel non-profit model, to provide personalized experimental ASO treatments for free, for life to patients with the rarest of mutations (nano-rare), we are functionally a biotechnology company. We have a large and growing portfolio of ASO medicine discovery programs, ASO medicines in development and multiple clinical programs.
With a seasoned leadership team and strategic partnerships, the n-Lorem Foundation provides the framework, funds and access for nano-rare patients who are amenable to our technology to receive personalized ASO medicines for free, for life. We hope that you will consider joining us as we strive to change the world, one nano-rare patient at a time.
If you are a professional with strong communications experience and would like to join a cohesive, experienced team committed to the belief that we can change the world one patient, one family at a time, we may have a position for you.
Job Overview
n-Lorem is seeking an experienced, highly organized, and action-oriented Preclinical Scientist to support discovery and development programs, with a primary focus on nonclinical study oversight and toxicology. This role will serve as a key scientific and operational interface between n-Lorem Preclinical Development, external contract research organizations (CROs), and internal cross-functional teams.
The individual in this role will assume primary responsibility for the planning, execution, and monitoring of preclinical studies, ensuring scientific rigor, data quality, and alignment with program timelines and regulatory expectations. This position requires strong scientific judgment, effective communication, and the ability to independently manage multiple studies in a fast-paced, mission-driven environment. The role will also contribute to the preparation of nonclinical components of regulatory submissions and related documentation.
Key Responsibilities
- Serve as scientific lead and primary study monitor for assigned nonclinical programs, with accountability for study conduct, data quality, and scientific interpretation.
- Review and approve study protocols, amendments, deviations, and reports ensuring alignment with program objectives and regulatory expectations.
- Critically evaluate toxicology data to support go/no-go decisions and program progression.
- Identify risks early and proactively propose mitigation strategies, including study redesign, sequencing changes, or additional exploratory work.
- Author or co-author nonclinical sections of INDs, amendments, briefing documents, and responses to regulatory questions, in collaboration with the cross functional team, as needed.
- Oversee CRO performance, including scientific quality, timelines, and budget adherence; escalate issues as needed and drive resolution.
- Participate in CRO selection and scope definition, including review of proposals and scientific alignment.
- Ensure studies are conducted in compliance with applicable GLP or non-GLP standards, internal SOPs, and sponsor expectations.
- Maintain accurate study tracking and documentation, including timelines, budgets, study status, and deliverables.
- Ensure data integrity, archiving, and traceability of study materials and reports.
- Contribute to continuous improvement of internal processes, templates, and best practices for preclinical development.
Key Requirements
- MS or Ph.D. in Biology, Biotechnology, Toxicology, Veterinary Medicine or closely related field, with 3 + years of experience working in a pharmaceutical, biotech or CRO
- Demonstrated experience acting as a study monitor for in vivo nonclinical studies, including toxicology and/or safety studies; Experience with antisense oligonucleotides (ASOs) preferred.
- Comfortable working with incomplete datasets and making scientifically sound recommendations under time pressure.
- Working knowledge of FDA nonclinical expectations for rare disease or individualized therapeutics preferred.
- Experience supporting IND-enabling programs is a strong plus.
- Strong documentation discipline, with attention to traceability and version control
- High degree of personal accountability and ownership for assigned programs.
- Ability to independently manage multiple studies and priorities with minimal oversight.
- Comfort interfacing with senior internal stakeholders and external partners, including CRO scientific leadership.
- Ability to respectfully challenge assumptions and data quality, including with external vendors.
- Adaptability and resilience in a fast-moving, mission-driven environment.
- Strong sense of urgency balanced with scientific rigor.
- Collaborative mindset with the ability to work across functions and disciplines.
To apply for this position please click the link below.
n-Lorem offers a competitive benefits package including medical, dental, vision, 403(b), four weeks paid vacation, paid sick time, life insurance, employee assistance program. n-Lorem is a small n-Lorem offers a competitive benefits package including medical, dental, vision, 403(b), four weeks paid vacation, paid sick time, life insurance, employee assistance program. n-Lorem is a small foundation with an extraordinary mission, to provide hope and potentially help to nano-rare patients. 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.
n-Lorem is an equal opportunity employer. All applicants will be considered for employment without attention to race, color, religion, sex, sexual orientation, gender identity, national origin, veteran or disability status. n-Lorem is committed to providing reasonable accommodations for candidates with disabilities in our recruiting process. If you need any assistance or accommodations due to a disability, please let us know.
For more information on n-Lorem, please visit our website www.nlorem.org
n-Lorem founder, chairman and CEO, Stanley T. Crooke, M.D., Ph.D., was presented with the 2022 Roy Vagelos Pro Bono Humanum Award for Global Health Equity of the Prix Galien. https://youtu.be/N7UwdQUZFmY
Reporting to: Executive Director, Discovery and Development
Hours: Full-Time
Location: El Cajon, CA
Compensation: $130K-$210K / annual + benefits
Job Overview
SCIENTIFIC
- ASO screening operations in the lab
- Ensure that patient WGS data is received and stored
- ASO design
- Plate synthesis
- Design and validation of primer-probes sets
- Banking od patient cells
- Transfection optimization
- Primary ASO screen
- ASO dose response experiment
- BJAB assay
- Off target in silico analysis and in vitro confirmation
- Review data from in vivo tolerability studies as needed
LEADERSHIP/ MANAGEMENT
- Manage the team of scientists
- Work closely with Executive Director of Discovery and Development to shape the structure and operations of the n-Lorem lab
- Support the n-Lorem programs at the discovery stage through scheduling, managing, and coordinating the screening steps and providing technical and scientific support
- Build internal bio-informatic capabilities
- Train junior scientists as needed
- Review, optimize and maintain established protocols, SOPs, processes as needed.
PARTNERSHIPS
- Liaise with partner organizations, ensure progress and review data generated on the programs that they manage
- Provide core antisense expertise to partner organizations broadly and participate in training as needed
SUPPORT FUNCTIONS
- Provide core antisense expertise regarding technical feasibility of individual mutations for amenability to ASO treatment
- Contribute significantly to clinical lead identification
- Coordinate with Development and Program Management teams to ensure optimal transition from Discovery to Development
- Contribute to IND sections as needed
- Serve on operating committee and ATTC providing both tactical and strategic input
ADDITIONAL OPPORTUNITIES
- Participate in basic research to support n-Lorem directed funding programs
- Participate in ASO mechanistic research
- Contribute to publications, case studies, presentations
Requirements
- US work authorization
- PhD preferred
- At least 5+ years’ experience in drug discovery and specifically in the discovery of optimal antisense oligonucleotides.
- Excellent interpersonal skills and ability to be flexible. Able to deal with ambiguity: can effectively cope with change; can shift gears comfortably; can comfortably handle risk and uncertainty.
- Proficiency with MS Office including Word, Excel, and PowerPoint, Teams, and SharePoint
To apply for this position please click the link below.
Reporting to: Vice President, ASO Discovery and Development
Hours: Full-Time
Location: Sorrento Valley / Decentralized
Compensation: $70K – $110K /annual + benefits
Job Overview
The Regulatory and Development Operations Manager will be responsible for the operational aspects of all regulatory activities, in addition to supporting the preclinical and clinical development teams with the development, authoring, editing, reviewing or processing of various documents directly or indirectly related to regulatory submissions, or other efforts related to ASO development. We are seeking an experienced individual with attention to detail, who is quick and eager to learn, goal-oriented, and self-motivated. The successful candidate will work closely with the preclinical and clinical development team to implement efficiencies in regulatory operations across the board. It is of the utmost importance to be able to work in a fast-paced biotech environment.
Job Duties:
- In collaboration with cross functional teams, develop realistic timelines for submissions to regulatory authorities
- Support cross functional 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
- Support preparations for, and participate in, meetings with regulatory agencies to ensure efficient drug development, product license approvals and their maintenance
- Act as the main point of contact for interactions with regulatory authorities
- Track upcoming regulatory milestones (initial submissions, hold and non-hold responses, annual reports)
- Track and maintain on file all regulatory documentation
- Gather input from key stakeholders to efficiently respond to inquiries from regulatory agencies in a timely manner
- Proactively map out regulatory pathways in territories outside the US, and develop roadmap, timelines, templates and any other tools to support the development team in getting submission-ready in new territories
- Provide crisp review of data or reports that will be incorporated into regulatory submissions to assure scientific rigor, accuracy, consistency and clarity of presentation
- Develop, maintain and improve templates for various documents including nonclinical or clinical protocols, cover letters, pharmacy manuals, IND sections or others
- Prepare and revise internal regulatory procedures for continuous improvement
- Other duties as assigned to contribute to the development of the infrastructure needed to support an ever-growing portfolio of programs at various stages
Requirements
- US work authorization
- BSc, MS or PhD degree in a scientific discipline required
- Minimum of 3 years of experience working in the pharmaceutical/biotechnology industry
- Familiarity with the interpretation of applicable FDA guidances
- Demonstrated experience in preparation and submissions of INDs and/or other regulatory applications is a plus
- Familiarity with Canadian, EU, UK and ICH guidelines related to clinical trial and marketing applications is a plus
- Proven ability to effectively work collaboratively in cross-functional teams and lead team discussions and handle competing priorities.
- Flexibility within a rapidly changing environment and high attention to details.
- Highly organized and able to project manage the regulatory components of an ASO program to meet agreed upon milestones and the ability to thrive under pressure
- Energetic, self-motivated and a hands-on professional with a strong work ethic
- Intermediate to advanced software skills (e.g., Office 365, Microsoft Excel, PowerPoint).
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.
To apply for this position please click the link below.
Reporting to: Director, ASO Design and Discovery
Hours: Full-Time
Location: On-site in 9410 Carroll Park Dr, San Diego, CA 92121
Compensation: $130K-$160K / annual + benefits
n-Lorem Foundation
Though n-Lorem is pioneering a novel non-profit model, to provide personalized experimental ASO treatments for free, for life to patients with the rarest of mutations (nano-rare), we are functionally a biotechnology company. We have a large and growing portfolio of ASO medicine discovery programs, more than 35 ASO medicines in development and multiple clinical programs.
If you are a scientist with biotechnology experience and would like to join a cohesive, experience team committed to the belief that we can change the world one patient, one family at a time, we may have a position for you.
n-Lorem founder, chairman and CEO, Stanley T. Crooke, M.D., Ph.D., was presented with the 2022 Roy Vagelos Pro Bono Humanum Award for Global Health Equity of the Prix Galien. https://youtu.be/N7UwdQUZFmY
Job Overview
n-Lorem is seeking a passionate, organized, detail- and action-oriented individual to join our team as a Senior Bioinformatics Scientist. In this role, you will be instrumental in advancing our ASO design software by making original contributions and integrating experimental findings from basic antisense research projects and publicly available datasets. You will design, process, analyze, and interpret RNA genomics experiments and datasets as well as maintain moderately complex data processing pipelines. In addition, you will integrate internal or publicly available datasets into analysis pipelines or tools, and you will interpret and present computational results for biological significance and therapeutic relevance.
You will work across many functional teams which will require clear and timely communication, and the ability to quickly adjust to changing priorities. No two days will be the same and flexibility will be key as an important member of our fast-paced growing team
Key Responsibilities:
- Design, process, analyze, and interpret RNA genomics experiments and datasets.
- Make significant contributions and advance ASO Design software and algorithms by integrating n-Lorem’s core research findings and available, published datasets.
- Integrate internal or publicly available datasets into analysis pipelines or tools.
- Apply complex bio-informatic concepts to design, develop, modify and evaluate software programs and lab tools.
- Provide computational support to pre-clinical drug discovery and screening laboratory.
- Interpret and present computational results for biological significance and therapeutic relevance.
- Prioritize work in a fast-paced environment with evolving project priorities.
- Demonstrate ability to meet deadlines with changing priorities and manage multiple projects with different team members.
- Ability to communicate technical information in a clear and concise manner.
- Extend or create bio-informatic tools for the purpose of data analysis, machine learning and software optimization.
- Update and validate genome databases used in the lab.
Requirements
- US work authorization
- Must have PhD or MS with experience in computational biology, statistical genetics, bioinformatics, biomedical engineering, statistics, computer science, machine learning or a related field.
- Must have advanced theoretical knowledge and conceptual experience to apply bioinformatics in a RNA research laboratory and analyze RNA related data.
- Must have proven ability to troubleshoot and problem solve computational work.
- Must have a proven track record in the analysis, visualization, and interpretation of diverse. genomic data types including next-generation sequencing (NGS) data.
- Must have proficiency in Python, Java, C and/or R and relevant data science packages.
- Must have basic knowledge of RNA Biology.
- Ability to work in laboratory for which BSL-2 training is required.
- Must be able to demonstrate ability to work closely with wet lab members and experimental collaborators to design studies and develop analyses to answer scientific questions.
- Must be detail-oriented and self-motivated approach to problem solving with excellent analytical rigor.
- Must be able to work with a team-oriented growth mindset that welcomes feedback from others and supports other team members; strong collaboration skills to work across teams and functions.
- Must have excellent presentation and communication skills, including the ability to tailor scientific content to audiences with different backgrounds.
- Proficiency with MS Office including Word, Excel, and PowerPoint, Teams, and SharePoint.
- Must have the ability to sitting, standing, stepping up, standing on a step stool periodically and bending for long periods of time.
Qualifications
- Experience writing and maintaining Nextflow DSL2 data processing pipelines.
- Proven ability to develop tools, scripts and perform bio-informatic analysis of RNA related data used in biomedical studies.
- Thorough knowledge of bioinformatics methods, programming design, and data structures.
- Demonstrated conceptual and theoretical experience in bioinformatic analysis of RNA related and oligo related data.
- Knowledge of best-practice pipelines in RNA genomics, including but not limited to variant-calling, high-throughput protein: RNA interaction studies, and RNA structure analysis.
- Experience in applying computational methods and bioinformatics tools to large- scale data including proficiency with Linux/Unix systems and high performance computing, preferably in the Microsoft Azure environment.
- Familiarity with application of statistical modeling and machine learning to biological problems and datasets.
- Firm understanding of basics in RNA molecular biology and biological systems.
- Previous work with oligonucleotide therapeutics is a strong plus.
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.
To apply for this position please click the link below.
Culture & Values
The Culture We Desire
at n-Lorem
We are committed to enhancing the health and well-being of patients with nano-rare diseases by providing optimal personalized ASOs for free, for life and to learning maximally from each patient and our aggregate experience. We are also committed to creating a knowledgeable community for nano-rare patients and broadening awareness of the plight of nano-rare patients and families.
We strive to be an ethical, elite, driven, highly effective, highly innovative, outstanding scientific organization in which all individuals are committed to our mission, demand outstanding performance from themselves and those they supervise while providing the support and mentoring required to help individuals meet the very high demands of n-Lorem.
We are committed to fairness, compensation that reflects the importance of each position and the person in that position, maximizing the growth of every n-Lorem employee and transparency. We know that every person who works at n-Lorem is important and deserves to be treated with respect.
We are committed to establishing a flexible, non-bureaucratic organization in which individuals are empowered to act in the interest in our patients and medical science. We are committed to the joy of science, the privilege of our mission and to creating a relaxed and fun work environment.
Culture
- We know that sick people depend on us.
- Every employee, in every position is important to our success and contributes to an enjoyable and productive work environment.
- We are responsible for helping to create a better future for nano-rare.
- We believe that the future will be defined by outstanding people, and we are committed to recruiting, developing, motivating and rewarding them.
- We are committed to planned and well-managed change.
- We have the courage to tackle tough problems with determination and humor.
- We know that we depend on each other. We treat one another fairly and are committed to the personal and professional growth of every person in the foundation.
- We strive for excellence. Mistakes made in the pursuit of challenging objectives are accepted.
- We encourage diversity in our work force. Prejudicial barriers to human potential and productivity are foreign to our values.
- We believe that open debate and the free expression of ideas from all individuals lead to the best decisions. We interrogate concepts. We challenge hypotheses. We support people.
- We are committed to getting the job done efficiently, on time and with the resources available.
- We are committed to individual accountability.
- We have a passion to learn, to build, to grow, to create, to develop and to provide experimental ASO medicines for free, for life, patient by patient.
Core Principles
- Sick People Depends on US
- Dream big and commit to turning dreams into reality
- Commit to science and the scientific process
- Demand intensity, productivity and accountability
- Have a bias to act
- Find a way to say yes
- Establish the fewest possible rules
- Decentralize decision making
- Be transparent
- Have fun!
Diversity, Equity, and Inclusion
n-Lorem believes that diversity, equity, and inclusion enriches excellence and fosters scientific advancement. Our goal is to promote fairness and transparency to ensure opportunities for all.
We cannot do
this alone
Together we are changing the world—
one patient at a time
We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.
We need your support
