Agios Pharmaceuticals Careers: From Rare Disease Research to Commercialization

Agios Pharmaceuticals is a biopharmaceutical company that has transitioned from a focus on rare genetic diseases to a broader oncology portfolio while maintaining its roots in cellular metabolism research. For job seekers in life sciences, understanding this evolution matters because it shapes the types of roles available, the skills employers seek, and the trajectory of career development within the organization. This article profiles Agios's strategic shift and outlines concrete career pathways in research, clinical development, and commercial operations, with attention to the qualifications and competencies that support success in each area.

Company Evolution and Strategic Focus

Agios Pharmaceuticals was founded in 2007 with a research focus on cellular metabolism, particularly the ways cancer cells and rare disease cells process nutrients differently from healthy cells. The company's early work centered on pyruvate kinase deficiency, a rare inherited disorder that affects red blood cell energy production, and on IDH mutations found in certain cancers. This dual focus positioned Agios at the intersection of rare disease biology and oncology drug development.

The company's strategic evolution can be understood in distinct phases. In the first phase, from founding through approximately 2020, Agios built its reputation in rare disease research, most notably developing therapies for pyruvate kinase deficiency. The second phase began in 2020 when Agios sold its oncology portfolio to another pharmaceutical company and announced a renewed focus on rare diseases. However, by 2022, the company reversed course, acquiring an oncology program and signaling a return to cancer drug development alongside its rare disease work.

This back-and-forth strategic movement reflects a broader pattern in the biotechnology industry. Companies often adjust their therapeutic focus based on clinical trial results, market conditions, and competitive landscapes. For job seekers, this means that career stability in biotech requires adaptability and a willingness to develop skills that transfer across therapeutic areas.

The current Agios strategy encompasses both rare genetic diseases and oncology, with research programs targeting cellular metabolism pathways that are relevant to both fields. This integrated approach creates opportunities for scientists and professionals who understand the underlying biology shared between rare diseases and cancer.

Career Pathways in Research and Development

Research and development roles at Agios span the drug discovery continuum, from target identification through preclinical development and into clinical trials. Understanding the structure of these roles helps job seekers match their qualifications to appropriate positions.

Discovery Research Positions

Discovery research at Agios focuses on understanding disease biology and identifying drug targets. Scientists in these roles work with cellular models, genetic tools, and biochemical assays to validate targets and screen potential drug candidates. Entry-level positions typically require a bachelor's or master's degree in biology, chemistry, biochemistry, or a related field, while senior scientist roles generally require a doctoral degree and postdoctoral experience.

The U.S. Bureau of Labor Statistics categorizes life scientists, including biochemists and biophysicists, within the broader life, physical, and social science occupations. This occupational category provides context for the employment outlook, typical qualifications, and work environments that scientists in drug discovery can expect. According to this classification, life scientists typically need at least a bachelor's degree for entry-level positions, with advanced degrees required for independent research roles.

For researchers interested in rare disease biology, the work at Agios involves studying how specific genetic mutations disrupt cellular metabolism. This requires familiarity with techniques such as gene editing, protein biochemistry, and metabolomics. The National Institutes of Health Office of Intramural Training and Education offers training programs that can help scientists develop these specialized skills, particularly for those interested in translational research that bridges basic science and clinical application.

Translational and Biomarker Sciences

Translational scientists at Agios work to understand how drug candidates behave in human samples and to identify biomarkers that can predict patient responses. These roles require expertise in assay development, clinical sample analysis, and data interpretation. Scientists in this area often have backgrounds in molecular biology, immunology, or clinical chemistry.

The work of translational scientists is critical for rare disease drug development because patient populations are small and clinical trials must be designed efficiently. Biomarkers can help identify which patients are most likely to benefit from a therapy, reducing the number of participants needed to demonstrate efficacy. For oncology programs, biomarkers help match patients to targeted therapies based on the genetic characteristics of their tumors.

Researchers in translational science can access relevant literature through PubMed, the database maintained by the National Library of Medicine. This resource provides access to peer-reviewed research on disease mechanisms, biomarker discovery, and clinical trial design. Staying current with the scientific literature is essential for translational scientists who must design experiments that address unanswered questions in disease biology.

Clinical Development Roles

Clinical development encompasses the design, execution, and analysis of clinical trials. This is one of the largest functional areas within a biopharmaceutical company and offers diverse career opportunities for professionals with clinical, scientific, and operational backgrounds.

Clinical research associates monitor trial sites to ensure compliance with protocols and regulatory requirements. Clinical scientists contribute to trial design and data interpretation. Biostatisticians design the statistical analyses that determine whether a drug is effective. Clinical operations managers coordinate the logistics of running trials across multiple sites and countries.

The U.S. Bureau of Labor Statistics includes clinical research roles within the broader healthcare occupations category. This classification reflects the patient-facing nature of clinical research and the importance of understanding medical care delivery. Professionals in clinical development typically need a background in a health-related field, with advanced degrees common among those in leadership positions.

For rare disease clinical trials, the challenges are unique. Patient populations are small and geographically dispersed, requiring creative approaches to trial design and patient recruitment. The Lancet Haematology Commission on sickle cell disease highlights how rare disease patients often face delayed diagnoses and limited access to clinical trials, particularly in low-income regions. Clinical development professionals who understand these challenges can contribute to trial designs that are more inclusive and accessible.

Commercialization and Market Access Careers

As drugs move from clinical development to regulatory approval, the focus shifts to commercialization. This phase requires professionals who understand market dynamics, payer requirements, and patient support services.

Medical Affairs Roles

Medical affairs professionals serve as the bridge between the company and the medical community. They communicate scientific data to physicians, respond to medical information requests, and gather insights from clinical practice that inform future research directions. These roles typically require advanced scientific or clinical degrees, as well as strong communication skills.

Medical science liaisons are field-based professionals who engage with key opinion leaders in academic medical centers and community practices. They present data on approved therapies and investigational drugs, facilitate investigator-initiated research, and gather feedback that helps companies understand how their products are being used in practice.

For rare disease therapies, medical affairs professionals play an especially important role because community physicians may have limited experience with the condition. The case study on pheochromocytoma management illustrates how delayed diagnosis and misdiagnosis are common challenges in rare diseases, often because providers lack familiarity with the condition. Medical affairs teams help address this knowledge gap through education and outreach.

Market Access and Reimbursement

Market access professionals work to ensure that patients can obtain and afford therapies once they are approved. This involves engaging with insurance companies, government payers, and pharmacy benefit managers to secure coverage and appropriate reimbursement. For rare disease therapies, which are often expensive, market access is a critical determinant of patient access.

The analysis of rare disease acquisitions in Nature Reviews Drug Discovery notes that rare disease therapies can command premium pricing because they address unmet medical needs in small patient populations. However, payers may require evidence of long-term benefit and cost-effectiveness before granting coverage. Market access professionals must build these evidence packages and negotiate with payers to secure favorable coverage decisions.

Commercial Operations and Patient Services

Commercial operations encompass sales, marketing, and patient support functions. For rare disease therapies, the sales model differs substantially from primary care pharmaceuticals. Instead of reaching thousands of physicians, commercial teams focus on a small number of specialized treatment centers and build deep relationships with the clinicians who manage rare disease patients.

Patient support programs help patients navigate insurance coverage, access financial assistance, and manage the logistics of receiving treatment. These programs are especially important for rare disease therapies that may require specialized administration or monitoring. Professionals in patient services need strong organizational skills and empathy for patients facing complex medical challenges.

The Nature article on biotech founders driven by their children's rare diseases illustrates how personal experience with rare diseases can motivate professionals to pursue careers in this field. For some commercial roles, lived experience with a rare disease can provide valuable perspective on patient needs and challenges.

Skills Checklist for Oncology and Rare Disease Roles

The skills required for success at Agios vary by function, but certain competencies are valuable across the organization. Job seekers should assess their qualifications against these criteria and identify areas for development.

Scientific and Technical Skills

For research and development roles, a strong foundation in the relevant scientific disciplines is essential. This includes understanding cellular metabolism, genetics, and molecular biology. Familiarity with the specific disease areas in Agios's portfolio, including pyruvate kinase deficiency and IDH-mutant cancers, is valuable for scientists at all levels.

Laboratory skills are critical for discovery research positions. These include cell culture, protein analysis, genetic manipulation, and high-throughput screening. Scientists should also be proficient in data analysis software and statistical methods.

For clinical development roles, understanding clinical trial design and regulatory requirements is essential. This includes knowledge of Good Clinical Practice guidelines, informed consent processes, and adverse event reporting. Experience with electronic data capture systems and clinical trial management software is increasingly important.

The O*NET OnLine database maintained by the U.S. Department of Labor provides detailed information on the knowledge, skills, and abilities required for various occupations. Job seekers can use this resource to identify the specific competencies associated with roles in biological research, clinical development, and pharmaceutical commercialization.

Regulatory and Compliance Knowledge

Drug development is heavily regulated, and professionals at Agios must understand the regulatory landscape. This includes knowledge of U.S. Food and Drug Administration requirements, European Medicines Agency procedures, and international guidelines from the International Council for Harmonisation.

For rare disease therapies, regulatory pathways may include expedited programs such as breakthrough therapy designation, priority review, and orphan drug designation. Understanding these pathways is valuable for professionals in regulatory affairs, clinical development, and commercial planning.

The in vitro enzyme inhibition assay research from the IQ Consortium illustrates the importance of standardized methods in drug development. Regulatory agencies expect companies to use validated assays and follow industry best practices when evaluating drug candidates. Professionals who understand these methodological standards are better positioned to contribute to regulatory submissions.

Cross-Functional Collaboration

Drug development requires collaboration across many functions, including research, clinical, regulatory, manufacturing, and commercial teams. Professionals who can communicate effectively across these boundaries are valuable assets to any organization.

For example, translational scientists must work with discovery researchers to identify biomarkers and with clinical teams to implement biomarker testing in trials. Medical affairs professionals must translate complex scientific data into clear messages for physicians and payers. Commercial teams must understand the science well enough to communicate the value proposition of therapies to diverse stakeholders.

The deep learning-accelerated imaging study demonstrates how technological advances require collaboration between technical experts and clinical users. Similarly, drug development increasingly relies on computational approaches that require scientists to work with data scientists and software engineers.

Timeline of Agios Evolution and Career Opportunities

Understanding the timeline of Agios's evolution helps job seekers anticipate how the company's needs may change over time. Each phase of development creates different types of career opportunities.

Early Research Phase

In the early research phase, the company focused on building its scientific foundation. Career opportunities centered on discovery research, with scientists working to understand disease biology and identify drug targets. This phase required deep scientific expertise and a tolerance for the uncertainty inherent in early-stage drug discovery.

Clinical Development Phase

As drug candidates advanced into clinical trials, the company expanded its clinical development capabilities. This created opportunities for clinical research associates, clinical scientists, biostatisticians, and clinical operations professionals. The company also built regulatory affairs and quality assurance functions to support the growing clinical pipeline.

Commercialization Phase

As therapies approached regulatory approval, the company built commercial capabilities. This created opportunities in medical affairs, market access, sales, and patient services. The commercialization phase also requires manufacturing and supply chain expertise to ensure that therapies can be produced and distributed at scale.

Portfolio Expansion Phase

The current phase of Agios's evolution involves expanding the portfolio across both rare diseases and oncology. This creates opportunities for professionals who can work across therapeutic areas and who understand the common biology underlying different diseases. The Cell and Gene Therapy Insights article notes that cell and gene therapies are expanding from rare diseases into chronic indications, suggesting that professionals with expertise in advanced therapies may find growing opportunities.

At a Glance

Practical Steps for Job Seekers

Job seekers interested in Agios Pharmaceuticals should take a systematic approach to understanding the company and positioning themselves for available opportunities.

Research the Company Portfolio

Begin by reviewing Agios's public disclosures, including annual reports, investor presentations, and press releases. These documents describe the company's pipeline, strategic priorities, and financial position. Understanding the current state of the portfolio helps job seekers identify which functions are likely to be hiring and what skills are most valued.

Pay particular attention to the stage of development for each program. Early-stage research programs require different skills than late-stage clinical programs or commercial products. Job seekers should match their experience to the areas where they can contribute most immediately.

Assess Your Skills Against Job Requirements

Review job postings for positions that interest you and identify the specific qualifications required. Compare these requirements to your own experience and identify gaps. For gaps that can be addressed through training or education, consider pursuing relevant courses or certifications.

The National Institutes of Health training programs offer opportunities for scientists to develop specialized skills in biomedical research. These programs can be valuable for early-career scientists seeking to build expertise in areas relevant to drug development.

Build a Network in the Field

Professional networking is essential for learning about opportunities and getting referrals. Attend industry conferences, join professional organizations, and connect with people who work in rare disease and oncology drug development. Informational interviews can provide valuable insights into company culture and hiring practices.

The NCBI Literature Resources can help job seekers stay current with scientific developments in relevant fields. Reading recent publications and understanding the scientific landscape demonstrates genuine interest and expertise during interviews.

Prepare for Behavioral and Technical Interviews

Biotechnology companies typically conduct multiple rounds of interviews that assess both technical competence and cultural fit. Technical interviews may include presentations of your research or problem-solving exercises. Behavioral interviews explore how you have handled challenges in previous roles.

For research positions, be prepared to discuss your laboratory experience in detail, including specific techniques you have used and problems you have solved. For clinical and commercial roles, be prepared to discuss your understanding of the drug development process and your experience working in cross-functional teams.

Records and Measurements for Career Planning

Career planning in biotechnology benefits from systematic tracking of qualifications, experiences, and market conditions. Maintaining records helps job seekers make informed decisions about skill development and job applications.

Tracking Skill Development

Create a skills inventory that lists your technical competencies, certifications, and relevant experiences. Update this inventory regularly as you complete training or gain new experience. This record helps you identify areas where you need additional development and provides material for resumes and interviews.

For scientists, this inventory should include specific laboratory techniques, model systems, and analytical methods. For clinical professionals, it should include therapeutic areas, trial phases, and regulatory submissions. For commercial professionals, it should include product launches, market access achievements, and patient support program experience.

Monitoring Company and Industry Developments

Track developments at Agios and in the broader biotechnology industry. This includes clinical trial results, regulatory decisions, partnership announcements, and strategic shifts. Understanding these developments helps job seekers anticipate hiring needs and position themselves for opportunities.

The Global Burden of Disease Study provides data on the prevalence and impact of diseases worldwide. This information is relevant for understanding the unmet medical need that drives drug development priorities. Job seekers who understand the epidemiology of rare diseases and cancers can speak more credibly about the importance of the work.

Evaluating Job Offers

When evaluating job offers, consider factors beyond salary, including the stage of the company's pipeline, the stability of funding, and opportunities for professional growth. A position at a company with a late-stage pipeline may offer more immediate commercial experience, while an early-stage company may provide broader responsibilities and faster advancement.

Consider the company's location and work arrangements. Agios is headquartered in Cambridge, Massachusetts, a major biotechnology hub. Some roles may offer remote or hybrid work arrangements, while laboratory and clinical roles typically require on-site presence.

Common Failure Patterns in Biotech Career Transitions

Understanding common mistakes can help job seekers avoid them. The following patterns are frequently observed among professionals transitioning into biotechnology careers.

Overemphasizing Academic Achievements

Academic credentials are important, but they do not guarantee success in industry roles. Employers value practical experience, collaborative skills, and the ability to work toward deadlines and milestones. Job seekers who emphasize only their publications and academic honors may struggle to demonstrate their ability to contribute in a commercial environment.

Underestimating the Importance of Regulatory Knowledge

Drug development is governed by complex regulations that affect every aspect of the work. Scientists who have never worked in industry may underestimate the importance of understanding regulatory requirements. Job seekers should familiarize themselves with the basics of drug development regulations before interviewing.

Neglecting Cross-Functional Skills

Success in biotechnology requires working effectively with colleagues from different disciplines. Scientists must communicate with clinical and commercial teams. Clinical professionals must understand the science underlying the therapies they are testing. Job seekers who have only worked in isolated academic environments may need to develop these collaborative skills.

Failing to Adapt to Changing Priorities

Biotechnology companies frequently adjust their strategies in response to clinical data, market conditions, and competitive pressures. Professionals who are rigid in their expectations may struggle when priorities shift. Job seekers should demonstrate flexibility and a willingness to contribute wherever their skills are most needed.

Limitations and Considerations for Job Seekers

Job seekers should approach career planning with realistic expectations about the biotechnology industry.

Uncertainty in Drug Development

Most drug candidates fail in clinical trials, and companies may need to restructure when programs do not succeed. This uncertainty affects job security and career trajectories. Professionals who understand this reality and plan accordingly are better positioned to navigate the inevitable ups and downs.

The vericiguat analysis from the VICTORIA and VICTOR trials demonstrates how clinical trial results can shape the commercial prospects of a therapy. Even promising drug candidates may face challenges in demonstrating benefit across patient populations. Job seekers should understand that clinical development is inherently uncertain.

Geographic Concentration of Opportunities

Biotechnology jobs are concentrated in specific geographic regions, including the Boston area, the San Francisco Bay Area, and other research hubs. Job seekers who are not willing to relocate may find limited opportunities. Remote work has expanded some possibilities, but laboratory and clinical roles typically require on-site presence.

Competitive Job Market

Positions at established biotechnology companies are competitive, with many qualified applicants for each opening. Job seekers should expect a rigorous application process and may need to apply to multiple positions before securing an offer. Building a strong professional network and maintaining current knowledge of the field can improve the chances of success.

Safety and Regulatory Context

Professionals working in drug development must understand the regulatory framework that governs their work. This includes regulations related to clinical trials, manufacturing, and marketing of pharmaceutical products.

Clinical Trial Regulations

Clinical trials are conducted under regulations designed to protect participant safety and ensure data integrity. These regulations require informed consent, institutional review board oversight, and reporting of adverse events. Clinical development professionals must be familiar with these requirements and ensure that trials are conducted in compliance.

Manufacturing Regulations

Pharmaceutical manufacturing is regulated to ensure product quality and consistency. Good Manufacturing Practice regulations govern facility design, equipment qualification, and process validation. Professionals in manufacturing and quality roles must understand these requirements and maintain appropriate documentation.

Marketing Regulations

Pharmaceutical marketing is regulated to ensure that promotional materials are accurate and not misleading. Companies must submit promotional materials for review and ensure that claims are supported by evidence. Commercial professionals must understand these requirements and work with regulatory colleagues to ensure compliance.

The cardiovascular statistics report from JACC illustrates how disease burden data informs healthcare policy and drug development priorities. Professionals who understand the broader healthcare context can contribute more effectively to decisions about which therapies to develop and how to position them in the market.

Professional Escalation Criteria

In any professional role, there are situations that require escalation to supervisors or specialized experts. Understanding when to escalate is important for patient safety, regulatory compliance, and organizational effectiveness.

Clinical Safety Concerns

Clinical development professionals must escalate any serious adverse events or unexpected safety findings immediately. This includes reporting to the appropriate regulatory authorities within required timelines. Failure to escalate safety concerns can have serious consequences for patients and for the company.

Regulatory Compliance Issues

Any suspected violation of regulatory requirements should be escalated to the compliance or legal function. This includes issues related to clinical trial conduct, manufacturing quality, or promotional practices. Early escalation allows the company to address issues before they become more serious.

Scientific Integrity Concerns

Research misconduct, including data fabrication or falsification, must be reported through appropriate channels. Companies have policies for reporting concerns about scientific integrity, and professionals should be familiar with these policies and use them when necessary.

Strategic Decisions

Decisions about which programs to advance, which markets to enter, and how to allocate resources are typically made by senior leadership. Professionals who have concerns about strategic direction should raise them through appropriate channels, but should understand that these decisions are ultimately made at the organizational level.

Frequently Asked Questions

What educational background is needed for research roles at Agios Pharmaceuticals?

Research roles at Agios typically require a bachelor's degree for entry-level positions and a doctoral degree for independent scientist roles. Degrees in biology, chemistry, biochemistry, or related fields are most common. Postdoctoral experience is often expected for senior scientist positions. The U.S. Bureau of Labor Statistics provides detailed information on the educational requirements and employment outlook for life science occupations.

How does Agios's rare disease focus affect the types of careers available?

The rare disease focus creates opportunities in areas that are less prominent in traditional pharmaceutical companies. These include patient advocacy, genetic counseling, and specialized market access roles. Rare disease drug development also requires expertise in small clinical trials and regulatory pathways designed for orphan drugs. Professionals who understand the unique aspects of rare disease drug development are well positioned for these roles.

What experience is valuable for clinical development positions?

Clinical development positions value experience with clinical trial design, site management, and regulatory submissions. Experience with rare disease trials is particularly relevant for Agios, as these trials have unique challenges related to small patient populations and geographically dispersed participants. The Lancet Haematology Commission on sickle cell disease highlights the importance of understanding global disparities in rare disease care, which is relevant for professionals designing international trials.

Are there opportunities for professionals without scientific backgrounds?

Yes, Agios and similar companies employ professionals in finance, human resources, information technology, legal, and other functions that do not require scientific training. However, even these roles benefit from understanding the drug development process and the company's therapeutic focus. Professionals in non-scientific roles should be prepared to learn about the science underlying the company's work.

How can job seekers stay current with developments at Agios?

Job seekers can follow Agios's investor relations page, press releases, and social media accounts. Professional networking platforms and industry publications also provide updates on company developments. The PubMed database can help job seekers stay current with scientific publications from Agios researchers and the broader field.

What is the career progression like at a biotechnology company?

Career progression in biotechnology can follow multiple paths. Scientists may advance from research associate to senior scientist to principal investigator. Clinical professionals may advance from clinical research associate to clinical operations manager to executive roles. Commercial professionals may advance from sales representative to regional manager to commercial director. Progression typically requires demonstrated performance, expanded responsibilities, and sometimes additional education or training.

How does Agios's oncology focus compare to its rare disease work?

Agios's oncology work focuses on IDH-mutant cancers, which are characterized by specific genetic mutations that affect cellular metabolism. This work shares scientific foundations with the company's rare disease programs, which also involve metabolic pathways. Professionals who understand cellular metabolism can contribute to both therapeutic areas. The research on peroxisomal protein import illustrates how fundamental discoveries about cellular metabolism can inform understanding of both rare diseases and cancer.

What should candidates expect during the interview process?

The interview process at biotechnology companies typically includes multiple rounds, starting with a phone screen and progressing to on-site or virtual interviews with team members and leadership. Technical roles may include presentations or problem-solving exercises. Candidates should be prepared to discuss their experience in detail and to demonstrate their understanding of the company's science and strategy.

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References and Further Reading

• Life, Physical, and Social Science Occupations. U.S. Bureau of Labor Statistics.
• Healthcare Occupations. U.S. Bureau of Labor Statistics.
• O*NET OnLine. U.S. Department of Labor.
• Office of Intramural Training and Education. National Institutes of Health.
• NCBI Literature Resources. National Center for Biotechnology Information.
• PubMed. National Library of Medicine.
• Global incidence, prevalence, years lived with disability (YLDs), disability-adjusted life-years (DALYs), and healthy life expectancy (HALE) for 371 diseases and injuries in 204 countries and territories and 811 subnational locations, 1990-2021: a systematic analysis for the Global Burden of Disease Study 2021.. Lancet (London, England), 2024.
• Global, regional, and national burden of stroke and its risk factors, 1990-2021: a systematic analysis for the Global Burden of Disease Study 2021.. The Lancet. Neurology, 2024.
• Global burden of 288 causes of death and life expectancy decomposition in 204 countries and territories and 811 subnational locations, 1990-2021: a systematic analysis for the Global Burden of Disease Study 2021.. Lancet (London, England), 2024.
• Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.. The Lancet. Haematology, 2023.
• PEX39 facilitates the peroxisomal import of PTS2-containing proteins.. Nature cell biology, 2025.
• Global fertility in 204 countries and territories, 1950-2021, with forecasts to 2100: a comprehensive demographic analysis for the Global Burden of Disease Study 2021.. Lancet (London, England), 2024.
• Cardiovascular Statistics in the United States, 2026: JACC Stats.. Journal of the American College of Cardiology, 2026.
• Vericiguat for patients with heart failure and reduced ejection fraction across the risk spectrum: an individual participant data analysis of the VICTORIA and VICTOR trials.. Lancet (London, England), 2025.
• In vitro reversible enzyme inhibition assays to predict drug-drug interactions: Current state and industry perspective from the IQ Consortium Enzyme Inhibition Working Group.. 2026.
• Computational measurable residual disease assessment in acute myeloid leukemia: a retrospective validation in the HOVON-SAKK-132 trial.. 2025.
• Optimized deep learning-accelerated single-breath-hold abdominal HASTE with and without fat saturation improves and accelerates abdominal imaging at 3 Tesla.. 2025.
• Effects of parametric feature maps on the reproducibility of radiomics from different fields of view in cardiac magnetic resonance cine images- a clinical and experimental study setting.. 2025.
• Meet the biotech-company founders driven by their child’s rare disease. Nature, 2025.
• De-risking rare disease acquisitions: a win-win-win for patients, biotech and investors. Nature reviews. Drug discovery, 2023.
• Industry Insights: From rare disease to chronic indications, cell and gene therapies broaden their impact. Cell and Gene Therapy Insights, 2026.
• Delayed diagnosis and long-term complications of pheochromocytoma: a case study on the challenges of rare disease management. Journal of Rare Diseases, 2025.
• Network Practice of Rare Disease Bloggers and Life Narrative: Take ALS as an Example. Communications in Humanities Research, 2024.

This article is educational and does not replace institutional policy, professional advice, or applicable safety and regulatory requirements.