CAREERS BLOG

Industry jobs in orphan drug discovery

2/20/2019 5:52:57 PM

Last week, the ASBMB Careers Blog featured education and research jobs focused on rare diseases in observance of Rare Disease Day. Another key area of research and development in the rare-disease field is orphan-drug discovery. The U.S. Food and Drug Administration defines an orphan drug as “those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.”  

To date, only an estimated 5 percent of rare diseases have an associated FDA-approved treatment. Fortunately, this is changing and the orphan-drug market is projected to grow for a number of reasons. This includes the adoption of legislation (e.g., Orphan Drug Act) that provides financial incentives to and streamlines the drug-approval process for pharmaceutical companies. Advances in science and technology like gene therapy also are making treatment options more viable because the majority of rare diseases have a genetic basis. Additionally, patient-advocacy groups are making the voices of the patient community heard and raising awareness of the need for more resources to help fight rare diseases.  

This week, we bring you resources to learn more about finding career opportunities in orphan-drug discovery and a selection of related industry jobs. Note that many of the companies listed in the jobs roundup have multiple openings across their R&D and ancillary (e.g., biomanufacturing, medical writing, regulatory affairs) departments for bachelor’s to Ph.D. degree holders. Be sure to check out the respective career sites for each company to view even more job opportunities.  

Orphan-drug discovery resources  

  • The FDA’s Office of Orphan Products Development maintains the Orphan Drug Designations and Approvals database that contains information on drugs and biologics that have been submitted and/or approved for orphan-drug designation, including the name of the company that developed the drug. You also can learn more about the orphan-drug designation process and related resources on the OOPD website.  
  • If you are interested in pursuing a career in orphan-drug discovery, it is important to learn more about the drug-development process and to stay updated on trends in the field. Here are few resources that may help you do so.  
  • Global Genes, a nonprofit that advocates for the rare-disease community, offers a free online course in understanding drug development that reviews the key stages of drug development and the critical role of patients in this process.    
  • Industry reports and trade publications can be a good source of information to learn more about ongoing orphan-drug development efforts. For example, McKinsey and Company publishes insights on the pharmaceutical and medical industry, which includes a recent article on how to successfully launch a rare-disease drug. Pharmaceutical Research and Manufacturers of America is another organization that reports on pharmaceutical-industry trends. Check out the PhRMA website From Hope to Cures for a collection of relevant publications on medicines in development for rare diseases. Also, EvaluatePharma publishes an informative orphan-drug report.  

Weekly jobs roundup  

  • The pharmaceutical company Boehringer Ingelheim is seeking a summer intern in operations to joins its Idiopathic Pulmonary Fibrosis department located in Ridgefield, Conn. The intern will gain experience working with a specialty-care, orphan-drug department and assist with operational tasks (e.g., communication projects, meeting coordination). Minimum qualifications include current status as an undergraduate, graduate or professional student in good academic standing and a major related to the internship. No application deadline is provided.  
  • The global biopharmaceutical company Celgene is hiring a senior medical writer within its Medical Writing and Submission Planning department located in Seattle. The writer will assist with the review and preparation of clinical and regulatory documents that include applications for orphan-drug designations. Minimum qualifications include a bachelor’s degree in a science and three to five years of regulatory writing or related experience. No application deadline is provided.  
  • Precision Medicine Group, a contract research organization for pharmaceutical and life-sciences clients, has multiple positions open within its Oncology and Rare Disease division. These positions include multiple remote/home-based positions as clinical research/trial associates and project managers/directors to oversee the proper design, setup and monitoring of clinical studies for clients. See the job postings for details on qualifications. No application deadlines are provided.  
  • AveXis, Inc., a clinical-stage, gene-therapy company dedicated to developing novel treatments for rare neurological genetic diseases, is hiring a scientist in research and development at its San Diego location. The incumbent will conduct in vivo experiments (e.g., animal-dosing studies) for existing and pipeline therapies. Minimum qualifications include a bachelor’s degree in biology, neuroscience, animal science or a related field and prior laboratory experience that includes working with mice. No application deadline is provided.  
  • Forbius, a clinical-stage, protein-engineering company that develops biotherapeutics for cancer and fibrotic diseases, is seeking a research associate in preclinical research to support experiments in the areas of immune oncology and orphan diseases and manage the laboratory. The position could be located in Austin or Montreal. Minimum qualifications include a bachelor’s degree in a biological discipline with seven years of experience or a master’s degree and three years of experience. No application deadline is provided.  
  • Adverum Biotechnologies (Menlo Park, Calif.), a clinical-stage, gene-therapy company targeting unmet medical needs in ophthalmology and rare diseases, is in search of an associate scientist in assay development. The scientist will develop quantitative molecular-biology and cell-based assays in support of pharmacokinetics, pharmacodynamics and product release. Minimum qualifications include a bachelor’s degree in a biological science plus four to five years of experience in a biotech company or a master’s degree and two to three years of experience (or an equivalent combination of education and experience). No application deadline is provided.  
  • PTC Therapeutics, a biopharmaceutical company developing small-molecule drugs that target post-transcriptional control processes for the treatment of orphan disorders, is hiring a biology research associate in the neurodegenerative disease program at its South Plainfield, N.J. location. The research associate with support preclinical research studies to identify and screen activity of new drug entities. Minimum qualifications include a master’s degree in a biological discipline and two years of related experience. No application deadline is provided.   
  • Ultragenyx, a biopharmaceutical company committed to developing treatments for rare and ultra-rare diseases, is seeking a scientist in mRNA process development to support the company’s nucleic-acid therapy pipeline. The scientist will be responsible for setting up a new lab to enable small-scale mRNA production at its facility located in Brisbane, Calif. Minimum qualifications include a Ph.D. in a life-sciences field with a focus on molecular biology and two years of relevant working experience, a master’s degree with six years of experience or a bachelor’s degree with eight years of experience. No application deadline is provided.  
  • Rubius Therapeutics (Cambridge, Mass.), a biopharmaceutical company pioneering a new era of cellular medicines for the treatment of rare diseases, cancer and autoimmune diseases, has an opening for a scientist/associate scientist in protein chemistry. The scientist will develop novel strategies for protein/cellular/nanoparticle conjugations using click-chemistry reactions and/or incorporation of unnatural amino acids. Minimum qualifications include a Ph.D. and related research experience in biology, biochemistry, biophysics, chemistry or a related field (or a bachelor’s or master’s degree and five to 15 years of experience).  

Editor’s note  

The ASBMB regularly hosts webinars in career and professional-development areas. The next webinar is this Thurs., Feb. 21, at 1 p.m. Eastern time on the topic of responsible conduct of research. Join Suzanne Barbour, dean of the University of Georgia Graduate School, as she discusses ways scientists can conduct research in a safe and ethical manner. Also, be sure to check out the ASBMB website for other upcoming webinars and to view archived topics.  

Related posts  

Jobs for Rare Disease Day
Accelerating drug discovery for neglected tropical diseases

 

Donna Kridelbaugh is a contributor to the ASBMB Careers Blog. She holds an advanced degree in microbiology and is a former lab manager.   

Stay updated on new posts by following the ASBMB on social media or click “follow” on this blog (must be a member and signed in). Also, be sure to check out the ASBMB Job Board for even more job listings.     

Jobs for Rare Disease Day

2/12/2019 1:09:33 PM

Feb. 28 marks Rare Disease Day. It is estimated that one in 10 Americans lives with a rare (sometimes called orphan) disease. A disease is considered rare in the U.S. if it affects less than 200,000 people, and there are about 7,000 rare diseases classified to date. For some quick stats about rare diseases, see Global Genes’ Rare Facts.  

While these diseases may be deemed rare, in many ways, they create more of a disease burden on patients and their families who may have a harder time in attaining an accurate diagnosis, accessing treatment options and getting coverage of healthcare and related expenses. Funding for disease research and drug development also can be a challenge due to basic economics, whereas there is less of a demand for treatment because there are fewer patients.   

This Rare Disease Day, the ASBMB has gathered a collection of personal and research stories to raise awareness of this important health observance. You also can share snippets of your own research or personal experiences with rare diseases on social media using the hashtag #RareDiseaseDay.  

There are a number of career opportunities to get involved with rare diseases, from basic research into disease mechanisms to advocating for more funding and resources to develop treatment options. This week, we feature some rare-disease resources that can help you find potential employers, along with a weekly jobs roundup. An upcoming blog post will highlight jobs in orphan drug discovery to advance therapies for rare diseases.  

Rare disease resources  

  • The National Center for Advancing Translational Sciences and NIH Clinical Center are sponsoring Rare Disease Day at NIH on Feb. 28 at the main campus in Bethesda, Md. The event highlights research collaborations ongoing to address rare diseases with topical speakers, posters and exhibits, and networking opportunities. Admission is free and open to the public. There also is a live webcast available for those who cannot attend in person, and you can follow along on Twitter using the hashtag #RDDNIH.  
  • Another way to locate potential researchers to work with is to identify research centers and networks focused on rare diseases. As an example, the Rare Diseases Clinical Research Network is an initiative of the NCATS Office of Rare Diseases Research. The RDCRN website lists the currently funded research groups, along with the diseases each focus on and ongoing studies.  
  • Clinicaltrials.gov can be used to search for current and past clinical trials related to rare-disease therapies. A search within the field of “condition or disease” for “rare diseases” yields 446 results. The search results provided relevant information on the sponsors of each study and related publications, which can be useful information for your job searches.  

Weekly jobs roundup  

  • The National Organization for Rare Disorders offers internship opportunities for undergraduate and graduate students in various areas (e.g., public policy and advocacy, education and research) at its multiple office locations. The deadline to apply for summer internships is May 1. See the website for more details.  
  • Global Genes (Aliso Viejo, Calif., or remote) is hiring a program manager to produce quality content and organize speakers for its educational conferences and resources targeted towards the rare-disease patient and advocate communities. Events include the annual RARE Patient Advocacy Summit and RARE Drug Development Symposium. Minimum qualifications include a bachelor’s degree in journalism, public health or life science and two to three years of science-communications experience. No application deadline is provided.  
  • ICF, a global consulting and technology services provider, is recruiting an information specialist for the NIH’s Genetic and Rare Diseases Information Center. The specialist will respond to public inquiries about rare and genetic disorders and share relevant resources and information. The position can be based in Rockville, Md., or remote. Minimum qualifications include a master’s degree in human genetics or genetic counseling, one year of experience in clinical genetics and preference for candidates who are bilingual in English and Spanish. No application deadline is provided.  
  • The Neuroendocrine Tumor Research Foundation (Boston, Mass.) is seeking a director of research to lead its scientific research program to find treatments and cures for rare neuroendocrine tumors. Responsibilities include managing the research grant programs, liaising with the advisory boards and organizing the annual research symposium. Minimum qualifications include a master’s degree in a related field and five years of relevant medical or research experience. No application deadline is provided.  
  • Boston Children’s Hospital has an opening for a post-bachelor’s research assistant in neurobiology to support the Human Neuron Core research group. The RA will be involved with disease-modeling, differentiation and phenotyping assays. This includes processing clinical samples from patients with rare neurological disorders. Minimum qualifications include a bachelor’s degree in biology, molecular biology, biochemistry, neurobiology or related field and experience with cell cultures. No application deadline is provided.  
  • The Department of Pediatrics and Division of Medical Genetics at the University of Pittsburgh School of Medicine, in conjunction with the Center for Rare Disease Therapy at the Children's Hospital of Pittsburgh, is seeking an open-rank, tenure-track faculty member to lead a basic research program in rare diseases using cutting-edge technologies. See the job posting for details on how to apply. No application deadline is provided.  
  • The National Institute of Dental and Craniofacial Research is seeking a staff scientist to research rare skeletal diseases. Minimum qualifications include a Ph.D. with experience in bone and mineral metabolism and skeletal stem cell research. The application deadline is Feb. 28.  
  • Additionally, the Gourh lab within the National Institute of Arthritis and Musculoskeletal and Skin Diseases is hiring two postdoctoral researchers in immunology/molecular biology and bioinformatics/genetics to assist with studies on the genetics of the rare autoimmune disease called systemic sclerosis (scleroderma) among African-American patients. See the postings for details. No application deadlines are provided.  

Bonus opportunity  

The NIH Academy Enrichment Program is accepting applications from recent college graduates of diverse backgrounds who are interested in gaining biomedical research experience while learning more about addressing health disparities. Participants spend a year or two conducting research with an NIH mentor and participate in academy activities to explore the role of science, policy and community engagement in eliminating health disparities. Applicants must be U.S. citizens or permanent residents, eligible for the NIH Postbac IRTA program and planning to apply for graduate school after the program. You must both apply to the NIH Postbac IRTA program and submit a letter of interest to the NAEP to be considered. See the website for application instructions and links. The application deadline is Feb. 26.

 

Related posts  

Metabolomics jobs

Lipid jobs on the brain

Working on autoimmune and autoinflammatory diseases

Donna Kridelbaugh is a contributor to the ASBMB Careers Blog. She holds an advanced degree in microbiology and is a former lab manager.   

Stay updated on new posts by following the ASBMB on social media or click “follow” on this blog (must be a member and signed in). Also, be sure to check out the ASBMB Job Board for even more job listings.   

Science-policy jobs roundup

2/4/2019 5:25:31 PM

The impact of the recent partial government shutdown on science was beyond detrimental and trickled down through every facet of the scientific enterprise. Early-career scientists especially (and unfairly) were hit hard, incurring career-affecting consequences that included the loss of experimental data and missed training opportunities. And, the shutdown effects will linger for months as federal agencies try to resume normal operations and process the backlog of requests.  

This shutdown has further amplified the climate of science-funding uncertainty and underscores the critical need for scientists to be actively involved in science policy and advocacy. You have to make sure your voice is heard to ensure adequate resources are in place to support the important work you do.  

If the latest government shutdown has you motivated to take action, the ASBMB public affairs team has many opportunities for early-career scientists to get engaged in science advocacy. One training event for graduate students and postdoctoral researchers is the annual ASBMB Capitol Hill Day with applications due by Feb. 10.  

For those of you interested in pursuing a lifelong career in the policy realm, this week we feature a roundup of science-policy jobs and training opportunities to help you make that transition.  

Weekly jobs roundup  

  • The American Association for Cancer Research is accepting applications for its two-year science policy fellowship program. Fellows gain policy experience working rotations within the U.S. Congress, AACR Office of Science Policy and Government Affairs and National Cancer Institute or U.S. Food and Drug Administration. Minimum qualifications include a terminal science degree received within the past five years and legal eligibility to work in the U.S. The application deadline is Feb. 20.  
  • The American Cancer Society Cancer Action Network (Washington, D.C.) is recruiting a senior policy analyst to support the organization’s efforts in evidence-based policy and legislative solutions that will eliminate cancer as a major health problem. This position focuses on drug/device development and approval, federal appropriations, clinical trials and access to care and prevention issues. Minimum qualifications include a bachelor’s or master’s degree with a five to seven years of related work experience. No application deadline is provided. (Note: The ACS CAN also hosts policy interns who are senior undergraduates or graduate students. See the posting for more details.)  
  • The American Society of Human Genetics (Rockville, Md.) is hiring a policy and advocacy manager to monitor policy issues, draft related policy statements and materials and promote member engagement in advocacy campaigns. Minimum qualifications include a bachelor’s degree in a relevant science or policy area and four years of experience with science or health policy. No application deadline is provided.  
  • The Board on Health Sciences Policy within the National Academies of Sciences, Engineering and Medicine (Washington, D.C.) has several open positions that include a senior program assistant and program officer. The HSP works to strengthen the rigor of biomedical research and ensure the translatability of research to policy-making. As such, the board considers the ethical, legal and social contexts of scientific and technologic advances. Check out the job listings for more details. No application deadlines are provided.  
  • The global healthcare company Novo Nordisk hosts summer interns in federal government affairs. Interns will work with its government affairs team in Washington, D.C., to conduct outreach to congressional offices on sponsorship of priority legislation and help support the annual Novo Nordisk Inc. Capitol Hill Advocacy Day. Minimum qualifications include enrollment in a bachelor’s or master’s degree program in political science, health policy, government, public health or a related discipline. No application deadline is provided.  
  • The DuPont Specialty Products Division is looking for a political action committee manager for its government affairs office located in Washington, D.C. The incumbent will manage the daily operations of the office, provide administrative support to the team and perform related support tasks (e.g., track congressional hearings, prepare lobbying reports). Minimum qualifications include a bachelor’s degree and a strong desire to transition into government affairs. No application deadline is provided.   
  • The biopharmaceutical company Regeneron is hiring several congressional liaisons to work in Washington, D.C., to advance its public-policy and legislative initiatives. Minimum qualifications include a bachelor’s degree and six years of experience in a policy capacity, such as working with federal legislators and regulators. No application deadline is provided.  

Author’s note  

If you need to gain more experience in science policy, consider forming your own science-policy group to work on issues that matter to your peer network. Check out this past Scientific American blog post by Thaiesha Wright with some good tips on how to do so. For funding such a startup, you can look into establishing the group as an official student organization and applying for mini-grants through professional societies and organizations. As an example, the National Science Policy Network is accepting applications for its second round of microgrants. The application deadline is Feb. 22.

 

Related posts  

Resolve to a career in science policy

Defining your own professional identity outside academia

Advocacy training and jobs


Donna Kridelbaugh is a contributor to the ASBMB Careers Blog. She holds an advanced degree in microbiology and is a former lab manager.   

Stay updated on new posts by following the ASBMB on social media or click “follow” on this blog (must be a member and signed in). Also, be sure to check out the ASBMB Job Board for even more job listings