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.   

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