Cambridge Healthtech Institute’s 2nd Annual

Drug Discovery for Rare Diseases

Exploring New Technologies, Targets and Drug Modalities

March 28, 2019

Rare diseases, or diseases that affect only a small percentage of the population, have grown in significance and prominence in recent years. According to the National Institutes of Health, there are nearly 7000 rare diseases and more than 25 million Americans who are affected. Approximately 80% of these rare diseases are genetic in origin. Cambridge Healthtech Institute’s symposium on Drug Discovery for Rare Diseases will bring together leading scientists, clinicians, executives and experts who are involved in finding new drug targets and drug modalities for treating rare disorders. This unique one-day event will encourage people from diverse backgrounds to discuss potential opportunities, as well as existing challenges in this field. It’s an opportunity for scientific and technical experts to come together to exchange ideas, experiences and to set up collaborations for rare disease research.

Coverage will include, but is not limited to:

  • New models and approaches to identify novel drug targets and modalities for treating rare diseases
  • Using CRISPR, microfluidics, patient-derived stem cells, and other innovative tools for rare disease research
  • Finding computational and bioinformatics tools for identifying disease pathways and drug targets
  • Exploiting proteomics, genomics, and metabolomics tools for drug and target discovery
  • Exploring the use of gene editing to enhance gene and cell therapy for rare diseases
  • Discussion on translational and regulatory challenges for orphan drug development
  • Case studies on successes or failures: valuable lessons learnt

If you would like to submit a proposal to give a presentation at this meeting, please click here.

The deadline for priority consideration is September 28, 2018.


All proposals are subject to review by session chairmen and/or the Scientific Advisory Committee to ensure the overall quality of the conference program. Additionally, as per Cambridge Healthtech Institute policy, a select number of vendors and consultants who provide products and services will be offered opportunities for podium presentation slots based on a variety of Corporate Sponsorships.


For more details on the conference, please contact:

Tanuja Koppal, PhD

Conference Director

Cambridge Healthtech Institute

For partnering and sponsorship information, please contact:

Carolyn Cooke

Business Development Manager

Cambridge Healthtech Institute

Phone: (+1) 781-972-5412