Cambridge Healthtech Institute’s 3rd Annual

Rare Disease Drug Development

New Drug Modalities and Emerging Trends

March 19, 2020


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, over 80% of which are genetic in origin, that affect more than 25 million Americans. Only 5% of these rare diseases have an approved treatment option, although precision medicine is now helping by allowing us to define numerous new types of “rare” diseases that are specific subsets of major disease categories. Cambridge Healthtech Institute’s annual symposium on Rare Disease Drug Development will bring together leading scientists, clinicians, executives and experts involved in finding new drug targets, drug modalities and innovative strategies for treating orphan diseases. This focused one-day event will encourage scientific and technical experts to share recent findings, new technologies and best practices for rare disease research and drug development.


Coverage will include, but is not limited to:


  • The changing face of “rare” diseases in the world of precision medicine
  • New models and approaches to identify drug targets for treating rare diseases
  • Emerging drug modalities for orphan drugs- oligos, mRNA, CRISPR, stem cells, gene and cell therapy
  • Exploiting AI, modeling and simulation for improving rare disease monitoring and drug development
  • Pursuing undruggable targets and untreatable rare diseases using new tools
  • Understanding translational and regulatory challenges and opportunities for orphan drug development
  • Effective use of predictive biomarkers for efficacy and safety
  • 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 9th 2020.


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

Senior 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

Oligonucleotide Discovery and Delivery