samedan logo
 
 
 
spacer
home > ebr > autumn 2021 > special delivery
PUBLICATIONS
European Biopharmaceutical Review

Special Delivery

EBR: What are some of the key issues in developing treatments for orphan diseases, especially when it comes to delivering payloads?

Sonya Montgomery: Despite scientific advances supported by regulatory and financial incentives to promote the development of therapies for rare and orphan diseases, fewer than 5% of the more than 7,000 rare diseases believed to affect humans currently have an effective treatment (1).

These conditions are complex and often not well understood, with many patients encountering significant delays in being properly diagnosed, making it challenging to develop effective treatments. Beyond the difficulty in characterising the pathophysiology and genotype to phenotype relationship in diseases with small patient populations to develop effective therapies, the greatest challenge developers face in addressing orphan diseases are two-fold: firstly, the ability to safely and repeatedly dose therapeutics without concern for immunogenicity, and secondly, to get therapeutics across biological barriers to target tissues in sufficient quantity to have a clinically meaningful benefit.

Read full article from PDF >>

Rate this article You must be a member of the site to make a vote.  
Average rating:
0
     

There are no comments in regards to this article.

spacer
Sonya Montgomery’s extensive experience in early development includes a focus on translational medicine, leading programmes in gene therapy, biologics, including antisense RNA, and small molecules. She has led successful interactions with health authorities globally, supporting investigational new drug/clinical trial applications, first-in-class new molecular entities, and negotiation of a special protocol assessment leading to new drug applications and marketing authorisation applications. Prior to joining Evox Therapeutics, Sonya was most recently at Gyroscope Therapeutics, where she established and led clinical development, translational medicine, and clinical genetics. Previously, Sonya has held positions in clinical development at ProQR, Relypsa, and Pfizer. She joined industry in 1997, and early in her career combined biopharmaceutical research with a clinical practice.
spacer
Sonya Montgomery
spacer
spacer
Print this page
Send to a friend
Privacy statement
News and Press Releases

Stevanato Group signs an agreement with Nelson Labs to provide best-in-class Extractables and Leachables testing services at its US Technology Excellence Center

Piombino Dese, Padua Italy and Salt Lake City, USA September 2, 2020: Italian-based Stevanato Group, a leading producer of glass primary packaging and provider of integrated services for combination products, and Nelson Laboratories, LLC, a global provider of lab testing and expert advisory services signed today a partnership agreement that will provide Extractables and Leachables testing at SG US Technology Excellence Center in Boston, MA.
More info >>

White Papers

Finding the Right End-to-End Safety Solution for Your Needs

Bioclinica

With upcoming changes, including the implementation of the International Conference on Harmonisation (ICH) E2B(R3) Electronic Transmission of Individual Case Safety Report and Identification of Medicinal Products (IDMP) standards, the current state of safety reporting can be confusing. Your existing safety system may not be flexible enough to accommodate these changing regulations, which are still moving targets regarding the details needed for a comprehensive solution with the right level of processes, company-to-company integrations and finalized regional rules.
More info >>

 

 

 

©2000-2011 Samedan Ltd.
Add to favourites

Print this page

Send to a friend
Privacy statement