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Rare Disease & Regenerative Medicine

This report provides industry specific statistics compiled from cell therapy, gene therapy, tissue engineering, and other regenerative medicine companies active in developing therapies for rare disease and provides context for the potential of regenerative medicine to improve upon the current standard of care.

As of the end of 2018:

There are 323 companies worldwide working on regenerative medicine therapies in rare disease.

There are currently 600+ products and product candidates in clinical development or marketed

There are an estimated 300 million patients suffering from rare diseases worldwide.

Full Report Here


Clinical trials underway
worldwide by the end of 2018

PH. 1


PH. 2


PH. 3


Clinical Trials by Technology Type

Gene Therapy
Cell Therapy
Cell Therapy
Tissue Engineering

Clinical Trials by Indication

Companies Active in Developing Regenerative Medicines for Rare Diseases



2018 Global Financings for Regenerative Medicines for Rare Disease

Total Global Financing

$9.7 Billion


Increase from 2017

Gene & Gene-modified
Cell Therapy

$8.2 Billion


Increase from 2017

Cell Therapy

$5.4 Billion


Increase from 2017

Tissue Engineering

$256.2 Million


Increase from 2017