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.


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

587+

Clinical trials underway
worldwide by the end of 2018

PH. 1

202

PH. 2

342

PH. 3

43

34.4%58.3%7.3%050100150200250300350Phase 1Phase 2Phase 3

Clinical Trials by Technology Type

Gene Therapy
27.2%62.9%9.8%020406080100120140Phase IPhase IIPhase III
Gene-Modified
Cell Therapy
43.1%52.7%0020406080100120140160Phase IPhase IIPhase III
Cell Therapy
25.6%65.4%9.0%0510152025303540455055Phase IPhase IIPhase III
Tissue Engineering
50.0%50.0%0-0.200.20.40.60.811.21.41.61.822.2Phase IPhase IIPhase III

Clinical Trials by Indication

72.4%5.6%0050100150200250300350400450OncologyHematologyEndocrine, Metabolic and Genetic DisordersOphthalmologyCentral Nervous SystemImmunology and InflammationMusculoskeletalDermatologyCardiovascularRespiratoryInfectious DiseasesGenitourinary DisordersGastroenterology

Companies Active in Developing Regenerative Medicines for Rare Diseases

 

Financings

2018 Global Financings for Regenerative Medicines for Rare Disease

Total Global Financing

$9.7 Billion

 48%

Increase from 2017

Gene & Gene-modified
Cell Therapy

$8.2 Billion

 39%

Increase from 2017

Cell Therapy

$5.4 Billion

 39%

Increase from 2017

Tissue Engineering

$256.2 Million

 2552%

Increase from 2017

Infographics