Search ARM

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

587+

Clinical trials underway
worldwide by the end of 2018

PH. 1

202

PH. 2

342

PH. 3

43

Clinical Trials by Technology Type

Gene Therapy
Gene-Modified
Cell Therapy
Cell Therapy
Tissue Engineering

CAR-T Therapies

73


TCRs

17


APC or Dendritic Vaccines

15


Other Gene-Modified Cell Therapies

16


Stem Cell Therapies

14


Other Cell Therapies

6

Adenoviral Vector

9


AAV Vector

9


Lentiviral Vector

8


Other Viral Vectors

10


Non-Viral Vectors

3


Gene Editing Trials

8

Clinical Trials by Indication

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