Rare Disease & Regenerative Medicine 2019


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 2019:

There are 420 companies worldwide working on regenerative medicine therapies in rare disease.

There are currently 647 ongoing clinical trials utilizing regenerative medicines to treat rare disease.

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

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A rare disease typically affects fewer than five in 10,000 people worldwide. Despite the rarity of each disease individually, when considered collectively, rare diseases affect a significant population – there are roughly 30 million people in the US and more than 300 million people worldwide living with a rare disease. There are nearly 7,000 different rare diseases currently identified, while only about 5% have an FDA-approved treatment. In addition, these diseases are often debilitating and potentially fatal; about 30% of children with a rare disease will die before their fifth birthday. Approximately 80% of rare diseases have identified genetic origins.

647+

Regenerative medicine clinical
trials in rare disease underway
worldwide by the end of 2019

38.9%54.6%6.5%050100150200250300350Phase 1Phase 2Phase 3

Clinical Trials by Technology Type

Gene Therapy
28.2%60.9%10.9%02030405060708090100110120130140Phase IPhase IIPhase III
Gene-Modified
Cell Therapy
49.6%47.9%0020406080100120140160180Phase IPhase IIPhase III
Cell Therapy
20.0%70.0%10.0%05101520253035404550Phase IPhase IIPhase III
Tissue Engineering
33.3%33.3%33.3%0-0.200.20.40.60.811.21.41.61.822.2Phase IPhase IIPhase III

Clinical Trials by Indication

Rare Cancers
469
Endocrine, Metabolic and Genetic Disorders
37
Hematology
34
Ophthalmology
21
Central Nervous System
21
Immunology and Inflammation
20
Musculoskeletal
17
Dermatology
9
Cardiovascular
6
Gastroenterology
5
Respiratory
4
Genitourinary Disorders
3
Lymphatic Diseases
1

Companies Active in Developing Regenerative Medicines for Rare Diseases

 

201North America7Oceania111Asia101Europe & Israel

Financings

2019 Global Financings for Regenerative Medicines for Rare Disease

Total Global Financing

$6.4 Billion

Gene & Gene-modified
Cell Therapy

$5.6 Billion

Cell Therapy

$3.5 Billion

Rare Cancers

$2.9B

Infographics