RARE Daily

Cell and Gene Therapy Companies Raised $6.4 Billion in 2019 for Rare Disease

February 24, 2020

Rare Daily Staff

Companies developing regenerative medicine therapies to treat rare diseases including gene therapies, gene-based therapies, and cell therapies, raised $6.4 billion in 2019, according to a new report from the Alliance for Regenerative Medicine.

The group found it was the second highest year on record for venture financing for rare disease in the regenerative medicine sector with more than $1.5 billion raised through venture financings.

CRISPR Therapeutics, which is developing gene-editing therapies from hemoglobinopathies completed the largest financing for a rare disease company in the regenerative medicine space in 2019 with a $274 million follow-on public offering.  Maze Therapeutics, which launched with an initial investment of $191 million a year ago, was the largest regenerative medicine private financing in the rare disease space in 2019. The company is seeking to modulate disease causing genes through the regulation of other genes.

U.S. Food and Drug Administration approvals in 2019 included the gene therapy Zolgensma for spinal muscular atrophy type 1, a progressive and deadly neuromuscular disease. The year also saw European Union approval of Zynteglo, BlueBird Bio’s gene therapy to treat beta thalassemia, a rare and debilitating blood disease.

The report also highlighted anticipated near-term approvals, which in the United States includes BioMarin’s gene therapy ValRox for hemophilia A, Enzyvant’s tissue engineering therapy for pediatric congenital athymia, and BlueBird Bio’s Zynteglo.

The report noted there are more than 400 companies worldwide working to develop regenerative medicines and advanced therapies to treat rare diseases and 647 ongoing clinical trials, 42 of which are late-stage studies.

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