5 Gene Therapy Stocks Back In Focus On AbbVie/Regenxbio Deal

Given the potential of gene therapies to treat complex diseases, the companies developing candidates using gene therapy that are a mix of large and small firms, are in focus. A successful medicine developed by any of these companies can have the potential to generate annual revenues of $1 billion or more. All the five companies have a Zacks Rank #3 (Hold). 

5 Gene Therapy Companies in Focus

uniQure N.V (QURE Quick Quote QURE - Free Report)

This company is a promising player in the gene therapy space. It is engaged in creating a pipeline of innovative gene therapies that have been developed both internally and through collaborations.

The company is developing AAV5-based gene therapy, Etranacogene dezaparvovec in late-stage studies for hemophilia B. A biologics licensing application (BLA) for Etranacogene dezaparvovec is expected to be filed in the first quarter of 2022. Also, a phase I/II study is ongoing for another gene therapy candidate, AMT-130 for Huntington’s disease in its portfolio.

 In June, uniQure announced the planned acquisition of Corlieve Therapeutics, a France-based pre-clinical gene therapy company.

Sarepta Therapeutics (SRPT Quick Quote SRPT - Free Report)

Sarepta’s lead gene therapy candidate is SRP-9001, an AAV-mediated micro-dystrophin gene therapy, which is being evaluated in a phase I/II study for DMD. The company plans to initiate a pivotal clinical study this year. The promising candidate has also led Roche to sign a collaboration deal with Sarepta. The company plans to seek FDA’s approval to start a pivotal study on its other gene therapy candidate, SRP-9003, in 2021 to evaluate it in patients with Limb–girdle muscular dystrophy (LGMD) type 2E. The company has several other pre-clinical and clinical-stage gene therapy candidates targeting additional indications like Rett Syndrome, cardiomyopathy, Emery-Dreifuss muscular dystrophy type 1, and multiple sclerosis.

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