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On Sep 13, AbbVie (ABBV Quick Quote ABBV - Free Report) announced a deal to co-develop and co-commercialize Regenexbio's (RGNX Quick Quote RGNX - Free Report) investigational gene therapy for chronic retinal diseases like wet age-related macular degeneration wet AMD.

RGX-314 is being evaluated in a pivotal study for wet AMD, utilizing the subretinal method of delivery. It is also being studied in two separate phase II studies for diabetic retinopathy (DR) and wet AMD utilizing in-office suprachoroidal delivery.

AbbVie will make a $370 million upfront payment to Regenxbio for rights to RGX-314. Additionally, Regenxbio will also be entitled to milestone payments of up to $1.38 billion. Per the deal, while Regenxbio will be responsible for the completion of the ongoing studies of RGX-314, AbbVie will share costs for future studies, which include a second pivotal study for wet AMD utilizing subretinal delivery.

AbbVie’s latest gene therapy deal has brought this space once again in focus.

Gene Therapy: A Rapidly Growing Field

Gene therapy is set to become one of the most vital spaces with high prospects in the biotech sector. Scientists have been investigating gene therapies for more than 50 years. In this, rapidly-growing field, genetic, or inherited, diseases are treated by repairing or replacing the faulty genes that cause them. The idea is to see if a healthy or functional gene can be used to restore the function of a defective or mutated gene.

The promising gene therapy approach is being evaluated for varied diseases, such as hemophilia, Duchenne muscular dystrophy (DMD), Parkinson's disease, eye disease, and cancer among others.

A few FDA-approved gene therapy products are Roche’s subsidiary, Spark Therapeutics’ Luxturna to treat biallelic RPE65 mutation-associated retinal dystrophy, and Novartis’ subsidiary, AveXis’ Zolgensma for spinal muscular atrophy. Both these drugs are the first and the only gene therapy FDA-approved products of their kind. Some gene therapy cancer medicines like Bristol-Myers’ Abecma and Breyanzi, Novartis’ Kymriah, and Gilead’s Yescarta and Tecartus are also approved by the FDA for some lymphoma/leukemia indications.

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.

MeiraGTx Holdings (MGTX Quick Quote MGTX - Free Report)

MeiraGTx along with partner J&J plans to begin a phase III study on its lead pipeline candidate, AAV-RGPR as a treatment for patients with X-Linked retinitis pigmentosa (XLRP) in the second half of 2021.  It will also initiate a pivotal phase III study on AAV-RPE65 for patients with RPE65-associated retinal dystrophy in the second half of 2021.

It also has a phase I study ongoing on AAV-AQP1 for grade 2/3 radiation-induced xerostomia and plans to file an investigational new drug application later this year to begin clinical studies for another candidate AAV-GAD for Parkinson’s Disease.

Voyager Therapeutics (VYGR Quick Quote VYGR - Free Report)

Voyager is developing gene therapies with its novel proprietary AAV capsids that have a significant potential to be more reliably on-target with less risk of dose-limiting toxicities. It has a rich early-stage/pre-clinical pipeline of new and second-generation programs in Huntington’s disease, Monogenic ALS (SOD1), spinal muscular atrophy, and diseases linked to GBA1 mutations.

Solid Biosciences (SLDB Quick Quote SLDB - Free Report)

Solid Biosciences’ lead gene therapy candidate is SGT-001, which is being evaluated in a phase I/II study in patients with DMD. Further, preclinical studies on its next-generation DMD gene therapy program, SGT-003, are progressing rapidly.

Some other key names in the gene therapy space are Wave Life Sciences, Sangamo Therapeutics, Regenxbio, Pfizer, and Roche’s subsidiary Spark Therapeutics.