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

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.

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