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4 Biotechs to Watch Amid Rising Prominence of Gene Therapies

Gene therapy is one of the novel mechanisms of treatment that is attracting several large and small pharma companies. Gene therapies treat a disease by altering or turning off problematic genes and adding genes that help to fight or treat a disease. Scientists have been investigating ways to modify genes or replace faulty genes for the last few decades with a few gene therapies already in the market. Gene therapy is set to become one of the most vital spaces with high prospects in the biotech sector.

These therapies provide the flexibility to develop one-time treatment options for genetic or inherited diseases with limited or no approved therapies available. Moreover, gene editing can directly affect cells — the basic building blocks of living things — and may help in developing highly effective therapies.

FDA-Approved Gene Therapies

There are already a few FDA-approved gene therapies targeting different difficult indications. In a historic move, the FDA approved the first gene therapy, Novartis’ Kymriah, for treating acute lymphoblastic leukemia in 2017. This was followed by the FDA approval of two more gene therapies — Gilead’s Yescarta and Roche’s Luxturna — for oncology and eye disorder indications, respectively, in the same year. The FDA approved two new gene therapies, Bristol-Myers’ Breyanzi and Abecma for treating different cancer indications, earlier in 2021. In 2022, BioMarin received approval for its hemophilia A gene therapy — Roctavian. The majority of approved gene therapies have shown strong sales growth since their approval.

Stocks to Watch

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 generate annual revenues of $1 billion or more. Here we discuss four biotech stocks with promising gene therapy candidates in their pipeline.

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CRISPR Therapeutics CRSP

The company is developing its lead pipeline candidate, exagamglogene autotemcel (formerly CTX001), in collaboration with Vertex Pharmaceuticals in late-stage studies as a potential treatment for transfusion-dependent beta-thalassemia and sickle cell disease. The gene-editing therapy candidate previously demonstrated a consistent and sustained response to treatment in the given patient population in earlier-stage studies.

CRISPR Therapeutics is actively seeking collaborations and leveraging its CRISPR/Cas9 gene-editing platform to make therapies for hemoglobinopathies, cancer, diabetes and other diseases.

Editas Medicine EDIT

The company’s lead pipeline candidate is EDIT-101, which employs CRISPR gene editing to treat LCA10 — a rare genetic illness that causes blindness. Editas has completed enrolling in the first pediatric cohort of the phase I/II BRILLIANCE study, which is evaluating EDIT-101 for LCA10. The study has several other cohorts evaluating the gene therapy candidate in pediatric as well as adult patients with LCA10. Editas is also pursuing the development of CRISPR candidates for eye diseases other than LCA10, including Usher Syndrome type 2A and recurrent ocular Herpes Simplex Virus type 1.

It is also designing novel medicines for non-malignant hematologic diseases, such as SCD and beta-thalassemia.

Sarepta Therapeutics SRPT

Sarepta’s lead gene therapy candidate is SRP-9001, an AAV-mediated micro-dystrophin gene therapy. The company is currently evaluating SRP-9001 in a pivotal clinical study as a one-time treatment for Duchenne muscular dystrophy patients. The promising candidate has also led Roche to sign a collaboration deal with Sarepta. Sarepta is currently planning to file a biologics license application in the fall of 2022, seeking accelerated approval for SRP-9001. The company plans to develop its other gene therapy candidate, SRP-9003, 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.

Beam Therapeutics BEAM

Beam Therapeutics has two pre-clinical gene editing candidates, BEAM-101 and BEAM-102, in its pipeline that are being developed as potential treatments for SCD. The company’s investigational new drug (IND) application to the FDA, seeking approval to start a clinical study on BEAM-101 was approved by the FDA earlier this year. Beam Therapeutics is preparing for the initiation of the study soon. The company is planning to file an IND application with the FDA for BEAM-102 in the rest of 2022.


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