2 Low-Priced Gene Therapy Stocks to Speculate On

 | Jan 09, 2024 02:06PM ET

  • Bluebird Bio has three FDA-approved gene therapy treatments that are planning to commercialize after its $150 million secondary offering of common shares priced at $1.50 per share.
  • Bluebird bio's latest FDA approval, LYFGENIA, is a one-time treatment for sickle cell disease, which is priced around $3.1 million per patient, which is 40% higher than Vertex Pharmaceutical's Casgevy at $2.1 million, which was approved on same day Dec. 8, 2023.
  • Sangamo Therapeutics has paused further investment into two gene therapy programs until it can find a collaboration partner, as it only has funds to operate to Q3 2024.

    • Gene therapy differs from gene editing, which is getting the lion's share of headlines. While gene editing is a form of gene therapy, there are basic differences. Gene-editing involves using techniques like CRISPR/Cas9 to precisely target and cut or modify the sequence of human genomes to manage hereditary diseases.

    Gene therapy adds or replaces genes with a functional copy into a patient’s cells utilizing various viruses or AAV capsids. A Global Data study expects cell and gene therapy (CGT) to be a top industry trend in 2024 and expects the market to grow to over $80 billion by 2029. Here are two low-priced gene therapy stocks in the medical sector that speculators may watch to keep an eye on.

    h2 Bluebird bio Inc/h2

    This clinical-stage biotech specializes in developing gene therapies for severe genetic and rare diseases. The company is focused specifically on sickle cell disease (SCD) and beta-thalassemia (BT). bluebird is also exploring gene therapies for rare diseases like transfusion-dependent thalassemia (TDT) and cerebral adrenoleukodystrophy (CALD).

    h2 FDA-approved gene therapies/h2

    Bluebird bio Inc (NASDAQ:BLUE) has three FDA approved gene therapies:

    ZYNTEGLO (betibeglogene autotemcel) is a gene therapy treatment for beta-thalassemia characterized by the inefficient production of healthy red blood cells. Patients receive functional copies of the beta-globin gene. Studies have shown that 89% of patients achieved transfusion independence.

    SKYSONA (elivaldogene autotemcel) is a gene therapy treatment for early active cerebral adrenoleukodystrophy (CALD). It’s the first and only gene therapy proven to slow the progression of neurologic dysfunction in boys with early and active CALD.

    LYFGENIA (lovotibeglogene autotemcel) was FDA-approved on Dec. 8, 2023, at the same time as Casgevy for the treatment of sickle cell disease (SCD). LYFGENIA is the longest-studied approved gene therapy for SCD. It's designed to allow the production of anti-sickling hemoglobin to decrease vaso-occlusive events.

    h2 Competing Sickle Cell Disease (SCD) treatments/h2
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    Incidentally, Vertex Pharmaceuticals Inc (NASDAQ:VRTX). and Crispr Therapeutics AG (NASDAQ:CRSP) received FDA approval for Casgevy, a CRISPR/Cas9 therapy for SCD for patients with recurrent vaso-occlusive crises (VOCs) on Dec. 8, 2023. Vertex announced its treatment would be priced at $2.2 million per patient, coming in cheaper than the $3.1 million per patient that Bluebird Bio is charging for its SCD therapy.

    h2 Raising more funds for commercialization/h2

    On Dec. 20, 2023, Bluebird Bio announced a $150 million secondary stock offering of 83.34 million shares priced at $1.50. This caused shares to gap down 41% from its previous trading day closing price of $2.43 on Dec. 19, 2023, to $1.45. Investors were taken by surprise as shares fell as low as $1.26 before staging a slow rally back towards $1.45. The company said the offering, combined with existing funds, should help the company fund operations through the first quarter of 2025. Net proceeds will be used to support the commercialization of its three approved gene therapies and to fund working capital and general corporate purposes.

    Bluebird bio analyst ratings and price targets are at MarketBeat. bluebird bio peers and competitor stocks can be found with the MarketBeat stock screener.