5 Gene Therapy Companies That May Transform the Medical Industry

The gene therapy field is growing dramatically and is expected by many to be the future of medicine for a myriad of diseases. Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect, the treatment of disease by repairing defective genes and in some cases reintroducing deleted genes into the body. It holds promise to treat, or even cure, a wide range of diseases. These types of treatments were presented in science fiction stories in the 1950s and became a scientific reality with the first approval by the US Food and Drug Administration (FDA) in the late 2000’s. 

According to the US Food and Drug Administration, there are at least five gene therapy technologies including: 

• Plasmid DNA
• Viral Vectors
• Bacterial Vectors
• Human Gene Editing Technology 
• Patient-Derived Cellular Gene Therapy 

With over 50 years of research, gene therapy is becoming a medical reality. In some treatments, gene therapy is on its way to becoming the leading standard treatment. Treatments are being developed primarily for rare disease, but more recently even for diseases with large patient populations with unmet medical needs. Such treatments are considered for but not limited to various forms of cancer, non-small cell lung cancer, small cell lung cancer, type-1 diabetes, type-2 diabetes, hemophilia, sickle cell anemia, cystic fibrosis, and HIV/AIDS, each utilizing gene therapy treatments. 

One study indicates the gene therapy market could have a CAGR of 20.2% with a 2029 valuation of $13 billion (USD). Here are 5 gene therapy companies to stay informed on. 

Bluebird Bio

Bluebird Bio is based in Sommerville, Massachusetts. They are focused on gene addition utilizing autologous hematopoietic stem cell transplant (HSCT) in conjunction with lentiviral vectors (LVV) that are uniquely customizable for approaching different diseases and their underlying causes. The stem cells used are from the patient themselves, so it does not require finding a matching donor, enables it to overcome graft rejection, and does not present any personal concerns with the origins of the stem cells used. This is a type of patient-derived cellular gene therapy.

Bluebird Bio has three diseases it targets in its gene therapy clinical trials. These include Beta-Thalassemia, Cerebral Adrenoleukodystrophy (CALD), and Sickle Cell Disease (SCD).

4D Molecular Therapeutics

4D Molecular Therapeutics (4DMT) is based in Emeryville, California. They are a clinical-stage biopharma company inventing and developing innovative products to unlock the full potential of genetic medicine to treat large-market diseases. 4DMT utilizes a transformative vector discovery platform to use gene therapy to deliver therapeutic payloads to the specified diseased tissue. This is a viral vector technology. A part of their platform incorporates Nobel-prize-winning Directed Evolution technologies that allow scientists to invent designer biologics with more desirable characteristics. Two of the diseases 4DMT specializes in are wet age-related macular degeneration (Wet AMD) and diabetic macular (DME). 

4DMT currently has 2 studies underway for Wet AMD and DME, with anticipation to have enrollment by Q3 2023 for DME treatments. 

Genprex, Inc.

Genprex, Inc. is based in Austin, Texas (Disclosure: The author of this article is the CEO of Quantum Media Group. Genprex, Inc. is a client of Quantum Media Group). They are a clinical-stage gene therapy company focused on treatments for patients with cancer and diabetes. Genprex’s oncology program utilizes its proprietary, non-viral ONCOPREX® Nanoparticle Delivery System, which may be the first systemic gene therapy delivery platform used for cancer in humans. Their lead product candidate, REQORSA®, is being evaluated in Phase 1/2 clinical trials for the treatment of non-small cell lung cancer (NSCLC) and small cell lung cancer. Each of Genprex’s clinical trials in NSCLC have received a Fast Track Designation from the U.S. Food and Drug Administration. Genprex is also developing a gene therapy treatment for Type-1 and Type-2 diabetes that restores and rejuvenates beta cells, that are responsible for producing insulin in the body. 

Poseida Therapeutics

Posedia Therapeutics is a San Diego based company developing several gene therapies to treat rare and life-threatening diseases including Ornithine Transcarbamylase Deficiency (OTCD), Hemophilia A and Phenylketonuria (PKU), three genetic liver diseases that are usually diagnosed in the early neonatal period. They also specialize in off-the-shelf plasmid DNA CAR-T treatments for liquid and hard tumors, which is widely considered revolutionary in its approach to treating prostate cancer. 

Poseida Therapeutics Founder, Eric M. Ostertag, M.D., Ph.D., is leading the company in next-generation solutions to tackle even more complex diseases and cancers while researching single-treatment cures. Dr. Ostertag’s public statement on the future of Poseida Therapeutics: “We are developing therapies to revolutionize the treatment of cancers and genetic disease in pursuit of single treatment cures.”

CRISPR Therapeutics

CRISPR Therapeutics is based in Zug, Switzerland while their research and development is conducted in Boston, Massachusetts. CRISPR Therapeutics is translating their specific, efficient, and versatile CRISPR/Cas9 gene-editing platform into therapies to potentially treat hemoglobinopathies, cancer, diabetes, and other diseases. They focus on four core franchises including hemoglobinopathies, immuno-oncology, regenerative medicine, and in vivo. Their hemoglobinopathy treatments are for Beta-Thalassemia and Sickle Cell Disease (SCD). Their immuno-oncology takes an allogeneic CAR-T approach to various types of lymphomas, and solid tumors, including renal cell carcinoma. This is a plasmid DNA technology. CRISPR’s regenerative medicine therapy is primarily for diabetes. Their in vivo franchise treats cardiovascular diseases, hemophilia A, Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), and cystic fibrosis (CF).

Investing in Gene Therapy

There is a lot of healthy excitement happening in the gene therapy space! Over the past few years, research and development in gene therapy have dramatically increased in the US and internationally, and the results are demonstrating positive healthy returns in this burgeoning space. Gene therapy has quickly become the next-gen exploration of the open frontier in the medical world, taking what was once science fiction and making it a reality for a healthier world.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.