Gene therapy is nothing short of incredible. It works to deliver correct versions of defective genes to cells, often through viral vectors. Many gene therapies aim to provide long-term or permanent treatment in a single dose, which is why they receive so much attention in the media and from investors alike. 

Gene therapy is particularly effective for rare diseases, as well as diseases that are debilitating and involve lifelong medication. In essence, gene therapy is the chance for a potential cure, removing the need for chronic treatment and the exponential cost of having a rare or life-long disease. Finally, the beauty of gene therapy is that it can be tailored for specific genetic mutations, which is why it’s so suited to rare diseases.

Our 3 Spotlight Organizations Revolutionizing Gene Therapy 

Allogene Therapeutics

Allogene are focussed on allogeneic CAR T-cell therapy, which is a form of gene therapy for cancer treatment. Headquartered in San Francisco and founded in 2018, their mission is to “create and lead the next revolution in cell therapy by delivering to patients the first allogeneic CAR T-cell (AlloCAR T™) products for blood cancers, solid tumors and autoimmune disease.” Although not specifically focused on rare diseases, Allogene’s pipeline targets a vast array of tumor types and autoimmune indications. 

“At Allogene, we are developing a pipeline of "off-the-shelf" CAR T cell candidates, with the goal of delivering readily available cell therapy faster, more reliably, and at greater scale to more patients.”

 To learn more about Allogene and the impact they’re having within the gene therapy space, click here. 

Enliven Therapeutics

Enliven Therapeutics works on developing gene therapies for cancer. They are a Biopharma developing kinase inhibitors* that enhance efficacy through better selectivity and combinability, combat resistance, address brain metastases, and improve safety and patient convenience. 

They are currently advancing two parallel lead product candidates, currently in Phase I of clinical trials. 

 Enliven is committed to providing solutions for patients who have exhausted currently available targeted treatments. For them, it’s about providing a cure, but equally providing a better quality of life for patients who have developed resistance to other medications and treatments. 

Enliven’s vision is accompanied by a robust Clinical and Regulatory strategy with high-quality datasets, to provide opportunities to enable future trials in earlier lines of therapy. 

To learn more about Enliven Therapeutics, click here.

*Certain kinases are more active in some types of cancer cells, tumors and blocking them may help keep the cancer cells from growing. Kinase inhibitors may also block the growth of new blood vessels that tumors need to grow. [National Cancer Institute]

T-Knife Therapeutics

 T-Knife Therapeutics have been in the gene therapy game for almost a decade, and have been instrumental in developing supercharged T-cell therapies. They work tirelessly, engineering T-cells with ‘tailored, best-in-class enhancements’ to create TCR-Ts. In layman terms, these are proteins that are genetically modified to treat various cancers. Some T-cell therapies have been promising in terms of results, including solid tumours and haematological malignancies. 

T-Knife’s pipeline is impressive, currently targeting cancers such as skin, uterus, ovaries, lungs, breasts, esophagus, kidneys, cervix, and head & neck (PRAME), colon, skin, brain, lungs, prostate, and breasts (MAGE-A1) and lungs, pancreas, colon, and rectum (KRASG12V).

 To learn more about T-Knife, click here.

Our 2 Spotlight Organizations Revolutionizing Gene Therapy for Rare Diseases

Sana Biotechnology

 Sana Biotechnology is focused on developing engineered cell therapies, inclusive of applications for rare diseases. 

Founded in 2020 and based in Seattle, Washington, Sana aims to harness the power of gene therapy and cell engineering to create innovative treatments that can correct or replace defective genes and enhance the body’s immune response to diseases.

“Repairing and controlling genes in cells or replacing missing or damaged cells can solve the underlying cause of many diseases. These emerging capabilities will create novel medicines that meaningfully improve patient outcomes, and Sana aims to lead the way.”

Their current pipeline includes 3 different product candidates in Phase I as well as 3 product candidates in pre-clinical stages. Sana’s long-term goal is to create “off-the-shelf” cell therapies that can be administered to patients without the need for extensive customization. Their therapeutic areas include rare diseases as well as haematological and solid tumors.

To learn more about Sana Biotechnology, click here.

Cargo Therapeutics

 Cargo Therapeutics is focussed on developing the next-generation transformational CAR T-cell therapies. Whilst their focus is slightly broader, they also explore gene therapies that can address rare diseases. Cargo’s scientific platforms enable multi-specific and multi-functional CAR T programs. 

"Our programs, platform technologies and manufacturing strategy are designed to address the problems of cancer resistance mechanisms and unreliable supply. We are developing technologies that incorporate multiple transgene therapeutic “cargo” to potentially extend the persistence of our CAR T-cell therapy candidates, with the goal of achieving durable responses that are curative for more cancer patients.”

Their pipeline is modest in comparison to others in the space; however, they are mid-way through Phase II of their Firi-cell program.

 To learn more about Cargo Therapeutics, click here. 

Currently, on the market

What Gene Therapies are currently live on the market for patients?

• Lenmeldy | Orchard Therapeutics: Approved for the treatment of metachromatic leukodystrophy (MLD).
• Casgevy | Vertex Pharmaceuticals and CRISPR Therapeutics: This is the first CRISPR-based gene editing therapy approved in the U.S. for sickle cell disease (SCD) and beta-thalassemia.
• Elevidys | Sarepta Therapeutics: Approved for Duchenne muscular dystrophy (DMD), Elevidys is a gene therapy that aims to increase the expression of micro-dystrophin, a protein crucial for muscle function

The companies leading the charge in gene therapy are offering solutions that could permanently modify or even cure the root causes of rare diseases, dramatically improving patient outcomes. Nevertheless, challenges in scaling production and ensuring cost-effectiveness for these complex therapies remain significant, although market forecasts suggest robust growth throughout the decade. In 2024, these enterprises are setting the stage for gene therapy breakthroughs that not only enhance the lives of rare disease patients but also reshape market dynamics and regulatory policies in the healthcare sector.

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