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Invest in CRISPR

Gene Therapy incorporates a wide variety of methods to repair and modify the human genes. Whether inserting new DNA or replacing damaged genes with healthier ones, gene therapy is an exciting emerging field in the medical arena.

Traditionally, gene therapy is accomplished by inserting a new gene into a cell with the use of a vector, a carrier that is typically a virus. Viruses are used because they can infect the cell, but do not cause disease. Vectors may be administered via either IV or syringe directly into the patient or blood can be extracted and the vector administered in a lab before putting it back into the patient.

One of the more impressive achievements in not only genetics, but all fields of science, is the discovery of Clustered Regularly-Interspaced Short Palindromic Repeats, more widely known simply as CRISPR. With CRISPR technology (a subtype of gene therapy), scientists can target specific genes to knock out, introduce specific genes into our genome, or even isolate a mutated gene to enhance it. CRISPR is a sequence of code found in bacteria and within this sequence of code is RNA. With RNA acting as a “guide”, the enzyme Cas9 is able to locate defected genes and “cut” a section of DNA and disables, repairs, or inserts new genes in its place. Although in its nascent stage, if CRISPR lives up to its hype it has potential to completely eradicate genetic disease(s).

The Benefits of CRISPR

The market is currently flooded with companies in the gene therapy space. Advancements and innovation within the industry has led to an endless amount of applications for the use of the technology. While various gene editing techniques exist, none has become as popular as CRISPR which has emerged as the clear favorite as it is the most cost effective, most simplistic, and ultimately has the greatest efficiency. Developed by Jennifer Doudna and Emmanuelle Charpentier, CRISPR made headlines in 2012 due to its possibilities in editing the human genome and was deemed as one of the most significant discoveries in the history of biology. As more companies enter the gene therapy space, CRISPR specifically has been a focal point of discussion and has progressed remarkably over the last few years.

Majory Developments

  • In January of 2018, China announced they had already edited the genes of 86 people and currently had 11 clinical trials using CRISPR. By the time the news hit, China already had removed cells from the bodies of 36 people battling cancers of the lungs, kidney, liver, and throat, altered these cells with CRISPR, and then injected the cells back into their bodies to fight the patients’ respective cancers. CRISPR related stocks enjoyed a jolt in their stock prices on this news, with one company’s stock, Crisper Therapeutics (NASDAQ: CRSP), doubling by the end of the month
  • CRISPR has been affiliated with the possible cure of many diseases including cancer, blood disorders, AIDS, blindness, Huntington’s disease, cystic fibrosis, and muscular dystrophy
  • Pharmaceutical companies Allergan, Novartis, Regeneron, Vertex, and Bayar AG have all either invested in the CRISPR space or formed strategic partnerships with current CRISPR companies and both Google Ventures and Bill Gates’ former chief scientific advisor, Boris Nikolic, has made prominent investments as well
  • In an event that caused worldwide controversy during the fall of 2018, Chinese scientist He Jiankui successfully created the world’s first genetically modified twin babies with CRISPR technology. Though Mr. Jiankui is facing punishment from the Chinese government, the procedure proved successful as he was able to completely disable a gene called CCR5 which is responsible for the HIV virus

The Future

Gene therapy is a field of science that has enamored our imaginations for some time. It seems like half the science fiction stories where experiments go horrendously wrong or astonishingly well have some mention of genetic editing. Yet this remarkable advancement in health technology is here, happening right now and even evolving. The market for gene therapy is projected to reach $12 billion by 2020.

As scientists continue to challenge the status quo and innovate in the field of gene therapy, society itself is set to reap the rewards. Despite the fact we are just beginning to tap into the possibilities of gene-based therapies and some, like CRISPR, are largely still unproven, the fact remains that the potential is there. Whether curing genetic diseases or instilling specific genetic traits into the next generation of human beings, gene-based therapies including CRISPR technology should play a major role in the future of healthcare.

CRISPR Stocks

Industry: Biotechnology

Sector: Healthcare

Website: http://www.crisprtx.com

Description: CRISPR Therapeutics AG, a gene editing company, focuses on developing transformative gene-based medicines for serious human diseases. It develops its products using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), a gene editing technology that allows for precise directed changes to genomic DNA. It has a portfolio of therapeutic programs in a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The company’s lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-dependent beta thalassemia or severe sickle cell disease in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. It is also developing CTX110, a donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19 positive malignancies. In addition, the company is developing allogeneic CAR-T programs comprising CTX120 targeting B-cell maturation antigen for the treatment of multiple myeloma; and CTX130 for the treatment of solid tumors and hematologic malignancies. Further, it engages in developing regenerative medicine programs in diabetes; and in vivo and other genetic disease programs to treat glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. The company has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland.

Fundamentals
Market cap$9.02B
P/E ratio
Earnings per share$ -5.29
Return on equity-26.80%
Profit margin0.00%
Total revenue$719.00K
Debt / Equity3.69
Price / Book5.31
52 week high$220.20
52 week low$42.83
Shares outstanding$75.13M
Previous close$118.53
Dividend Analysis
Dividend yield0.00%
Payout ratio0.00%
Trailing Annual Dividend Rate$
Five Year Avg Dividend Yield0.00%
Ex-Dividend date0

Industry: Biotechnology

Sector: Healthcare

Website: http://www.intelliatx.com

Description: Intellia Therapeutics, Inc., a genome editing company, focuses on the development of therapeutics. It utilizes a biological tool known as the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. The company develops in vivo programs focusing on liver diseases, including transthyretin amyloidosis and hereditary angioedema, as well as other research programs comprising primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Its ex vivo pipeline includes proprietary programs focused on developing engineered cell therapies to treat various oncological and autoimmune diseases; partnered programs focused on chimeric antigen receptor T cells and hematopoietic stem cells; and acute myeloid leukemia. Intellia Therapeutics, Inc. has license and collaboration agreements with Novartis Institutes for BioMedical Research, Inc.; Regeneron Pharmaceuticals, Inc.; Ospedale San Raffaele; and GEMoaB Monoconals GmbH. The company was formerly known as AZRN, Inc. and changed its name to Intellia Therapeutics, Inc. in July 2014. Intellia Therapeutics, Inc. was founded in 2014 and is headquartered in Cambridge, Massachusetts.

Fundamentals
Market cap$4.93B
P/E ratio
Earnings per share$ -2.40
Return on equity-33.69%
Profit margin-231.46%
Total revenue$57.99M
Debt / Equity7.46
Price / Book9.14
52 week high$92.00
52 week low$12.19
Shares outstanding$67.73M
Previous close$71.09
Dividend Analysis
Dividend yield0.00%
Payout ratio0.00%
Trailing Annual Dividend Rate$
Five Year Avg Dividend Yield0.00%
Ex-Dividend date0

Industry: Biotechnology

Sector: Healthcare

Website: http://www.editasmedicine.com

Description: Editas Medicine, Inc. operates as a clinical stage genome editing company. The company focuses on developing transformative genomic medicines to treat a range of serious diseases. The company develops a proprietary genome editing platform based on CRISPR technology, which includes CRISPR/Cas9, CRISPR/Cas12a, and engineered forms of both of these CRISPR systems to target genetically addressable diseases and therapeutic areas. It also develops EDIT-101, which is in Phase 1/2 clinical trial for the treatment of Leber Congenital Amaurosis type 10, a genetic form of vision loss that leads to blindness in childhood. In addition, the company develops EDIT-102 for the treatment of Usher Syndrome 2A, which is a form of retinitis pigmentosa that also includes hearing loss; autosomal dominant retinitis pigmentosa 4, a progressive form of retinal degeneration; and EDIT-301 treat sickle cell disease and beta-thalassemia. It has a research collaboration with Juno Therapeutics, Inc. to develop engineered T cells for cancer; strategic alliance and option agreement with Allergan Pharmaceuticals International Limited to discover, develop, and commercialize new gene editing medicines for a range of ocular disorders; strategic research collaboration Asklepios BioPharmaceutical, Inc. to develop a therapy to treat a neurological disease; and research collaboration with Sandhill Therapeutics, Inc. to develop allogeneic healthy donor derived NK cell medicines for the treatment of solid tumors. The company was formerly known as Gengine, Inc. and changed its name to Editas Medicine, Inc. in November 2013. Editas Medicine, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.

Fundamentals
Market cap$2.78B
P/E ratio
Earnings per share$ -1.98
Return on equity-35.36%
Profit margin-127.82%
Total revenue$90.73M
Debt / Equity6.64
Price / Book6.55
52 week high$99.95
52 week low$20.98
Shares outstanding$67.36M
Previous close$40.40
Dividend Analysis
Dividend yield0.00%
Payout ratio0.00%
Trailing Annual Dividend Rate$
Five Year Avg Dividend Yield0.00%
Ex-Dividend date0

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