Biotech – gene therapy stocks

Genes are part of the DNA that provides the blueprint instructions to the cells for making proteins. A mutation or a defect in the gene can prevent the cells from creating an accurate expression of the protein or a diminished ability to do so. Gene therapy is the science of altering DNA to restore normal protein expression by the cells or even silence proteins that may cause debilitating diseases.

• Significant progress has been made in improving the understanding of using different delivery vehicles or vectors to safely deliver the therapeutic DNA altering payload. For example, CRISPR, which makes addition and deletion edits directly to the DNA strand, is a popular editing tool in gene therapy.
• The first gene therapy approved in Western Europe, UniQure's Glybera, turned out to be a flop. The company withdrew it from the market in 2017 and is shifting its focus to hemophilia treatments.

ETFs:

The Nasdaq Biotech (IBB) and the S&P Biotech (XBI) indexes

(by market cap)                        
Company name Mkt Cap▼

1. VRTX     Vertex Pharmaceuticals   73.83B; Massachusetts
2. ALNY     Alnylam Pharmaceuticals     23.28B; Massachusetts
3. BMRN   BioMarin Pharmaceutical     15.90B; California
4. SRPT    Sarepta Therapeutics        9.79B Massachusetts
5. IONS     Ionis Pharmaceuticals        6.18B; California
6. CRSP     CRISPR Therapeutics AG     4.09B; Switzerland
7. NTLA     Intellia Therapeutics        3.96B; Massachusetts
8. BEAM     Beam Therapeutics       3.26B; Massachusetts
9. PTCT     PTC Therapeutics        3.19B; New Jersey
10. RARE     Ultragenyx Pharmaceutical     2.76B; California
11. VERV     Verve Therapeutics        2.16B; Massachusetts
12. RGNX      REGENXBIO            981.75M; Maryland
13. QURE     uniQure N.V.           846.73M; Netherlands
14. EDIT     Editas Medicine           823.47M; Massachusetts
15. SGMO     Sangamo Therapeutics    673.18M; California
16. BLUE     bluebird bio        463.50M; Massachusetts
17. MGTX     MeiraGTx            338.48M; New York
18. SELB     Selecta Biosciences  248.92M; Massachusetts
19. VYGR      Voyager Therapeutics    198.26M; Massachusetts
20. ADVM     Adverum Biotechnologies  91.35M; California
21. FIXX    Homology Medicines     85.53M; Massachusetts
22. SLDB     Solid Biosciences 51.29M; Massachusetts
23. AVRO     AVROBIO  29.40M; Massachusetts
24. AGTC  Applied Genetic Technologies  27.66M; Florida
25. ABEO  Abeona Therapeutics  25.76M; New York
26. SIOX  Sio Gene Therapies  22.04M; New York; formerly known as Axovant Gene Therapies (NASDAQ: AXGT)

Acquisitions

• Alexion Pharma (ALXN) acquired by AstraZeneca (AZN) for $175 per share. (Dec 2020)
• Prevail Therapeutics (PRVL) acquired by Lilly (LLY) for $22.50 per share or ~$880 mln.  (Dec 2020)
• Audentes Therapeutics (BOLD) acquired by Astellas (ALPMY) for $60/share. (December 3, 2019)
• Nightstar Therapeutics (NITE) acquired by Biogen (BIIB) for $800 mln. (March 2019)
• Spark Therapeutics (ONCE) acquired by Roche (RHHBY) for $4.8 bln. (February 2019)
• Celgene (CELG) acquired by Bristol-Myers Squibb (BMY) for $102.43/share or approx. $74 billion.  (January 2019)
• TESARO (TSRO) acquired by GlaxoSmithKline (GSK) for $5.1B. (January 2019)
• AveXis (AVXS) acquired by Novartis (NVS) for $8.7 bln.  (April 9, 2018)
• Wilson Therapeutics acquired by Alexion Pharma (ALXN) for $855 Million. (April 2018)
• Kite Pharma (KITE) acquired by Gilead Sciences (GILD) for approximately $11.9 billion (October 2017)

©Artremis / EAS (10/27/22)

Biotech - gene therapy

The concept of gene therapy, which originated in the late 1960s, has come a long way - having had its own share of significant setbacks and gargantuan success.

Gene therapy is a technique that uses genes to treat or prevent diseases caused by defective or missing genes. This approach helps to address the underlying cause of the disease, rather than offering solutions that focus only on the disease symptoms.

CRISPR the technology — not to be confused with Crispr Therapeutics, the company — builds on a project that sequenced the human genome. The first map cost $2.7 billion and was completed in 2003.  Since then, the cost to map an individual's genome has dropped precipitously and could come down to just hundreds of dollars in the next few years.

The world's first gene therapy, Gendicine, was approved in China in 2004. Developed by SiBiono GeneTech Co., Ltd., Gendicine is indicated for head and neck squamous cell carcinoma. A year later, i.e., in 2005, another gene therapy product - Oncorine - was launched in China, by Shanghai Sunway Biotech Co. Ltd., for the treatment of head and neck cancer.

Russia launched its first gene therapy, Neovasculgen, to treat Peripheral Arterial Disease in 2011.

The first gene therapy approved by the European Commission was Glybera. Developed by uniQure N.V. (QURE), Glybera was approved in October 2012 for adult patients diagnosed with familial lipoprotein lipase deficiency and was launched in early 2015. However, the drug was withdrawn from the European Union in 2017 due to commercial failure. The second gene therapy to be greenlighted in the EU is Strimvelis, which was approved in 2016, for a rare disease called severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, dubbed "Bubble Boy" disease.

 

In the U.S., the first-ever gene therapy to win the regulatory nod is Novartis' (NVS) Kymriah. Approved by the FDA on August 30, 2017, Kymriah is indicated for Acute Lymphoblastic Leukemia, and Large B-Cell Lymphoma.

2017 saw two more gene therapies get the FDA nod - Kite/Gilead's (GILD) Yescarta, approved in October 2017, for the treatment of adults with diffuse large B-cell lymphoma, and Spark Therapeutics' Luxturna, approved in December 2017, to treat children and adult patients with an inherited form of vision loss that may result in blindness.