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)