Saturday, November 14, 2020

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

SGMO +8.51%, VYGR +5.68%, AXGT +4.91%, RGNX +4.47%, FIXX +3.85%, SLDB +3.70%, AVRO +3.38%, ADVM +2.49%, BMRN +2.30%, RARE +2.20%, SRPT +2.04%, 
uniQure (QURE +15%)

Editas (EDIT), Crispr (CRSP), Solid Biosciences (SLDB), Spark Therapeutics (ONCE), Homology Medicines (FIXX), Sarepta Therapeutics (SRPT), Bluebird (BLUE), Regenxbio (RGNX), PTC Therapeutics (PTCT), Meiragtx (MGTX), and Intella Therapeutics (NTLA).

AVXS +81.23%, BOLD +9.57%, ADVM +7.25%, ABEO +7.14%, CRSP +5.27%, NTLA +5.04%, SGMO +4.42%, BLUE +4.39%, VYGR +3.34%, EDIT+3.01%, IONS +1.77%, ONCE +1.60%, ALNY +1.13%; other gene therapy stocks: QURE, SELB, AGTC, NITE

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)

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