Gene Therapy Stocks 2019

Gene Therapy Stocks 2019

Why has gene therapy become so significant within the last five years? While it is not well-known, the genes we are born with can be flawed and there are >7000 genetic diseases in the world today. These genetic diseases result in the loss of critical enzymes or proteins in our bodies. Loss of normal proteins and enzymes can lead to disease conditions that range from mild to deadly.

Although these diseases are considered rare, one-in-ten people have a rare disease. The total cost of treatment is between $2 Trillion and $4 Trillion per year; or as much as 40% of global healthcare spending. Gene therapy provides an alternative to lifetime palliative treatment of these rare diseases. Gene therapy gives us the ability to replace or edit defective genes to relieve the underlying cause of these conditions.

Gene therapy public companies have been gaining ground, raising funds, and merging with larger companies. The race to purchase gene therapy developers is intensifying by Pharma companies. Over the past two years, some notable acquisitions include the following:

  • Biogen acquired Nightstar Therapeutics for a transaction value of $800 Million (March 2019 press release)
  • Roche acquired Spark Therapeutics for a total transaction value of approximately $4.3 Billion (February 2019 press release)
  • Bristol-Myers Squibb acquired Celgene for approximately $74 billion (January 2019 press release)
  • GlaxoSmithKline (GSK) acquired TESARO (TSRO) for $5.1B (January 2019 press release)
  • Novartis acquired AveXis Inc (AVXS) for $8.7 Billion cash (April 2018 press release)
  • Alexion Pharmaceuticals, Inc., acquired Wilson Therapeutics for $855 Million (April 2018 press release)
  • Novartis licensed Spark Therapeutics Luxterna Gene Therapy in all markets outside the U.S. (January 2018 press release)
  • Celgene acquired Juno Therapeutics for $9 Billion (January 2018 press release)
  • Sanofi acquired BioVerativ for 11.6 Billion (January 2018 press release)
  • Gilead acquired Kite Pharma for approximately $11.9 Billion (October 2017 press release)

These acquisitions are gaining the attention of investors. For a list of the public gene therapy stocks currently being traded in the marketplace, see below:

Abeona Therapeutics, Inc.NASDAQ:ABEOSanfilippo syndrome types A and BPublic
Applied Genetic Technologies CorporationNASDAQ:AGTCDiseases caused by broken genesPublic
Arcturus Therapeutics Ltd.NASDAQ:ARCTTreatment of liver and respiratory diseasesPublic
Audentes TherapeuticsNASDAQ:BOLDRare muscle diseasesPublic
AvrobioNASDAQ:AVROCancer and rare diseasesPublic
BioMarinNASDAQ:BMRNDevelops and commercializes innovative biopharmaceuticals for serious diseases and medical conditionsPublic
Bluebird BioNASDAQ:BLUEGenetic and orphan diseasesPublic
BridgeBioNASDAQ:BBIOGenetic DiseasesPrivate but set to go Public
CRISPR TherapeuticsNASDAQ:CRSPSickle cell diseasePublic
Editas Medicine, Inc.NASDAQ:EDITTreat the underlying cause of a broad range of diseases at the genetic levelPublic
Genprex, Inc.NASDAQ:GNPXCancerPublic
GenSight BiologicsEPA:SIGHTRetinal degenerative diseasesPublic
Krystal Biotech, Inc.NASDAQ:KRYSRare debilitating disordersPublic
LogicBio Therapeutics, Inc.NASDAQ:LOGCHIV/AIDS and methylmalonic acidemiaPublic
LYSOGENEEPA:LYSSevere genetic pathologies with central nervous system involvement and high unmet medical needsPublic
Neurologix, Inc.OTCPINK:NRGXQDisorders of the brain and central nervous systemPublic
Nightstar TherapeuticsNASDAQ:NITERare inherited retinal diseasesPublic
Orchard TherapeuticsNASDAQ:ORTXMetachromatic leukodystrophy, Wiskott Aldrich syndrome, X-linked chronic granulomatous diseasePublic
Oxford BioMedicaLON:OXBLentiviral vector and cell therapy researchPublic
REGENXBIO Inc.NASDAQ:RGNXMetabolic disorders, muscle diseases, hematologic disorders, and ocular diseasesPublic
RocheSWX:ROGCancer and transplantationPublic
Solid BiosciencesNASDAQ:SLDBDuchenne muscular dystrophyPublic
Spark TherapeuticsNASDAQ:ONCEOne-time gene therapy products for debilitating diseasesPublic
TocagenNASDAQ:TOCATreating a primary tumors and metastatic diseasesPublic
uniQureNASDAQ:QUREGenetic or acquired diseasesPublic
Vical Inc.NASDAQ:VICLDNA vaccines and non-viral gene therapyPublic
Voyager TherapeuticsNASDAQ:VYGRNeurological diseasesPublic

In the last 5 years, there has been a significant surge in private gene therapy companies becoming publicly traded. Some examples of companies reportedly considering going public are as follows:

  • BridgeBio expects to go public with an offer of $225 Million, after a $299.2 Million financing round in January 2019. It will use the IPO proceeds to fuel its research. They opted for a NASDAQ symbol “BBIO” (March 2019 MedCityNews Article)
  • Prevail Therapeutics is eyeing a $100 million IPO and plans to use the IPO proceeds to fuel its Phase 1/2 study of its lead candidate. It has filed an IND to the FDA to begin clinical trials and has opted for a NASDAQ symbol “PRVL” (March 2019 Xconomy Article)
  • Atreca will be submitting an FDA IND application to test its lead experimental drug by the end of 2019. Following its $125 million third round of private fundraising, the preclinical biotech company aims for a $100 million public offering debut. If successful, Atreca is planning to list under the symbol “BCEL” (March 2019 EndPoints Article)

Why are private gene therapy companies moving toward filing an Initial Public Offering (IPO)? Some wish to ‘strike while the iron's hot.’ The IPO window is currently open. With gene therapies getting FDA approval, and with the attractive returns on valuation that come with these approvals, investments in developing gene therapy companies are attractive.

There are many considerations for a private company to evaluate before deciding to go public. For instance, research and development require a lot of funding. Is the funding available to the company from private sources? If not, public markets are well-suited to supply the needed capital. Or, early-stage investors may desire the liquidity offered by a publicly listed company. For companies, the decision to “go public” is not done lightly.

Yet, the above transactions and the breakthroughs made in gene therapy have made the last five years significant for the industry. The stage is set for the next five years to be incredible!

Resources, Articles, and Links to Company Sites:

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