Intellia’s gene editing breakthrough is historic

Intellia’s gene editing breakthrough is historic by Saxo Group
Intellia’s breakthrough result on its gene editing technology could be a game changer for the future of disease treatments and our NextGen Medicine theme basket.

Back in January we launched our NextGen Medicine theme basket consisting of 30 companies within what we call the frontier of biotechnology. There is the mature biotechnology industry, which is closer to the old pharmaceutical industry, and then there is this new frontier of biotechnology companies working antibody therapies, gene editing, T-cell therapy, immunology, mRNA technology and genetic diagnostics.

Over the weekend, Intellia Therapeutics and Regeneron Pharmaceuticals (part of the mature biotechnology industry) released a landmark phase 1 clinical data showing significant reduction in disease-causing protein following CRISPR therapy for Transthyretin (ATTR) Amyloidosis. Experts have called this a major breakthrough for the promising gene editing technology CRISPR, pioneered by the two female scientists Charpentier and Doudna which won them the Nobel Prize in Chemistry, which is necessary to increase the potential of adoptive cell therapy (ACT). The news lifted Intellia’s share price by 50% yesterday and our NextGen Medicine basket by 3.9% showing how Intellia’s result is lifting the outlook for many of these new emerging biotechnology companies within these new technologies.

The table below shows the 30 companies in our NextGen Medicine basket and provide a broad-based exposure to this part of the biotechnology industry. It is our expectation that the biggest breakthroughs in medicine over the coming decades will come from this part of industry. Some of the other gene editing companies in the list are CRISPR Therapeutics, Beam Therapeutics, Editas Medicine and PTC Therapeutics.

Name Domicile Mkt cap in USD mn R&D in % 12M Rev in USD mn Burn rate Insti %
Illumina Inc US 70,299 1.0 3,473 NM 99.7
Moderna Inc US 89,517 1.9 2,732 NM 58.3
Seagen Inc US 29,236 2.9 2,273 NM 106.4
Genmab A/S DK 27,338 1.9 1,693 NM 64.6
Exact Sciences Corp US 22,475 2.8 1,546 NM 102.3
BioNTech SE DE 54,547 1.4 551 16.3 NA
Grifols SA ES 16,167 2.2 6,105 NM 62.9
CRISPR Therapeutics AG (**) CH 11,489 2.6 1 5.5 70.5
Argenx SE NL 16,177 1.6 42 5.1 59.8
Mirati Therapeutics Inc US 8,861 3.7 13 4.2 114.9
Fate Therapeutics Inc US 8,380 1.5 40 10.2 110.2
Natera Inc US 10,257 1.2 449 3.0 115.1
Invitae Corp US 6,888 3.9 319 1.0 101.3
Ultragenyx Pharmaceutical Inc US 6,505 6.9 334 2.9 132.2
Twist Bioscience Corp US 6,177 0.8 113 2.5 126.1
Denali Therapeutics Inc US 9,095 2.4 340 NM 102.1
Arrowhead Pharmaceuticals Inc US 8,974 1.8 89 NM 74.1
Iovance Biotherapeutics Inc US 4,116 4.9 0 2.4 127.3
Pacific Biosciences of California Inc US 7,010 1.0 92 4.0 91.8
Galapagos NV BE 4,666 13.4 639 10.3 45.6
Beam Therapeutics Inc US 6,812 4.0 0 2.3 106.8
Swedish Orphan Biovitrum AB SE 5,533 3.5 1,612 NM 107.9
Sage Therapeutics Inc US 3,345 8.6 1,113 NM 119.8
Abcam PLC GB 4,485 1.2 346 NM 89.3
Intellia Therapeutics Inc US 9,094 1.7 52 8.7 109.3
Editas Medicine Inc US 2,988 5.5 92 2.2 87.1
PTC Therapeutics Inc US 3,067 17.0 430 4.8 124.2
Allogene Therapeutics Inc US 3,616 5.7 38 4.5 125.7
CareDx Inc US 4,951 1.1 221 59.1 110.5
MorphoSys AG DE 2,662 6.7 144 5.3 75.2

Source: Bloomberg and Saxo Group

Table explanations: R&D in % is the 12-month R&D expense in % of market cap, Burn rate is the number of years the company can sustain current negative free cash flow (NM means not measurable and is used for those with positive free cash flow), Insti % is the institutional ownership of the floating shares (those that are tradeable, so if all shares are not publicly available then the ratio can be above 100%) in %.

** Peter Garnry has holdings in these companies

Topics: Equities Health Care Technology United States

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