Intellia’s gene editing breakthrough is historic

Equities 5 minutes to read
Peter Garnry

Head of Equity Strategy

Summary:  Intellia Therapeutics' share price rose 50% yesterday as the biotechnology company showed promising results against a rare genetic liver disease. The gene editing technology is one of the most promising technology within disease treatments and the key to unlock the full potential of adaptive cell therapies.


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.

Source: Saxo Group

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.

NameDomicileMkt cap in USD mnR&D in %12M Rev in USD mnBurn rateInsti %
Illumina IncUS70,2991.03,473NM99.7
Moderna IncUS89,5171.92,732NM58.3
Seagen IncUS29,2362.92,273NM106.4
Genmab A/SDK27,3381.91,693NM64.6
Exact Sciences CorpUS22,4752.81,546NM102.3
BioNTech SEDE54,5471.455116.3NA
Grifols SAES16,1672.26,105NM62.9
CRISPR Therapeutics AG (**)CH11,4892.615.570.5
Argenx SENL16,1771.6425.159.8
Mirati Therapeutics IncUS8,8613.7134.2114.9
Fate Therapeutics IncUS8,3801.54010.2110.2
Natera IncUS10,2571.24493.0115.1
Invitae CorpUS6,8883.93191.0101.3
Ultragenyx Pharmaceutical IncUS6,5056.93342.9132.2
Twist Bioscience CorpUS6,1770.81132.5126.1
Denali Therapeutics IncUS9,0952.4340NM102.1
Arrowhead Pharmaceuticals IncUS8,9741.889NM74.1
Iovance Biotherapeutics IncUS4,1164.902.4127.3
Pacific Biosciences of California IncUS7,0101.0924.091.8
Galapagos NVBE4,66613.463910.345.6
Beam Therapeutics IncUS6,8124.002.3106.8
Swedish Orphan Biovitrum ABSE5,5333.51,612NM107.9
Sage Therapeutics IncUS3,3458.61,113NM119.8
Abcam PLCGB4,4851.2346NM89.3
Intellia Therapeutics IncUS9,0941.7528.7109.3
Editas Medicine IncUS2,9885.5922.287.1
PTC Therapeutics IncUS3,06717.04304.8124.2
Allogene Therapeutics IncUS3,6165.7384.5125.7
CareDx IncUS4,9511.122159.1110.5
MorphoSys AGDE2,6626.71445.375.2

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


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