John Leonard constructed Intellia Therapeutics with Jennifer Doudna, the Nobel Prize-winning scientist who pioneered gene modifying know-how. The corporate has discovered the right way to alter disease-causing genes inside sufferers, however earlier than any breakthrough therapies come, it should first remedy itself of its authorized and monetary ills.
John Leonard, CEO of Intellia Therapeutics, has simply completed an hour-long assembly within the firm’s Boston headquarters, which is a five-minute jog from the MIT campus. He stretches his arms, interlocks his fingers and rests them behind his head. For a second, the 64-year-old is relaxed. However he instantly reanimates when explaining the science of DNA modifying, excitedly utilizing markers, a duster and no matter else he can discover to make his level. “I’ve used my spouse’s necklaces now and again,” he says. Leonard suggests imagining human DNA as a necklace made of three billion beads and 4 completely different colours. “The problem 54is how do you discover 20 beads to the exclusion of all the things else,” he provides. The beads he’s referring to are genes, sections of DNA which give cells the directions they should do their work.
John Leonard
TEN BRIDGE COMMUNICATIONS
Many corporations use Crispr, a revolutionary technique of exactly modifying DNA which was the idea for the Nobel Prize in Chemistry in 2020, to snip disease-causing genes within the lab after which inject the “fastened” cells again into sufferers. Intellia does that too. However it’s its different gene modifying platform which has grabbed eyeballs at Wall Road. The $3.6 billion market cap firm has discovered the right way to use Cripsr exterior of the lab, inside a residing human. Their work may have main implications in creating new medicine for genetic ailments that at present solely have restricted or no therapies. “Intellia is the primary to do in vivo genome modifying in a systemic method. I feel that is the actual differentiating issue for me,” says Jack Allen, senior analyst at Baird Fairness Analysis.
Regardless of the novelty of its gene modifying know-how, the corporate is going through important headwinds. Within the final 12-months it misplaced $277 million on revenues of $33 million. Revenues have been declining on a quarterly foundation since 2020 whereas losses have been widening. The corporate has raised a complete of $1.8 billion, together with $115 million when it when public in 2016, they usually nonetheless have $1 billion in money available. However at current burn charges, that may solely a few years. Intellia does has one promising drug in early-stage medical trials, however round 90% of therapies at this stage fail to succeed in the market. After which there’s the patent battle over their core know-how.
It sounds fairly grim, and Intellia’s inventory has taken a beating. For the reason that begin of the 12 months, Intellia shares are 62% down in comparison with 23% for the Nasdaq and 24% for Nasdaq’s biotech index. Nonetheless Intellia has not less than one ace within the gap: The person who runs the corporate is not any stranger to those challenges. Leonard, a medical physician by coaching, has a monitor document which few others can level to on this business. In 1992, Leonard joined Abbott the place his workforce’s analysis earned an FDA approval for HIV drug, Norvir and Kaletra, which helped curtail the AIDS epidemic within the nineties. And in 2013 he joined Abbvie, Abbott’s biopharmaceutical spin off, the place he was instrumental within the improvement of Humira, which had gross sales of $21 billion final 12 months, making it one of many world’s best-selling medicine.
“I labored on Humira for 13 years,” Leonard says. “I realized quite a lot of rules about a company. What makes it work? What makes it not work typically.”
His experiences of getting these blockbuster medicine from lab bench to the market may assist flip Intellia’s liver drug NTLA-2001, which it’s co-developing together with Regeneron Prescription drugs, into a hit. The drug is an injectable gene modifying remedy for the therapy of ATTR amyloidosis- a uncommon genetic situation of the liver which impacts 1 in 100,000 Individuals and kills round 850 of them a 12 months.
The marketplace for treating this illness was $585 million in 2019. However extra individuals in all probability have the illness and should not being correctly recognized. Higher diagnoses could lead on this to turn into a $14.1 billion market inside 7 years, in accordance with a report by London-based consultancy GlobalData. There are at present three FDA authorised medicine which gradual the progress of the illness, however none of them are everlasting cures and sufferers typically find yourself nonetheless needing liver transplants. In February, Intellia launched early information from its medical trials exhibiting a sustained, constructive impact on individuals, with out worrying negative effects.
Regardless of promising information, Intellia’s highway head isn’t bump free. The corporate licenses the Crispr know-how which it makes use of to carry out in vivo gene edits from the College of California, the College of Vienna, and pathogen researcher Emmanuel Charpentier (collectively referred to as the CVC group). College of California biochemist Jennifer Doudna who received the Nobel together with Charpentier for locating the Crispr modifying system is an Intellia cofounder, though she has restricted day-to-day duties.
These patents held by the CVC group are the place the authorized bother is available in. Patents obtained by the CVC group are at odds with others owned by the Broad Institute, the powerhouse medical analysis middle began by the late billionaire Eli Broad and affiliated with each Harvard and MIT. That spawned a authorized battle, beginning in 2016, with tens of thousands and thousands of {dollars} in royalties at stake, about who was first to invent the Crispr gene modifying software utilized in human and plant cells. In in over 80 international locations together with China, Japan and the 27 nations within the European Union, it has been held that Doudna’s CVC group invented it first. However within the U.S., a latest verdict by the Patent Trial and Enchantment Board (PTAB) dominated in favor of the Broad Institute. The CVC group is interesting.
“Fortuitously, this ruling doesn’t affect the event of CRISPR in anyway,” says Doudna. “Buyers are persevering with to place cash into the house,” she provides.
After all, even when Intellia loses in court docket, they are going to nonetheless be capable of license the know-how. “Anybody who doesn’t have a license from the Broad Institute and that’s conducting work with [Crispr] might be going to must get one in some unspecified time in the future. I’d think about that that would come with Intellia,” says Jacob Sherkow, a professor of regulation on the College of Illinois.
“Fortuitously, this (PTAB) ruling doesn’t affect the event of CRISPR in anyway. Buyers are persevering with to place cash into the house.”
Leonard is trying past the mental property battle. His focus at present is on increasing the corporate’s improvement pipeline to incorporate therapies for a lot of extra ailments, akin to hereditary angioedema, hemophilia, blood and ovarian cancers. However first, Leonard should clear up the patent mess and lift more cash. Given the promise of Intellia’s know-how, he’s optimistic.
“I feel when individuals make judgments about the place to place their money to work, they take a look at the actual risk of [drug development] applications making it to {the marketplace}. We’re undoubtedly in that class,” Leonard says. “So we expect we’re effectively positioned to proceed the funding of the corporate as we go ahead.”
He’s much more optimistic about the way forward for Crispr-based medicine, which have the potential to relegate a complete host lethal ailments to the historical past books. “Within the coming years we might be restricted not by know-how however by creativeness.”
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