Need to keep on high of the science and politics driving biotech at the moment? Join to get our biotech publication in your inbox.
Howdy! Right now we study whether or not AI would possibly assist hasten the event of n-of-1 therapies (trace: not but), discover why the FDA hasn’t supplied Orange E book steering on drug-device combos, and speak about CRISPR. Lots.
The necessity-to-know this morning
- AC Immune has sold global licensing rights to an experimental immunotherapy for Alzheimer’s illness to the Japanese drugmaker Takeda. AC Immune obtained $100 million from Takeda for the remedy, known as ACI-24.060, and is eligible for one more $2.1 billion in future funds.
- Merck reported negative results from an interim evaluation of a Part 3 examine involving the usage of its experimental anti-TIGIT antibody as an adjuvant therapy for sufferers with high-risk melanoma.
- Most cancers drug developer OnKure goes public by way of a reverse merger with Reneo Pharma.
AI may not but assist develop ‘n-of-1’ therapies
Can AI be used to assist create “n-of-1” therapies for the rarest of genetic ailments? The jury’s nonetheless out, a brand new examine suggests.
There’s been a wave of antisense oligonucleotide medicines designed particularly to focus on the only mutation inflicting a affected person’s signs. It is a compelling however time- and cost-prohibitive strategy for many corporations, which has led some, like Creyon Bio, to show to synthetic intelligence as a option to pace alongside the method.
Final week, Creyon stated it efficiently dosed a affected person with a customized therapy that confirmed some indicators of efficacy. What’s much less clear, nevertheless, is whether or not machine studying helped make the drug sooner or safer, STAT’s Jason Mast writes. Creyon stated initially it could use AI to create the brand new drugs inside seven months; as a substitute, it took simply over a yr, which is not any higher than different n-of-1 approaches. Milasen, the primary bespoke drug, took 10 months to develop. And Creyon’s drug didn’t price “appreciably much less” than different n-of-1 medicines, which run over $1 million to create.
Learn extra.
The FDA’s hands-off stance with drug-device combos
Drugmakers have lengthy requested the FDA for clearer tips on easy methods to listing patents in a particular registry known as the Orange E book. That is essential as a result of it helps branded drugmakers inform generic opponents about their mental property. Nevertheless, regulators haven’t supplied the required steering for drug-and-device mixture merchandise like inhalers and auto-injectors, STAT’s Ed Silverman writes. That is irritating for business gamers and affected person advocates alike.
Just lately, the FTC has stepped in, warning corporations about improper patent listings within the Orange E book and taking authorized motion in opposition to people who try to prolong their monopolies.
“The FDA wants to point out some management and say which stuff gained’t go within the Orange E book, however as a substitute, they’ve been flip-flopping for years,” the chief of a nonprofit that focuses on pharmaceutical patent points advised STAT. “There may be simply an excessive amount of uncertainty that hasn’t been cleared up.”
Learn extra.
We now know Tome’s gene enhancing goal
From STAT’s Jason Mast: For all of the hype surrounding CRISPR, the instrument has been largely efficient at knocking out genes or making small edits. So when two scientists emerged a pair years in the past touting technology able to inserting giant genes into human cells with out doing main injury to the DNA, traders agreed to pour over $200 million to attempt to make it a actuality.
That spherical, for Tome Biosciences, was introduced in December. On Friday, at a significant gene remedy convention, CEO Rahul Kakkar laid out for the primary time how the corporate plans to make use of the know-how: initially to deal with a uncommon illness known as phenylketonuria and to design a pure killer cell remedy closely engineered to soundly deal with autoimmune ailments. Past which can be plans to make a coronary heart illness therapy and coverings for uncommon ailments homocystinuria and hemochromatosis.
The corporate will, after all, have competitors. Tessera has additionally showed animal knowledge for treating phenylketonuria with a gene enhancing system for altering particular person letters. And Prime Medication has a really comparable know-how for big gene insertion, although it has but to disclose a lot element on particular ailments.
A CRISPR strategy didn’t treatment HIV
An effort to make use of CRISPR enhancing to excise HIV from affected person genomes didn’t work. A Part 1 trial from Excision Therapeutics administered a CRISPR-based remedy to 5 sufferers with HIV. Researchers then took three of those sufferers off of standard antiviral therapy, with hopes that they wouldn’t have to resume every day treatment. However regardless of the CRISPR therapy, virus ranges rebounded shortly.
That is probably as a result of the gene enhancing strategy wasn’t environment friendly sufficient, STAT’s Jason Mast writes. Excision used an adeno-associated virus to distribute the gene enhancing instrument all through the physique — and AAVs don’t attain all cells. So even when the virus was excised from a part of the physique, it’s probably it lingered elsewhere.
However there have been indicators of hope: One affected person’s an infection didn’t rebound for about 4 months after stopping antivirals, whereas sometimes it comes again after three or 4 weeks. This means that CRISPR enhancing would possibly in the end be coupled with different approaches to treatment HIV.
Learn extra.
Maze will get one other probability to companion on Pompe drug
Final yr, Sanofi was set to pay $150 million in money and inventory to license an experimental Pompe illness therapy made by Maze Therapeutics — however the FTC blocked the deal and it fell via. Now, Maze has partnered with Japanese drugmaker Shionogi to additional develop MZE001, once more with $150 million up entrance, STAT’s Andrew Joseph writes.
Sanofi already sells two intravenous infusions for Pompe, which prompted concern for regulators, who stated an acquisition would prolong Sanofi’s monopoly within the illness and dissuade opponents from getting into the area. The French drugmaker walked away from the deal with out personally informing the corporate: CEO Jason Coloma advised STAT final yr that he was “personally disillusioned” within the Sanofi deal falling via.
“Shionogi has a monitor document of creating and delivering revolutionary medicines to sufferers worldwide, and we’re assured they’re the appropriate companion to proceed to advance MZE001 via scientific trials in order that it might attain sufferers with this life-threatening situation as quickly as potential,” Coloma stated in a press release.
Learn extra.
Extra reads
- Pfizer and AstraZeneca announce new investments of practically $1 billion in France, Reuters
- First particular person to obtain a genetically modified pig kidney transplant dies practically 2 months later, The Boston Globe
- ‘Pfizer for All’ shopper platform goals to offer post-Covid enhance, Financial Times
- Harvard scientists unveil essentially the most detailed map of the mind ever: ‘It’s an alien world inside your personal head, The Boston Globe
