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Maze, AC Immune, Merck, OnKure

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Hello! Today we learn whether AI could help accelerate the development of n-of-1 therapies (hint: not yet), explore why the FDA hasn’t offered Orange Book guidance on drug-device combinations, and talk about CRISPR . A lot of.

The must-know this morning

  • AC Immune has worldwide licensing rights sold on an experimental immunotherapy for Alzheimer’s disease to the Japanese drug manufacturer Takeda. AC Immune received $100 million from Takeda for the therapy, called ACI-24,060, and is eligible for an additional $2.1 billion in future payments.
  • Merck reported negative results from an interim analysis of a phase 3 trial using the investigational anti-TIGIT antibody as an adjunctive treatment for patients with high-risk melanoma.
  • Developer of anti-cancer drugs OnKure goes public via a reverse fusion of Reneo Pharma.

AI may not yet help develop ‘n-of-1’ treatments

Can AI be used to create ‘n-of-1’ treatments for the rarest genetic diseases? The jury is still out, a new study suggests.

There has been a wave of antisense oligonucleotide drugs designed specifically to target the single mutation causing a patient’s symptoms. This is a compelling, yet time and cost effective approach for most companies, which has led some, like Creyon Bio, to use artificial intelligence as a way to speed up the process.

Last week, Creyon said it had successfully administered a modified treatment to a patient that showed some signs of efficacy. What’s less clear, however, is whether machine learning has made the drug faster or safer, writes STAT’s Jason Mast. Creyon initially said it would use AI to create the new drug within seven months; instead it took just over a year, which is no better than other n-of-1 approaches. It took ten months to develop Milasen, the first tailor-made drug. And Creyon’s drug did not cost “significantly less” than other n-of-1 drugs, which cost more than $1 million to produce.

Read more.

The FDA’s hands-off attitude toward drug-device combinations

Drug makers have long asked the FDA for clearer guidance on how patents should be listed in a special registry called the Orange Book. This is important because it helps brand drug manufacturers inform generic competitors about their intellectual property. However, regulators have not provided the necessary guidance for combination products such as inhalers and auto-injectors, writes STAT’s Ed Silverman. This is frustrating for industry players and patient advocates alike.

Recently, the FTC has stepped in, warning companies about inappropriate patent listings in the Orange Book and taking legal action against those who try to expand their monopolies.

“The FDA needs to show some leadership and say what things don’t make it into the Orange Book, but instead they’ve been freaking out for years,” the leader of a nonprofit focused on pharmaceutical patent issues told STAT. “There’s just too much uncertainty that hasn’t been cleared up yet.”

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We now know Tome’s gene editing target

From STAT’s Jason Mast: Despite all the hype surrounding CRISPR, the tool has been largely effective at knocking out genes or making minor edits. So when two scientists showed up a few years ago and advertised technology Because it was able to insert large genes into human cells without causing major damage to DNA, investors agreed to invest more than $200 million in an effort to make it happen.

That round, for Tome Biosciences, was announced in December. On Friday, CEO Rahul Kakkar explained for the first time at a major gene therapy conference how the company plans to use the technology: initially to treat a rare disease called phenylketonuria and to design a natural killer cell therapy that developed to safely treat autoimmune diseases. diseases. In addition, there are plans to develop a treatment for heart disease and treatments for the rare diseases homocystinuria and hemochromatosis.

The company will obviously have competition. Tessera did that too showed animal data for the treatment of phenylketonuria with a gene editing system for changing individual letters. And Prime Medicine has a very similar big gene insertion technology, although not many details about specific diseases have yet to be revealed.

A CRISPR approach has not cured HIV

An attempt to use CRISPR editing to remove HIV from patients’ genomes didn’t work. In a Phase 1 study from Excision Therapeutics, a CRISPR-based therapy was administered to five patients with HIV. Researchers then took three of these patients off conventional antiviral treatment, hoping they would not have to resume daily medication. But despite the CRISPR treatment, virus levels quickly recovered.

This is likely because the gene editing approach was not efficient enough, writes STAT’s Jason Mast. Excision used an adeno-associated virus to spread the gene-editing tool throughout the body – and AAVs don’t reach all cells. So even if the virus was cleared from one part of the body, it likely lingered elsewhere.

But there were signs of hope: One patient’s infection did not clear up for about four months after stopping antivirals, while it typically returned after three or four weeks. This suggests that CRISPR editing could eventually be linked to other approaches to cure HIV.

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Maze gets another chance to collaborate on the Pompe drug

Last year, Sanofi was set to pay $150 million in cash and stock to license an experimental Pompe disease treatment made by Maze Therapeutics – but the FTC blocked the deal and it fell through. Now Maze is working with Japanese drugmaker Shionogi to further develop MZE001, again with $150 million upfront, writes STAT’s Andrew Joseph.

Sanofi already sells two intravenous infusions for Pompe, raising concerns among regulators, who said a takeover would increase Sanofi’s monopoly on the disease and deter competitors from entering the market. The French drugmaker walked away from the deal without personally notifying the company: CEO Jason Coloma told STAT last year that he was “personally disappointed” by the failure of the Sanofi deal.

“Shionogi has a track record of developing and delivering innovative medicines to patients around the world, and we are confident they are the right partner to further develop MZE001 through clinical trials to help patients with this life-threatening condition can reach as quickly as possible. ,” Coloma said in a statement.

Read more.

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  • Pfizer and AstraZeneca announce new investments of almost $1 billion in France, Reuters
  • The first person to undergo a genetically modified pig kidney transplant dies nearly two months later, The Boston Globe reports
  • Consumer platform ‘Pfizer for All’ aims to provide a post-Covid boost, Financial times
  • Harvard scientists unveil the most detailed map of the brain ever: ‘It’s an alien world inside your own head, The Boston Globe