Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.
After being unanimously passed by the U.S. House Dec. 1, the bipartisan Mikaela Naylon Give Kids a Chance Act seemed to be headed for sure passage in the Senate before it adjourned late last week.
Shionogi & Co. Ltd. will acquire global rights to U.S. FDA approved amyotrophic lateral sclerosis therapy edaravone through a $2.5 billion acquisition deal with Tanabe Pharma Corp. Under the terms, Tanabe will form a new entity harboring both oral and intravenous (I.V.) infusion formulations of edaravone that are marketed in the U.S. as Radicava ORS and I.V. Radicava.
Biomarin Pharmaceutical Inc. followed up its May takeover of Inozyme Pharma Inc. with a much larger agreement to acquire Amicus Therapeutics Inc. for $14.50 per share in an all-cash transaction, tipping the equity-value scale about $4.8 billion.
The next stop on the comeback tour for the U.S. FDA’s Rare Pediatric Disease Priority Review Voucher program is the Senate, after the House unanimously passed the Mikaela Naylon Give Kids a Chance Act, H.R. 1262, Dec. 1.
Q32 Bio Inc. handed off rights to phase II-stage complement inhibitor ADX-097 in a deal with Akebia Therapeutics Inc. that helps the former extend its cash runway to focus on lead candidate bempikibart in alopecia areata and bolsters the latter’s efforts to build a rare kidney disease pipeline.
In an article that reads like informal guidance, U.S. FDA Commissioner Marty Makary and CBER Director Vinay Prasad discussed the criteria for using the agency’s novel plausible mechanism pathway for personalized treatments when a randomized trial isn’t feasible, as well as future uses of the approval path that could expand beyond gene and cell therapies to other biologics and even small molecules.
The U.K. Medicines and Healthcare Products Regulatory Agency has laid out an approach to medical products for rare diseases, which the agency said afflict 3.5 million U.K. citizens. One of the objectives of this program is to simplify evidence requirements for these therapies with the hope of providing patients with more rapid access to much-needed therapeutic options.
The U.K. Medicines and Healthcare products Agency (MHRA) has committed to major reforms of how it regulates drugs for rare diseases, making it easier to run clinical trials and get approvals. The new rules will be published in full early in 2026 and come into effect later in the year, but following consultation with industry, academics and patients’ groups, the agency has released a position paper setting out its plans.
Chugai Pharmaceutical Co. Ltd. and Rani Therapeutics LLC have entered into a collaboration and global license agreement to develop and commercialize an oral product that encompasses Rani’s oral delivery technology, the Ranipill, and Chugai’s rare disease antibody in development.
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