Bristol Myers Squibb Co.’s decision this week to snag Bluebird Bio Inc. spinout 2seventy Bio Inc. for $102 million net – just weeks after investors bid $30 million for Bluebird itself – seemed to place a final blow on what was once a promising gene therapy company. The space in general has struggled to make business sense out of the one-time therapies that often involve complicated manufacturing and exorbitant prices, despite the life-changing value of gene therapies for patients. But despite some recent setbacks, biopharmas continue to plow forward with promising research in the field. Stepping back recently, though, was Pfizer Inc., which terminated development and commercialization of Beqvez (fidanacogene elaparvovec) less than a year after gaining U.S. FDA approval, ending its license agreement with Spark Therapeutics Inc., a unit of Roche AG.

Visen announces HKD$603M Hong Kong IPO

Visen Pharmaceuticals Co. Ltd. announced its initial public offering (IPO) on the Hong Kong Stock exchange to raise roughly HKD$603.3 million (US$77.6 million) to advance its pipeline of endocrinology assets. As previously reported by BioWorld, Visen said in April 2024 that it was targeting an IPO on the Hong Kong stock exchange to advance its three rare endocrine disease therapies in-licensed from Denmark’s Ascendis Pharma A/S.

Healios posts more upbeat data on Multistem cell therapy for ARDS

Latest findings on Healios K.K.’s stem cell therapy to treat acute respiratory distress syndrome (ARDS), coined Multistem (invimestrocel; HLCM-051), found the regenerative medicine effective in reducing the number of patient days on ventilator treatment, as well as mortality benefits. The findings are expected to support and boost Healios’ domestic regulatory filing of HLCM-051 for conditional and time-limited approval to treat ARDS, for which the company engaged Japan’s Pharmaceuticals and Medical Device Agency as early as 2022.

Torqur’s actinic keratosis data catches pharma eye, says CEO 

Term sheets are being drawn up with potential pharma partners after Torqur AG reported positive interim results from an ongoing phase II trial of bimiralisib topical gel in the treatment of actinic keratosis. Overall, 60% of patients who completed the initial treatment and follow up visit had complete, or partial clearance (of at least 75%), of their lesions. In the four-week, once daily treatment group 70% of patients responded, while in the two-week treatment group, 50% did so. Full results will be available by the end of June 2025, and Vladimir Cmiljanovic, CEO and co-founder of Torqur said planning for phase III development is in hand.

Oz promises major improvements if confirmed to lead CMS

Cardiothoracic surgeon and television personality Mehmet Oz, President Donald Trump’s nominee for administrator of the Centers for Medicare & Medicaid Services (CMS), faced the U.S. Senate’s finance committee on March 14, suggesting the implementation of artificial intelligence (AI) technologies and the reduction of drug prices would be among his top priorities, if confirmed. AI would not only help reduce fraud, waste and abuse, it could bring better health care to rural hospitals, liberate practitioners from paperwork, offer a more consistent pre-authorization process, and speed up payments for approved drugs and medical device products, he said. Tackling the “pricing spread” caused by pharmacy benefit managers is another concern he plans to address. “If we can insist on complete and radical transparency on what’s going on with our drug markets, it will help CMS tremendously,” Oz told the committee.

Trump’s ‘transgender mice’ remark makes waves

The matter of science has proven contentious during the terms of U.S. President Donald Trump, and the latest kerfuffle involves his claim that the government has spent $8 million on “making mice transgender.” Mainstream media outlets turned critical eyes to the remark. Some speculated that he meant to say “transgenic mice,” while others insisted that he meant what he said, and has the evidence to prove what he called the waste of tax dollars.

GSK’s $1.15B Idrx buyout leads slow M&A market as deals stay strong

Biopharma deal activity climbed in early 2025, reaching $37.38 billion in the first two months, up from $35.66 billion a year ago. February saw $8.76 billion in deals, down 69% from January’s $28.63 billion, which included 11 transactions valued at $1 billion or more. Early 2025 saw the second-highest biopharma deal total on record, trailing only the $42.19 billion logged in the first two months of 2022.

Also in the news

Allarity, Allovir, Armata, Blue Earth, Boehringer Ingelheim, Chromadex, Cosciens, Dogwood, Epilepsia, Ennodc, Grifols, Heidelberg, Illexcor, Immunoprecise, Johnson & Johnson, Kalaris, Meiragtx, Merck, Mira, Montara, Neural, Nosis, Nurix, Otsuka, Quralis, Rivus, Salipro, Senti, Shionogi, Sotio, Springworks, Tarsus, Theratechnologies, Transcode, Viiv, Zai Lab