Life science news 6 July 2026: the FDA expands the world’s first CRISPR gene therapy to children as young as two, Ipsen commits up to $1.75 billion for a late-stage blood cancer drug, and two first-in-class FDA approvals reshape the cell therapy landscape in a busy week for pharma, biotech and healthcare.
Vertex Pharmaceuticals announced on 1 July that the FDA has expanded approval of Casgevy (exagamglogene autotemcel) to patients aged two years and older with sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia. The supplemental approval makes Casgevy the first and only gene therapy cleared for children as young as two in both conditions, extending eligibility to approximately 5,500 additional children in the United States. The decision was supported by data from the ongoing Phase 3 CLIMB-141 and CLIMB-151 studies, in which all eight efficacy-evaluable patients with sickle cell disease aged five to eleven achieved the primary endpoint of no severe vaso-occlusive crises for at least twelve consecutive months, and eight of nine evaluable patients with beta thalassemia achieved transfusion independence for twelve consecutive months. The FDA granted approval in just 53 days under the Commissioner’s National Priority Voucher programme. Vertex said regulatory review for the label expansion is also underway in the United Kingdom and Saudi Arabia.
The FDA approved Tregzi (Orca Bio) on 30 June, the first regulatory T cell-based immunotherapy cleared for use in the United States. Tregzi is approved for use in matched-donor haematopoietic stem cell transplantation to improve chronic graft-versus-host disease-free survival in adults with haematological malignancies, including acute leukaemia and myelodysplastic syndrome. The approval was based on the pivotal Precision-T Phase 3 trial, in which 187 patients were randomised to receive either Tregzi or conventional donor grafts, with the Tregzi arm demonstrating a one-year chronic GVHD-free survival rate of 78% compared with 38% for the control. Orca Bio has priced Tregzi at a wholesale acquisition cost of $428,000 and has established a commercial manufacturing facility in Sacramento, California.
Ipsen agreed to acquire Kartos Therapeutics for $450 million upfront, with the total deal value potentially reaching $1.75 billion through regulatory approval and sales-related milestones. The acquisition gives the French pharma company access to navtemadlin, an oral MDM2 inhibitor designed to restore the function of the tumour-suppressing protein p53 in myelofibrosis. Navtemadlin is currently in the pivotal Phase 3 POIESIS trial, enrolling more than 600 patients across over 250 sites globally as an add-on to ruxolitinib in patients with a suboptimal response to that treatment, with top-line data expected in 2027. The deal is Ipsen’s second major transaction of the week, following its proposed acquisition of Memo Therapeutics for in excess of €700 million. The Kartos transaction is expected to close in the third quarter of 2026 and begin contributing to Ipsen’s core operating income from 2029.
Zymeworks entered a definitive agreement to acquire Theravance Biopharma for $17.00 per share in cash, representing an equity value of approximately $929 million, adding the COPD therapy Yupelri (revefenacin) to its portfolio. Yupelri is the only approved nebulised, once-daily long-acting muscarinic antagonist for the maintenance treatment of chronic obstructive pulmonary disease, commercialised in the United States through a collaboration with Viatris. The deal, unanimously approved by both boards, also includes a contingent value right tied to potential future monetisation of ampreloxetine, a candidate that failed a Phase 3 trial in neurogenic orthostatic hypotension in March 2026. Zymeworks said the transaction would meaningfully expand its revenue sources beyond its existing oncology portfolio, with immediate cash flow from Yupelri. The acquisition is expected to close in the second half of 2026, subject to shareholder and regulatory approvals.
In further life science news 6 July 2026, the week also brought a billion-dollar financing for a commercial-stage genetic medicines company, a significant fundraising milestone for a UK-listed biotherapeutics group, and two notable UK regulatory developments from the MHRA.
BridgeBio Pharma raised $1 billion in convertible preferred equity on 1 July, led by Sixth Street with participation from HealthCare Royalty, a business of KKR. Sixth Street funded $800 million and HealthCare Royalty $133.9 million, with the preferred shares carrying a 7% initial dividend and an initial conversion price of approximately $138 per share, representing a premium of more than 100% to BridgeBio’s 30-day volume-weighted average trading price. The financing comes as Attruby continues to generate strong commercial revenue, and as BridgeBio prepares for three additional potential US product launches over the next twelve months across BBP-418 for limb-girdle muscular dystrophy type 2I/R9, encaleret for autosomal dominant hypocalcaemia type 1, and infigratinib for achondroplasia.
London-listed PureTech Health announced on 2 July that its founded entity Celea Therapeutics has completed a $180 million financing to advance deupirfenidone (LYT-100), an investigational next-generation antifibrotic, into Phase 3 testing for idiopathic pulmonary fibrosis. Participants included RA Capital Management, Leaps by Bayer, and PureTech Health, alongside a US-based healthcare fund and a sovereign wealth fund. The proceeds will support the planned early third-quarter 2026 initiation of the SURPASS-IPF trial, the first head-to-head Phase 3 study in IPF, which will evaluate the superiority of deupirfenidone against pirfenidone. Deupirfenidone is a deuterated form of pirfenidone designed to offer improved tolerability, and showed potential to stabilise lung function decline over 26 weeks in the Phase 2b ELEVATE IPF trial. Celea and the transferred assets were valued at $100 million before the round and $302.5 million after it on a fully diluted basis.
The MHRA announced on 1 July that it has authorised an intravesical delivery system containing gemcitabine (Janssen-Cilag) for adults with BCG-unresponsive high-risk non-muscle invasive bladder cancer with carcinoma in situ who are ineligible for or have chosen not to undergo radical cystectomy. The marketing authorisation was granted on 24 June via the International Recognition Procedure Route B. The system is placed into the bladder by a healthcare professional and provides sustained local release of gemcitabine over approximately three weeks before removal. In the supporting clinical study, 82% of patients achieved a complete response, with 51% of responders maintaining their response for at least twelve months. The product was previously approved in the United States by the FDA in September 2025.
The MHRA published a new position paper on 2 July setting out its data requirements to support regulatory decision-making, highlighting significant gaps in the UK’s health data infrastructure. The paper acknowledges that the agency currently lacks access to sufficient regulatory-grade real-world data to meet all its needs, a finding echoed by the Sudlow Review in 2024, which concluded that the UK’s health data systems are not yet fit for purpose. The MHRA has framed the document in the context of the government’s £600 million investment in the UK Health Data Research Service, announced in April 2025, which aims to improve access to datasets relevant to public health and strengthen the UK’s position as a leader in life sciences regulation. The paper outlines specific use cases across pharmacovigilance, benefit-risk assessment, and proactive safety monitoring, and will inform how the new service is designed to support regulatory functions.
That’s your life science news digest for 6 July 2026, back next Monday with the latest from pharma, biotech and healthcare at www.lifesciencedaily.news. Catch up on the 29 June news roundup
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