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In the high-stakes world of drug development, a pill is never just a pill. Every oral drug that reaches the market has passed through a tightly managed series of steps—none more critical, or more vulnerable, than tech transfer. 15 July 2025
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A privately-held biotech company developing multiple-dose gene therapies to treat patients with genetic and idiopathic diseases, focusing first on hemophilia B.
Latest News & Features of interest to Lonza
Pharmaceutical
In the high-stakes world of drug development, a pill is never just a pill. Every oral drug that reaches the market has passed through a tightly managed series of steps—none more critical, or more vulnerable, than tech transfer. 15 July 2025
Biotechnology
Ultragenyx Pharmaceutical has been dealt a regulatory setback, as the American medicines regulator has declined to approve its gene therapy UX111 (formerly ABO-102) for Sanfilippo syndrome type A. The decision, delivered in a complete response letter, will delay any potential approval until at least 2026. 14 July 2025
Biotechnology
Dutch privately held Amarna Therapeutics, which specializes in transformative gene therapies announced the formalization of an agreement with biologics manufacturing partner, Sweden’s NorthX Biologics, to accelerate the development of Nimivec, Amarna’s next-generation gene therapy platform targeting immune-mediated diseases. 9 July 2025
Biotechnology
Japan’s JCR Pharmaceuticals closed 15% higher after Wednesday’s trading, as it signed a new licensing deal. 9 July 2025
Biotechnology
Spanish gene therapy specialist VIVEbiotech has named Patricio Massera as its new president, bringing in a seasoned CDMO leader to help scale the business following a fresh investment from US firm Ampersand Capital Partners. 4 July 2025
Biotechnology
UK-based contract research, development and manufacturing organization Ingenza has named Nick Challoner as its new chief executive, effective October 1, 2025. He replaces founder Ian Fotheringham, who will move into the role of president. 3 July 2025
Biotechnology
Bayer’s cell therapy subsidiary BlueRock Therapeutics is shifting its priorities, cutting around 50 jobs and shutting its research labs in Massachusetts to streamline operations and concentrate on advanced-stage programs. 26 June 2025
Biotechnology
The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to detalimogene voraplasmid (previously known as EG-70), Canadian company enGene’s lead investigational therapy for the treatment of high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS). 26 June 2025
Biotechnology
The Alliance for Regeneration Medicine (ARM), a trade group that represents more than 400 members companies across 25 countries, including emerging and established biotech firms, has taken aim at the powers that be in US healthcare. 23 June 2025
Pharmaceutical
US drugmaker Vertex Pharmaceuticals has announced simultaneous presentation and publication of updated data from the Phase I/II portion of the Phase I/II/III FORWARD-101 clinical trial of zimislecel (VX-880), an investigational stem cell-derived, fully differentiated islet cell therapy, in people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). 23 June 2025
Latest In Brief for Lonza
Biotechnology
US biotech Nanoscope Therapeutics has begun a rolling submission to the American medicines regulator for MCO-010, the first gene-agnostic therapy seeking approval for retinitis pigmentosa (RP). 15 July 2025
Latest Cell & Gene Therapy Ones To Watch News
German biotech TQ Therapeutics today announced the acquisition of Juno Therapeutics GmbH, a cell therapy company, through a share purchase agreement. Financial terms of the agreement were not disclosed. 28 May 2025
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29 March 2025
Cell & Gene Therapy Ones to Watch Companies
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