Mission Therapeutics has dosed the first patient in its Phase Ib proof-of-mechanism study of MTX325 in patients with Parkinson’s disease (PD). MTX325 is a first-in-class, highly potent and selective, orally bioavailable and brain-penetrant inhibitor of the deubiquitinating enzyme (DUB) USP30. It has been designed to… Read More
Beam Therapeutics will present initial data from the ongoing Phase I/II clinical trial evaluating BEAM-301 for the treatment of glycogen storage disease type Ia (GSDIa) at the 33rd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). BEAM-301 is a liver-targeting lipid-nanoparticle (LNP) formulation… Read More
BioNTech has announced the first topline results from the AHEAD-MERIT Phase II/III clinical trial for its investigational mRNA cancer immunotherapy BNT113 (tenumomeran/raplemomeran or tenu-ra). The clinical trial is evaluating BNT113 in combination with pembrolizumab compared to pembrolizumab monotherapy as a first-line treatment for patients with unresectable recurrent… Read More
The US Food and Drug Administration (FDA), a division within the US Department of Health and Human Services (HHS), has announced the Rare Disease Framework Initiative to accelerate the development of drugs and biologics for rare diseases. According to the regulator, the initiative will develop an optimised,… Read More
Sapreme Therapeutics has shared new preclinical data from its lead Duchenne muscular dystrophy (DMD) programme, SPM-2551, at the 22nd Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), taking place in Denver, Colorado, US. SPM-2551 is intended to restore dystrophin to more than 15% of normal levels –… Read More
Despite a challenging funding landscape, 2026 has seen two groundbreaking advances in mRNA vaccine discovery; the first-ever mRNA seasonal influenza vaccine for adults and the first positive Phase III readout for an mRNA-based cancer therapy. This eBook explores how the mRNA platform has evolved since its use in Covid-19 vaccines,… Read More
This eBook explores how the mRNA platform has evolved since its use in Covid-19 vaccines, from the prevention of seasonal illnesses to personalised therapies for a range of diseases.
The Medicines and Healthcare products Regulatory Agency (MHRA) has published its 2030 Strategy, setting out ambitious plans to translate scientific and technological innovation into better health outcomes. The regulator says that the commitments will enable it to keep pace with rapid advances in science, technology and personalised… Read More
Roche and Zealand Pharma have shared positive topline results from the Phase II ZUPREME-2 trial evaluating petrelintide versus placebo in people living with overweight or obesity and type 2 diabetes. The trial met its primary endpoint, demonstrating that once-weekly subcutaneous injections of petrelintide (escalated every fourth week)… Read More
argenx has revealed that it will discontinue the Phase III UNITY study of Vyvgart Hytrulo (efgartigimod subcutaneous – efgartigimod alfa and hyaluronidase-qvfc) in adults with moderate-to-severe Sjögren’s disease. The company said the decision is based on the recommendation from an Independent Data Monitoring Committee (IDMC) to stop… Read More
CSL and Alentis Therapeutics have entered into an exclusive global collaboration agreement to co-develop and co-promote lixudebart, which has the potential to become a first-in-class therapy targeting claudin-1 across a range of kidney, liver and other diseases. Under the terms of the agreement, Alentis will receive a… Read More
Scientists have uncovered a previously unknown mechanism that helps breast cancer cells survive the intense stresses of being malignant, revealing a promising new target for future cancer therapies. A new study has shown that breast cancer cells become highly dependent on a cell membrane protein called GPR89… Read More
The results of a new study show AI/ML investment jumping after three flat years, with three quarters (76%) of respondents planning to increase spending on technology in the next two years, up from 60% in 2023, 62% in 2024, and 63% in 2025. However, the leading barrier… Read More
Caribou Biosciences has announced that it is discontinuing its two allogeneic CAR-T cell therapy programmes due to financing constraints. It will discontinue development of vispa-cel for the treatment of relapsed or refractory (r/r) B cell non-Hodgkin lymphoma and CB-011 for the treatment of r/r multiple myeloma. … Read More
Alector has granted Genentech worldwide rights to develop and commercialise its AL050 programme, a brain-penetrant engineered glucocerebrosidase (GCase) enzyme replacement therapy (ERT). As part of the deal, Alector will receive a $100 million upfront payment, with potential for up to $1.17 billion in additional milestone payments, as… Read More
Rewind Therapeutics has announced additional financing to advance the development of its lead GPR17 antagonist programme, a potential disease modifying oral therapy intended to promote remyelination and restore neurological function in multiple sclerosis (MS). MS is characterised by damage to myelin, but there are no approved therapies… Read More
