News

#CNS #Gene therapy #Skylark Bio

Skylark Bio expands gene therapy pipeline into CNS

Skylark Bio has revealed it is expanding its pipeline with SKY-AS, a differentiated gene therapy candidate designed to restore UBE3A expression in individuals with Angelman syndrome (AS).   SKY-AS represents the company’s first central nervous system (CNS) development programme and expands Skylark’s portfolio of precision, locally delivered… Read More

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#Biotechnology #Cancer research #DDII #DDIIDigitalForum #Drug discovery #Investment #Technology #Tumours

This week in Drug Discovery (21-25 September 2026) 

News round-up for 21-25 September 2026 by Megan Thomas, Content Team at DDW.  This week’s news roundup covers the upcoming Drug Discovery Innovators & Investors Digital Forum and the Drug Discovery Innovators & Investors Conference in London, as well as the latest developments in drug discovery,… Read More

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#European Commission #European Medicines Agency #Minoryx Therapeutics #Neuraxpharm #Neurodegenerative disorders #Neuroscience

EC approves first treatment for rare neurodegenerative disease

The European Commission (EC) has granted marketing authorisation under exceptional circumstances for Nezglyal (leriglitazone), an orally bioavailable, brain penetrating, selective PPAR gamma agonist, as a treatment for male cALD patients.  It is approved in patients aged two to 12 years with Gadolinium (Gd)-negative brain lesions and is… Read More

#Editor's Choice #News #Regulatory

Antibody drug conjugate
#Antibody drug conjugate (ADC) #Ona Therapeutics #Oncology #Phase I trial

Ona Therapeutics moves to Phase I with ADC for advanced cancers

Ona Therapeutics has progressed to a Phase Ia clinical development of its lead first-in-class antibody-drug conjugate (ADC), ONA-255, for the treatment of advanced cancers.   The company says that more than half of the patients planned for the study have already been enrolled, with the trial continuing to… Read More

#Cancer Research #News

Video on function-focused antibody discovery workflows with Envisia

Explore how Envisia could support antibody discovery through controlled live single-cell experiments, sequential droplet merges, functional readouts and viable cell recovery. See how function-focused workflows could help researchers being functional insight earlier into antibody discovery and connect cellular behaviour with the cells responsible. Explore Envisia and live single-cell functional analysis. Read More

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#Drug discovery #Drug discovery events #Events #UK

Six reasons to register for Drug Discovery Innovators & Investors UK Conference

Early bird rates for the Drug Discovery Innovators & Investors (DDII) UK Conference close on 30 September 2026. Taking place on 4 November 2026 at etc.venues Monument in London, UK, the conference brings together pharma and biotech companies, technology suppliers, academia, start-ups, spin-outs and investors for a focused day of… Read More

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#Bipolar #Johnson & Johnson #Neuroscience #Phase III trial #Psychiatry

Phase III study supports lumateperone use in bipolar mania

Johnson & Johnson has announced positive topline results from a Phase III study evaluating Caplyta (lumateperone) for the treatment of manic episodes with or without mixed features in adults with bipolar I disorder.   The study met its primary endpoint, with Caplyta demonstrating a statistically significant and rapid… Read More

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Lungs
#Asthma #AstraZeneca #European Commission #European Medicines Agency

EC approves first triple-combination therapy for asthma maintenance

AstraZeneca’s fixed-dose triple-combination Trixeo Aerosphere (budesonide/glycopyrronium/formoterol fumarate dihydrate or BGF) has been approved in the European Union (EU) as a maintenance treatment for asthma.  It is approved in patients 12 years of age and older who are not adequately controlled by a combination of a medium dose… Read More

#News #Regulatory

Sleep study in older man
#AstronauTx #Funding #Neuroscience #Parkinson's

Grants support research into sleep mechanisms in Parkinson’s

AstronauTx has received funding from The Michael J Fox Foundation for Parkinson’s Research (MJFF), while Parkinson’s UK is funding an additional complementary research effort in collaboration with University College London (UCL).  The MJFF-funded project, titled ‘Ligand-Gated Ion Channel Modulation as a Disease-Modifying Treatment for Prodromal Synucleinopathies’, will… Read More

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A member of staff at the Edinburgh Genome Foundry
#Artificial intelligence (AI) #Edinburgh Innovations #Funding #Genetics / genomics #The Advanced Research and Invention Agency (ARIA)

Funding boost transforms AI-enabled biological innovation

The Edinburgh Genome Foundry (EGF), one of the world’s leading bio-foundries, is collaborating with The Advanced Research and Invention Agency (ARIA) to accelerate biological research and unlock new discoveries that benefit everyone.  The three-year programme will provide ARIA with access to and expansion of EGF’s automated laboratory… Read More

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Neuroscience Parkinson's disease
#Ariadne Bio #Neurodegenerative disorders #Neuroscience #Parkinson's #Spinout #Start-ups

Ariadne Bio spins out to target apathy in Parkinson’s disease

Palomar Labs (formerly Negev Labs), a venture studio that builds companies around neuropsychiatric drug development programmes with prior human validation, has announced the spinout of Ariadne Bio, its first portfolio company.   Ariadne Bio launches with lead programme AB-300, a clinical-stage-ready, non-hallucinogenic serotonin 2A receptor agonist in development… Read More

#Business & Investment #News #Start-up Zone

#BioIVT #Cancer research #Drug development #Drug discovery #iOnctura #Kazia Therapeutics #Sartorius #Vyriad Therapeutics #Webinar

Last chance to register: Drug discovery challenges in cancer research

Hosted by Drug Discovery World and supported by Sartorius and BioIVT, this webinar will explore the opportunities and challenges that exist within cancer research drug discovery and development. You will hear from Dr Sudha Rao, Chief Scientific Officer of Kazia Therapeutics, Karol Budzik, PhD, Business Development Associate at Vyriad Therapeutics… Read More

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Novo Nordisk head office
#Anthropic #Artificial intelligence (AI) #Cardiometabolic diseases #Collaboration #Novo Nordisk

New deals boost Novo Nordisk’s AI drug discovery ambitions

Novo Nordisk and Orbis Medicines have announced a strategic collaboration and license agreement to discover and develop oral macrocycle therapeutics for cardiometabolic diseases.   Under the terms of the agreement, Orbis Medicines is eligible to receive up to $1.4 billion in upfront and potential development and commercial milestone… Read More

#Business & Investment #News

Chemistry
#Artificial intelligence (AI) #Deep tech #Funding #Molecular medicine #Spinout #University of Glasgow

Scottish deep-tech company secures £22 million in grants

Scottish deep-tech company Chemify has received £22 million ($29.7 million) in grants from Scottish Enterprise and the UK Government.   The funding will support a significant expansion of Chemify’s capacity to digitise molecular discovery, synthesis and scale-up through a new Chemifarm.  The grant package includes £16… Read More

#Business & Investment #News

Novartis
#Monoclonal antibodies #Neurodegenerative disorders #Novartis #Phase III trial

Novartis faces September’s fourth trial disappointment with ALS drug

Novartis has revealed plans to pause development of Lifonebart (VHB937), an experimental monoclonal antibody drug targeting the TREM2 protein to treat neurodegenerative diseases.  The news comes just days after the company announced its Phase III trial of antibody oligonucleotide conjugate (AOC) del-desiran had failed.  VHB937… Read More

#News #Trending News

Heavy-chain-only antibody
#3DEXPERIENCE cloud platform #Affinity maturation #Antibodies #BIOVIA #Deep learning #Webinar

Register now: Antibody affinity maturation through deep learning and mutation analysis

This webinar, hosted by DDW, explores how deep learning and physics-based methods can be combined to improve antibody affinity maturation and epitope mapping using workflows available in BIOVIA Discovery Studio Simulation on the 3DEXPERIENCE cloud platform. Through case studies involving antibody-antigen and nanobody-receptor complexes, the work demonstrates how virtual mutagenesis,… Read More

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