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Global initiative to tackle gaps in ALS/MND drug discovery 

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A global initiative has launched to close the gap in ALS/MND drug discovery. 

The ALS Therapy Development Institute (ALS TDI), LifeArc, and Axol Bioscience announced the launch of ‘Patient induced pluripotent stem cell (iPSC)-based Research to Improve Sporadic ALS Modelling’ (PRISM ALS), a collaborative effort to expand access to high-quality, patient-derived stem cell models that better reflect the biological complexity of amyotrophic lateral sclerosis (ALS). 

The initiative aims to develop, evaluate, and make available a diverse panel of well-characterised, patient-derived induced pluripotent stem cell (iPSC) models that capture both genetic and sporadic forms of ALS. 

For researchers and drug developers, those standardised, human-relevant models could allow them to better understand disease mechanisms, identify therapeutic targets, and evaluate treatments across distinct biological subtypes.  

For people living with ALS, the effort could mean therapies are developed and tested in models that more closely mirror their own biology, increasing the likelihood that discoveries will translate into meaningful treatments. 

“We know that ending ALS will require delivering the right treatments to the right individuals,” said Dr Fernando Vieira, CEO and Chief Scientific Officer at ALS TDI.  

“By characterising iPSC-derived motor neurons from sporadic ALS and making these cells broadly accessible, PRISM ALS will facilitate global drug discovery. This programme is only possible thanks to the people living with ALS who contributed samples and data through the ARC Study.” 

ALS is a heterogeneous disease and, while 10-15% of cases are linked to inherited mutations, nearly 85% are sporadic. Much of ALS drug discovery has relied on models representing a limited number of rare genetic subtypes, constraining target discovery, limiting therapeutic testing across patient populations, and contributing to the high failure rate of clinical trials. 

 

 

 

 

 

 

The post Global initiative to tackle gaps in ALS/MND drug discovery  appeared first on Drug Discovery World (DDW).

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New webinar: Tackling drug discovery challenges in cancer research

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Cancer cells

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 and Lars van der Veen, Chief Scientific Officer at iOnctura.

Presentations will cover how cancer treatments have shifted towards reprogramming the biology driving tumour growth, immune escape and treatment resistance, the trajectory that in vivo CAR-T treatments are taking, and how challenging tumours burdened by stroma and immune-mediated resistance can be tackled.

Q&A with the speakers follows the presentations.

Register for free now.

The post New webinar: Tackling drug discovery challenges in cancer research appeared first on Drug Discovery World (DDW).

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Psilocybin proves promising in neuropathic pain mouse study

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Amid the rise of psychedelics in the mental health space, researchers have begun to explore psilocybin as a treatment for chemotherapy-induced peripheral neuropathy.

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New Spectrometry Technique Could Aid Formulation Development

New Spectrometry Technique Could Aid Formulation Development

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A new technique combining two forms of spectrometry could help biopharmaceutical companies improve their choice of formulation buffer for antibody manufacturing by revealing how molecular forms and three-dimensional shapes of complex biologics respond to their environment. That’s the view of Christian Bleiholder, PhD, a professor at Florida State University who helped develop the technique.

According to Bleiholder, what happens structurally when a complex biological molecule, such as an antibody or viral spike protein, binds to its target is currently poorly understood.

“This is where [this approach] can help with the bioprocessing and formulation,” he says, as structural changes “can affect the lifespan [of the product] and lead to issues, such as aggregation.”

Because antibodies are complex, existing techniques tend to be powerful at different levels of complexity, he explains. Mass spectrometry is particularly powerful for distinguishing molecular composition, while structural approaches such as X-ray crystallography and cryo-electron microscopy can provide high-resolution structural information.

The challenge is understanding the link between these things within a heterogeneous sample, he says.

To overcome this, Bleiholder and his team worked with Bruker Daltonics to develop Tandem-Trapped Ion Mobility Spectrometry (Tandem-TIMS). This combines tandem ion mobility spectrometry with tandem mass spectrometry to disentangle three overlapping layers of molecular complexity: molecular form, three-dimensional shape, and binding or assembly state, he says.

He explains that, if the proteins have different structures, they can be characterized with tandem ion mobility spectrometry, and then mass spectrometry can be used to look at their molecular forms and binding states.

Going forward, Bleiholder hopes the technique can be used for formulation development but also earlier, during drug discovery of new products, such as multi-specific antibodies, to determine which molecular states are important and how those change when a biologic engages its target. He also plans to look at automating the technique.

Bleiholder spoke about using Tandem-TIMS at the Bioprocessing Summit in Boston earlier this year.

The post New Spectrometry Technique Could Aid Formulation Development appeared first on GEN – Genetic Engineering and Biotechnology News.

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