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Beam reports positive data for genetic disease treatment

Biotechnology company Beam Therapeutics has reported encouraging data for its treatment for the genetic disease Alpha-1 Antitrypsin Deficiency (AATD).
Beam announced BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation designed to directly correct the underlying genetic mutation that causes AATD, has shown a well-tolerated safety profile and efficacy in a Phase I/II trial.
According to results, 26 patients were treated with single-dose BEAM-302, which proved a well-tolerated safety profile up to 75 mg. Adverse events (AEs) were mild to moderate, with no serious AEs reported and no dose-limiting toxicities as of the data cutoff.
Treatment with BEAM-302 led to rapid and durable increases of total and functional AAT, decreases in mutant Z-AAT, and new production of corrected M-AAT.
“AATD is a serious genetic disease that can lead to significant liver disease over an individual’s lifespan along with progressive lung disease in adults, often leaving patients with limited treatment options and challenging, lifelong disease management,” said Jeffrey Teckman, Professor of Pediatrics, Saint Louis University School of Medicine.
“What makes BEAM-302 particularly compelling is its ability to directly correct the underlying genetic mutation in the SERPINA1 gene that drives both lung and liver manifestations of the disease.
“By enabling the liver to produce corrected M-AAT for the first time while reducing the toxic mutant protein, this approach has the potential to fundamentally transform how we as clinicians treat AATD and represents a meaningful advance for patients.”
“The strength and consistency of this dataset support our selection of 60 mg as the go-forward dose and give us confidence in our ability to rapidly execute this next phase of pivotal development in pursuit of an accelerated approval pathway,” added John Evans, CEO of Beam Therapeutics.
“We remain deeply committed to advancing this potentially transformative, one-time treatment for the AATD community.”
The post Beam reports positive data for genetic disease treatment appeared first on Drug Discovery World (DDW).
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New webinar: Tackling 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 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.
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
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
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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