Quantum-inspired power could brighten drug discovery

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In the realm of drug discovery, the Harvard-educated chemist Nick Paras, Ph.D. compares the early stages of identifying new compounds to a game of Zelda, in which a player at times must navigate through dark rooms and solve puzzles to progress. Similarly, medicinal chemists and biochemists too must traverse the vast, often obscure landscape of cellular assays and biochemical screens in pursuit of potential treatments.

“You’re in a dark room, fumbling around for the light switch,” said Paras, a professor at the Institute for Neurodegenerative Diseases at University of California, San Francisco (UCSF). He suggests that the early stages of drug discovery can, in some ways, mirror the experience of navigating the dark, puzzling dungeons of a game like The Legend of Zelda, where players confront a complex, unknown environment. Much like this, researchers often find themselves…

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Capsida Biotherapeutics to present preliminary findings on engineered AAV capsids at ASGCT annual meeting

The gene therapy platform company Capsida Biotherapeutics (Thousand Oaks, California) is gearing up to unveil initial data on its engineered adeno-associated virus (AAV) capsids at the upcoming American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16–20 in Los Angeles.

In developing these capsids, Capsida Chief Scientific Officer Susan Catalano explained that the company employed directed evolution and a high-throughput screening platform to optimize capsid performance across key domains. “We start with a disease and aim to create the best possible therapy, as opposed to trying to mold a disease profile around the limitations of wildtype capsids,” Catalano said.

Preliminary results suggest these engineered AAV capsids can reach up to 68% of neurons across multiple brain regions when delivered intravenously. That finding could translate to new possibilities in gene therapies targeting common neurological diseases that have…

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Brain breakthroughs: Aprinoia Therapeutics’ harnesses AI and strategic partnerships to propel neurodegenerative disease research

Neurodegenerative disease research is witnessing significant advances. To that end, Hong Kong-headquartered Aprinoia Therapeutics is embracing a ‘precision neuroscience’ approach to neurodegeneration diagnostics. The company’s lead program, APN-1607, represents a new generation of advanced tau positron emission tomography (PET) tracers, which play a crucial role in effective and efficient diagnosis of patients suffering from neurodegenerative disorders.

Aprinoia Therapeutics’ strategy to neurodegenerative disease research focuses in part on artificial intelligence (AI) and forging strategic collaborations. Paul Tempest, the head of medicinal chemistry at Aprinoia, leads a team of experts in the field of neuroscience, AI, and pharmaceutical development. In a recent interview, Tempest explained how this multifaceted approach supports innovation in the development of novel diagnostic tools and therapies for neurodegenerative diseases.

AI in dr…
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Lonza and ABL Bio partner on bispecific antibody development for immuno-oncology and neurodegenerative diseases

Lonza (LON:0QNO) and South Korea-based ABL Bio (KOSDAQ:298380) have joined forces to accelerate bispecific antibody development for immuno-oncology and neurodegenerative diseases. Over email, Basel, Switzerland–headquartered Lonza noted that its experience with novel molecular formats enables the company to handle ABL Bio’s unique challenges.

In the partnership, Lonza will provide submission-ready chemistry, manufacturing and controls (CMC) data for investigational new drugs (INDs), explained Andrew Brown, Lonza’s head of global process development support. “Leveraging more than three decades of expertise and innovative technologies, we provide an integrated drug substance and drug product program,” Brown said. “Thanks to our experience with new molecular formats, we know how to handle [ABL Bio’s] specific challenges.”

In the domain of bispecific antibody development, Lonza supports customers with problem-solving aiming…

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QRL-201, a novel therapy targeting STMN2 expression, enters phase 1 ALS trial

Cambridge, Massachusetts-based QurAlis has dosed the first patient with  the biotherapeutic QRL-201 in a Phase 1 clinical trial (ANQUR) in Canada. QRL-201 is a novel therapy targeting the restoration of STATHMIN-2 (STMN2) expression in amyotrophic lateral sclerosis (ALS) patients. QurAlis believes ANQUR the first study to evaluate such a treatment.

Stathmin-2 (STMN2), also known as superior cervical ganglia neural specific 10 (SCG10), belongs to the stathmin protein family involved in the regulation and dynamics of microtubules. Predominantly expressed in neurons, STMN2 serves key functions in neuronal development, axonal growth and regeneration.

Recent research has revealed the significance of STMN2 in neurodegenerative diseases, especially ALS and frontotemporal dementia (FTD). Researchers have linked the pathogenesis of those conditions to the loss of STMN2 function.

“In mice, STMN2 deletion causes axonal degeneration and loss of muscle innervation, w…

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QurAlis targets UNC13A RNA mis-splicing in ALS and FTD with novel FlexASO platform

QurAlis, a privately-held company specializing in precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, has debuted its latest program. This initiative targets UNC13A RNA mis-splicing, a critical gene alteration present in ALS and frontotemporal dementia (FTD). FTD has recently gained renewed attention following the disclosure of Bruce Willis’ diagnosis of the condition. UNC13A is a vital regulator of neurotransmitter release at synapses, and the genetic alteration resulting in mis-splicing is estimated to occur in 58% of ALS patients and up to half of FTD cases.

To tackle this gene alteration, QurAlis has developed its proprietary FlexASO splice modulator platform. This technology uses antisense oligonucleotides (ASOs) to correct mis-splicing, restore UNC13A protein production, and reduce cryptic exons that may contribute to disease progression. Kasper Roet, QurAlis CEO, explained that “FlexASOs operate on the s…

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FDA launches 5-year initiative for rare neurodegenerative diseases 

FDA has unveiled its Action Plan for Rare Neurodegenerative Diseases, including amyotrophic lateral sclerosis (ALS).

The plan will involve the use of public-private partnerships and incorporate feedback from patients. The initiative will encompass regulatory science initiatives, changes to existing programs and new policy initiatives.

Milestones of the initiative include the creation of an FDA rare neurodegenerative diseases task force and public-private partnerships for rare neurodegenerative diseases in the fiscal year 2022. In addition, from fiscal years 2022 to 2026, the plan aims to develop disease-specific science strategies.

The initiative is an outgrowth of a Public Law 117-79, the “Accelerating Access to Critical Therapies for ALS Act” (ACT for ALS) President Biden signed into law on December 23, 2021.

“We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases,” said…

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Scientists find new potential drug targets for neurodegenerative diseases

Maria Clara “Maca” Franco, center, with Kyle Nguyen, left, and Lydia Bastian. Franco’s research investigates neurodegenerative diseases. Image courtesy of Oregon State University.

Researchers at Oregon State University (OSU) have discovered a new class of potential drug targets for diseases like Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis (ALS).

The scientists are working to identify the best method to attack the targets — oxidized proteins. The most potent oxidant of the bunch is peroxynitrite, which is produced in conditions involving inflammation. Oxidized proteins and free radicals can damage DNA, lipids and proteins implicated in neurodegenerative diseases and other conditions.

Peroxynitrite is produced thanks to the diffusion-limited reaction of nitric oxide and superoxide.

Peroxynitrite appears to be especially pernicious when it oxidizes heat shock prote…

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What is needed to develop disease-modifying therapies for Parkinson’s and Alzheimer’s 

Photo by Edward Jenner from Pexels

Developing disease-modifying therapies for neurodegenerative diseases remains a pressing need.

The incidence of neurodegenerative disease is ramping up in the U.S. and elsewhere as much of the global population ages. One out of three seniors dies with Alzheimer’s disease or another form of dementia, according to the Alzheimer’s Association.

Parkinson’s disease is also becoming more widespread. Between 2015 and 2040, the number of people with Parkinson’s could nearly triple, rising from 6.3 to 17.5 million.

But developing drugs that can slow or stop the progression of such diseases poses a significant challenge for drug developers. Eli Lilly’s donanemab, for instance, showed promise earlier this year in treating Alzheimer’s in a Phase 2 study summarized in NEJM.

Another Alheimer’s candidate, aducanumab from Biogen, has also shown promise, although late l…

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