News and Resources from the WTWH Media Life Science Network - Page 213 of 968

Science unbound: AI and open data accelerate the pace of discovery

[Tartila/Adobe Stock]

Scientists have long been perceived and portrayed in film as old people in white lab coats perched at a bench full of bubbling fluorescent liquids. The present-day reality of scientific research is quite different from old stereotypes, with AI-driven scientific breakthroughs emerging as a major driving force behind new discoveries. Scientists are increasingly data jockeys in hoodies sitting before monitors analyzing enormous amounts of data. Modern day labs are more likely composed of sterile rows of robots doing the manual handling of materials, and lab notebooks are now electronic, in massive data centers holding vast quantities of information. Today, scientific input comes from data pulled from the cloud, with algorithms fueling scientific discovery the way bunsen burners once did.

Advances in technology and especially instrumentation, enable scientists to collect and process data at an …

Development challenges and regulatory changes for cell and gene therapies

[BillionPhotos.com/Adobe Stock]

Cell and gene therapies are the fastest growing area for drug development. The groundwork for this category was laid with the mapping of the human genome in 2003, and the field has developed rapidly in the intervening decades. These powerful therapies have significant potential for the treatment of cancer and other previously “undruggable” diseases. But cell and gene therapies also have unique characteristics that can lead to manufacturing challenges and extended regulatory timelines. As of February 2020, cell and gene therapies account for 12% of the preclinical pipeline and 16% of the clinical pipeline.

Given the scale of investments made to support these development efforts and advancements possible, drug sponsors have looked to the U.S. FDA to consider accelerating gene and cell therapy development. The regulatory body has responded by shifting its departmental structure to st…

Q&A: USC researchers uncover potential osteoarthritis treatment breakthrough with R805/CX-011

The left image depicts osteoarthritic cartilage damage in animal studies without any treatment, while the right image reveals articular cartilage regeneration following a knee injection of the novel drug R805/CX-011. [Image: Evseenko Lab/USC Stem Cell]

Researchers at the Keck School of Medicine at the University of Southern California (USC) have discovered a drug compound, R805/CX-011, with the potential to alleviate the painful hyperinflammation from osteoarthritis. The findings concerning the prospective osteoarthritis treatment breakthrough, published in Science Translational Medicine, showed positive effects in animal models and could pave the way for human trials.

We recently had the opportunity to speak with Dr. Denis Evseenko, a professor of orthopedic surgery, stem cell research, and regenerative medicine at the Keck School of Medicine, about the research. In the Q&A, Dr. Evseenko discusses the poten…

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…

The 50 best-selling pharmaceuticals of 2022: COVID-19 vaccines poised to take a step back

[sizsus/Adobe Stock]

The COVID-19 pandemic has had a profound impact on the best-selling pharmaceuticals, leading to shifts in the list with Pfizer and BioNTech’s Comirnaty surpassing AbbVie’s Humira for the No. 1 spot in 2021. That momentum continued in 2022, with Pfizer and BioNTech jointly raking in $59.1 billion in revenue from the sales of the COVID-19 vaccine. Although Comirnaty maintained its position as the best-selling pharmaceutical of 2022, it experienced a roughly 5% drop in sales compared to the previous year.

As the best-selling pharmaceuticals of 2022 demonstrate, signs are emerging that the reign of COVID-19 vaccines and other therapies appears to be slipping.. The two companies — and Moderna — plan on hiking prices to address the weakening demand.

Humira: The second best-selling pharmaceutical of 2022 looking strong

Meanwhile, the heavyweight tumor necrosis factor (TNF) block…

Using AlphaFold, Insilico Medicine produces AI drug discovery in record time

This is the AI-powered autonomous robotics lab called Life Star in Suzhou that Insilico opened in January. [Insilico Medicine]

Capitalizing on AI drug discovery, an international group of researchers employed DeepMind’s deep learning-driven AlphaFold protein structure database to swiftly design and synthesize a potential hepatocellular carcinoma (HCC) drug in only 30 days.

The AI drug discovery project consisted of Insilico Medicine, the University of Toronto’s Acceleration Consortium and researchers including Nobel laureate Michael Levitt. The team applied AlphaFold to Insilico’s end-to-end AI-powered drug discovery platform, Pharma.AI. With the integration of the biocomputational engine PandaOmics and the generative chemistry engine Chemistry42, the AI drug discovery project identified a novel treatment pathway for HCC and developed a potent inhibitor based on a predicted protein structure. Read more

Using AlphaFold, Insilico Medicine produces AI drug discovery in record time

This is the AI-powered autonomous robotics lab called Life Star in Suzhou that Insilico opened in January. [Insilico Medicine]

Capitalizing on AI drug discovery, an international group of researchers employed DeepMind’s deep learning-driven AlphaFold protein structure database to swiftly design and synthesize a potential hepatocellular carcinoma (HCC) drug in only 30 days.

The AI drug discovery project consisted of Insilico Medicine, the University of Toronto’s Acceleration Consortium and researchers including Nobel laureate Michael Levitt. The team applied AlphaFold to Insilico’s end-to-end AI-powered drug discovery platform, Pharma.AI. With the integration of the biocomputational engine PandaOmics and the generative chemistry engine Chemistry42, the AI drug discovery project identified a novel treatment pathway for HCC and developed a potent inhibitor based on a predicted protein structure. Read more

Amylyx Pharmaceuticals launches phase 2 trial for AMX0035 Wolfram syndrome therapy

Cambridge, Massachusetts–based Amylyx has dosed the first participant in its phase 2 HELIOS clinical trial of AMX0035 focusing on Wolfram syndrome. The condition is an ultra-rare genetic disorder involving the endocrine system. Symptoms of Wolfram syndrome can include diabetes insipidus, diabetes mellitus, optic atrophy and hearing loss.

In September, Amylyx won FDA approval for AMX0035 (sodium phenylbutyrate and taurursodiol), marketed as Relyvrio, for adults with amyotrophic lateral sclerosis (ALS).

Both sodium phenylbutyrate and taurursodiol are smal molecules demonstrating neuroprotective effects in preclinical studies.

Trial design

The HELIOS is an exploratory open-label proof of biology study. Amylyx anticipates topline results from HELIOS in 2024.

The HELIOS trial is an exploratory open-label, single-group proof of biology study, with Amylyx anticipating topline results in 2024. The trial aims to assess the safety and efficacy of AMX0035 in…

Chiesi Farmaceutici acquires Amryt Pharma to bolster access to rare disease therapies

Chiesi Farmaceutici, a family-owned international pharma company, has completed its $1.25 billion acquisition of rare disease-focused biopharma Amryt Pharma (Nasdaq:AMYT). The companies announced the deal in January and projected it would close in the first half of 2023.

Strengthening Chiesi’s global rare disease focus

Chiesi’s decision to acquire Amryt Pharma is part of a broader trend of increased M&A activity in the pharma sector. In the first few months of 2023, Pfizer, Sanofi, AstraZeneca, and Amgen have also announced significant acquisition plans.

The Chiesi acquisition aims to strengthen Chiesi’s Global Rare Diseases business division. Amryt Pharma’s product portfolio includes three approved commercial products:

Lojuxta/Juxtapid (lomitapide) for treating homozygous familial hypercholesterolemia. This condition involves high levels of low-density lipoprotein cholesterol (LDL-C) in the blood, contributing to premature car…

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…

Accelerating Alzheimer’s research: ADDF’s chief scientific officer reflects on the Lauder Foundation’s $200M gift

Beta-amyloid plaques and tau in the brain. [Image from National Institute of Aging]

The Estée Lauder family has donated $200 million to the Alzheimer’s Drug Discovery Foundation (ADDF), a nonprofit they founded in 1998 to support Alzheimer’s research. The gift is the largest ADDF has received.

“We’ve deployed about $270 million so far for about 700 programs in 19 countries of drug discovery and development over the past 25 years,” said Dr. Howard Fillit, co-founder and chief science officer of the nonprofit.

The Lauder gift will sustain ADDF’s philanthropic model for the next 10 or 15 years, Fillit said. “We want to use our donors’ money solely for the advancement of the development of new drugs for Alzheimer’s disease,” he explained. The organization ensures that every cent of each dollar donated goes towards drug research, with no deductions for administrative costs, salaries, rent…

AbbVie and Janssen voluntarily withdraw Imbruvica from accelerated approvals for MCL and MZL

AbbVie (NYSE:ABBV) and Janssen Pharmaceutical (NYSE:JNJ) have revealed their intent to voluntarily withdraw the accelerated approvals for Imbruvica (ibrutinib) for patients with mantle cell lymphoma (MCL) and marginal zone lymphoma (MZL) in the U.S.

Ibrutinib is a selective Bruton’s tyrosine kinase (BTK).

The main reasons for the move relate to FDA’s request for additional studies to confirm clinical benefits that relate to the accelerated approval status granted by the FDA for MCL and MZL. Initially, FDA authorized the accelerated approval program for the two indications following the publication of positive overall response rate data in phase 2 trials.

While the phase 3 shine study in previously untreated MCL met its primary endpoint of progression-free survival, there was an increased rate of adverse reactions when combining Imbruvica and chemotherapy compared to the placebo-controlled arm.

Conversely, the phase 3 SELENE study in re…