Drug Target Review

JUNE 15, 2023

In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.

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Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. These fields explore highly precise biological processes related to RNA, specific proteins and gene expression mechanisms.

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Elrig

MARCH 25, 2025

This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).

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Drug Target Review

DECEMBER 6, 2024

This includes verifying the experimental design and understanding how the data was generated whether it was from RNA sequencing, mass spectrometry, or other biological assays. About the authors Dr Raminderpal Singh Dr Raminderpal Singh is a recognised visionary in the implementation of AI across technology and science-focused industries.

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Broad Institute

JANUARY 8, 2024

Now the researchers describe how they re-engineered both eVLPs and parts of the prime editing protein and RNA machinery to boost editing efficiency up to 170 times in human cells compared to the previous eVLPs that deliver base editors. DOI: 10.1038/s41587-023-02078-y Tags: Liu Lab Gene editing Rare Disease Gene-based therapy delivery

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Broad Institute

OCTOBER 23, 2024

By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.

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Broad Institute

AUGUST 19, 2024

I’m so proud of this team and the partnership we formed to follow the science and set up some major positive impacts on women’s health,” Blainey said. Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. Paper cited Zhu M et al.

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The Pharma Data

SEPTEMBER 28, 2020

This included compiling RNA data, as well as other data points such as patient demographics, clinical treatment arm and patient diagnosis. However, for others, it did not work – or did so only marginally, or with serious side effects,” said Stuart Henderson, global Life Sciences lead for Accenture.

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The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets. About Novartis.

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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KIF1A

JULY 29, 2023

KIF1A in New York: The 2023 KAND Family & Scientific Engagement Conference We won’t be posting next Saturday, but not for a lack of science! Genetic and RNA-based therapies seek to correct mutant KIF1A – improving its force generation, velocity, and distance traveled by increasing the ratio of healthy to mutant KIF1A.

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. But even if gene therapy could reach sufficient muscle cells to improve quality of life, another hurdle looms: the dystrophin gene that’s deleted in DMD is huge, the largest in a human genome at 2.2

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Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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The Pharma Data

NOVEMBER 17, 2021

Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma. doi: 10.1126/sciadv.abg9551. Source link: [link].

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Drug Target Review

APRIL 3, 2024

Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. Being an only child of non-academic parents, I learned to work and study independently early on.

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Drug Target Review

JULY 23, 2024

I studied Natural Sciences at the University of Cambridge, where I was exposed to a range of disciplines from chemistry to physiology, specialising in my last year in biochemistry. Pushing the boundaries of science always opens the door to new, impactful developments, although there are always challenges along the way.

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DrugBank

MAY 15, 2024

This method was more about serendipity than science. Another promising avenue is the use of technologies like RNA interference and gene editing, which allow scientists to turn off the production of certain proteins altogether. But as molecular biology has advanced, so too has our approach to finding new drugs.

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The Pharma Data

OCTOBER 21, 2020

In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.

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Broad Institute

DECEMBER 13, 2023

The studies are from groups at the Broad, Allen Institute for Brain Science, the Salk Institute for Biological Studies, and other institutions that are part of the National Institutes of Health’s Brain Research Through Advancing Innovative Neurotechnologies ® Initiative, or The BRAIN Initiative — Cell Census Network ( BICCN ). and Virginia W.

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Drug Target Review

SEPTEMBER 28, 2023

They had the science and the technology but not the business model. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays. Often, DNA technology is used in the development of new therapies. Twist was officially founded in 2013. At that time synesthetic biology wasn’t cool.

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Drug Target Review

MAY 27, 2024

Caris Life Sciences® (Caris) has been at the forefront of precision medicine. Caris Discovery, the therapeutic research arm of Caris Life Sciences, was launched to address the paucity of novel oncology drug targets and is uniquely poised to discover first-in-class ADC targets.

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. TfR1 is almost becoming table stakes for Pharma and biotech looking to deliver to the brain and / or muscle.

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The Pharma Data

DECEMBER 8, 2020

December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. BioAge is on the cusp of taking pilot therapies BGE-117 and BGE-175 into clinical trials, targeting the first half of 2021.

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Dark Matter Blog

JULY 19, 2021

This was a great gig – good people, good science. However, in June I happened to attend the Gordon Research Conference on Chemical Biology and High-throughput Chemistry where I saw a session on small molecules and RNA. Making small-molecule drugs against RNA structures looked like a really cool problem – I want to do THAT!

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The Pharma Data

OCTOBER 19, 2020

20, 2020 /PRNewswire/ — Caris Life Sciences® , a leading innovator in molecular science focused on fulfilling the promise of precision medicine, announced today that Winship Cancer Institute of Emory University (Winship) has joined Caris’ Precision Oncology Alliance (The Alliance/POA). . IRVING, Texas and ATLANTA , Oct.

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Drug Target Review

AUGUST 29, 2023

It’s also where I got exposed to career options beyond academia and learned about Flagship Pioneering, a venture creation firm that conceives, resources, and builds life science bio platform companies in health care and sustainability. One of my mentors asked me if I was ready to fail 90 percent of the time.

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Elrig

DECEMBER 14, 2023

Learn how RNA's role in axons opens avenues for targeted therapies reshaping neurological condition treatments. Federico Dajas-Bailador, an expert in axon biology, discusses groundbreaking research, unraveling neuronal function and the potential for treating neurological disorders.

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Dark Matter Blog

JULY 16, 2021

As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. I wanted to use molecular biology to create drugs.

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Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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The Pharma Data

JANUARY 12, 2021

JTX-8064 is the first tumor-associated macrophage candidate developed from Jounce’s Translational Science Platform. Jounce currently has multiple development stage programs ongoing while simultaneously advancing additional early-stage assets from its robust discovery engine based on its Translational Science Platform.

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The Pharma Data

MARCH 28, 2022

While these were not the results we were hoping for, they are clear and will inform future research across our broad pipeline of investigational ALS therapies. Biogen Inc. Nasdaq: BIIB) and Ionis Pharmaceuticals, Inc. Vice President and Head of the Neuromuscular Development Unit at Biogen.

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Dark Matter Blog

JULY 19, 2021

Or should I say back to retirement, as he was comfortably relaxing at home back in 2016 when Jen Petter lured him back into the fray with the siren call of developing a new class of RNA-targeted small-molecule medicines here at Arrakis. I won’t dwell on Jim’s many accomplishments here. Don’t be a stranger!

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The Pharma Data

MARCH 8, 2021

Gilead Sciences, Inc. In both studies, >98% of participants who initiated treatment with Biktarvy and remained in the study achieved and maintained an undetectable viral load (HIV-1 RNA <50 copies/mL) through four years of follow-up (n=235/237 for study 1489, n=241/243 for study 1490).

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Drug Target Review

SEPTEMBER 18, 2024

What specific cost-cutting measures can AI provide in the manufacturing process of cell and gene therapy, and how do these compare to traditional methods? The cell and gene therapy (CGT) space provides a great example of the potential of AI. AI can vastly improve cell culture, a critical step in cell therapy production.

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The Open Targets Blog

MAY 23, 2024

In a paper published today in Nature Communications , Open Targets researchers and collaborators used single-cell RNA sequencing to create a high resolution molecular map of immune cells in NSCLC tumours, to better understand the role of these cells in disease progression. This could then pave the way for subtype-specific therapies.

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The Pharma Data

NOVEMBER 22, 2020

Photo courtesy of Science Advances. Their research – the first of its kind – suggests that the system can be utilized to address cancer in animals, according to Professor Dan Peer, whose peer-reviewed research was published in the Science Advances journal. Cas-9 represents the Cas9 protein, an enzyme that cuts foreign DNA.

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The Pharma Data

JANUARY 31, 2021

Similar to other enveloped RNA viruses such as HIV, RSV and Influenza, SARS-CoV-2 is also an RNA virus that has a trimeric spike (S) protein on its viral envelope. GSK is a science-led global healthcare company with a special purpose: to help people do more, feel better, live longer. About Dynavax. About GlaxoSmithKline.

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