Dark Matter Blog

AUGUST 27, 2020

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. In fact, it was these early pharmaceutical successes that gave us the confidence that we would ultimately succeed in systematically drugging a wide range of RNA structures.

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Codon

AUGUST 26, 2024

Credit: OpenStax College The innate immune system has been evolutionarily successful for three reasons: its tools target the most fundamental properties of viruses , act as prophylactics —meaning they prevent diseases, rather than treat them—and are widespread. Existing medicines only pose a partial solution.

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The Pharma Data

FEBRUARY 8, 2021

The amended protocol being implemented for all ongoing adult and adolescent fitusiran clinical studies was presented today at the 14 th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD). We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.

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Drug Target Review

JULY 24, 2023

Through phylogenetic analysis and microscopy techniques, they identified a nuclear-encoded apicoplast RNA polymerase σ subunit called ApSigma. The study suggests that this regulatory system could be a potential target for future malaria treatments, offering hope for combatting this deadly disease that affects millions worldwide.

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Chemical Biology and Drug Design

SEPTEMBER 15, 2023

Abstract Chronic rhinosinusitis (CRS) is an inflammatory disease of paranasal sinuses. CRS rabbit models were constructed by Staphylococcus aureus infection and rabbits were injected with lentiviral vectors of short hairpin RNA-targeting Chemerin (shChemerin), followed by micro-computed tomography (CT) scan.

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The Pharma Data

SEPTEMBER 6, 2021

First data to be presented from the phase II coopERA Breast Cancer study evaluating neoadjuvant giredestrant treatment for oestrogen receptor (ER)-positive, HER2-negative breast cancer. Breast Cancer Highlights. Lung Cancer Highlights.

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Drug Target Review

APRIL 9, 2024

How has nucleolar stress (NS) been linked to age-related diseases, and what approach did researchers use to investigate its toxicity? The nucleolus (and nucleolar stress) had been previously linked to aging and age-related diseases such as cancer or degeneration. However, the mechanism of this observation was not fully understood.

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KIF1A

OCTOBER 15, 2024

In February 2021, genetic testing revealed the name of my disease: KAND (KIF1A Associated Neurological Disorder), of which autism is one of the frequent symptoms. They say I was very sweet, but I didn’t try to stand up, walk, or point at what I wanted. Shortly after, I was diagnosed with autism. It was an incredible experience!

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The Pharma Data

MARCH 8, 2021

The data were presented at the 28th Conference on Retroviruses and Opportunistic Infections ( virtual CROI 2021). In patients with chronic kidney disease, assess serum phosphorus. In patients with chronic kidney disease, assess serum phosphorus. Pregnancy and lactation. Source link:[link].

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Broad Institute

AUGUST 19, 2024

Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. Jude’s, the team identified a group of genes involved in processing uLCFAs that are present only in the non- Lactobacillus iners species. At the Ragon Institute, Kwon’s lab is working to move this research toward a human clinical trial. “We

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KIF1A

SEPTEMBER 16, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. This is also important when newborns present as having a “common” disorder that may have a rare genetic component, like KIF1A in genetic cerebral palsy.

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DrugBank

MAY 22, 2024

In the dynamic world of drug discovery, the notion of "undruggable" targets presents both a significant challenge and an intriguing frontier for researchers and pharmaceutical companies. Beyond Proteins: DNA and RNA Frontier The story doesn’t end with proteins.

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Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. This approach can be highly targeted, with the potential to correct mutations that lead to specific diseases or deliver therapeutic genes to improve heart function.

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The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. The German study is also scheduled for presentation on Saturday at the American Society of Hematology’s virtual annual meeting.

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Drug Target Review

MAY 22, 2024

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context.

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DrugBank

JULY 24, 2024

This is a game-changer, especially in the fight against cancer and other complex diseases. The versatility and potential of PROTACs are vast, paving the way for new therapies that could transform how we treat diseases. This can potentially correct the root causes of some diseases at the genetic level.

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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. A Scant Protein with Outsize Importance Slowing the muscle decline of DMD presents two huge challenges: the ubiquity of muscle and a giant gene. The gene presents a complex landscape.

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Drug Target Review

SEPTEMBER 25, 2024

5 Here, the original oncogene is still present but the transdifferentiated cancer cells are less dependent on it. Investigating non-oncogene resistance as a parallel defense mechanism used by cancer cells to evade therapies could therefore present a significant opportunity to address drug resistance more comprehensively.

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The Pharma Data

MARCH 8, 2021

These preliminary findings were presented today during Science Spotlights TM at the 2021 Conference on Retroviruses and Opportunistic Infections (CROI 2021). Findings from the primary efficacy and safety endpoints and additional secondary objectives will be presented at an upcoming medical meeting.

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PLOS: DNA Science

JUNE 13, 2024

But the list leaves out the most likely scenario, which is where Chan’s discussion begins: the virus was present, or arose, more than a decade ago in a mineshaft in Yunnan Province in China and/or a cave in Laos – as a virus called RaTG13. ” So ended my 2021 blog post a month later. .” b) in an abandoned mine shaft?

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Drug Target Review

NOVEMBER 13, 2023

Regulatory T cells (Treg) cells are specialised immune cells that are essential for maintaining peripheral tolerance, preventing autoimmune diseases, and limiting chronic inflammatory diseases. However, they also limit beneficial responses by suppressing sterilising immunity and limiting antitumour immunity.

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The Pharma Data

JULY 12, 2021

Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Ridgeback Biotherapeutics announced today the presentation of previously announced Phase 2 interim results from two Phase 2/3 clinical trials (MOVe-OUT and MOVe-IN) of molnupiravir (MK-4482/EIDD-2801), an investigational oral antiviral therapeutic. About Molnupiravir.

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The Pharma Data

JULY 6, 2021

New research from first-in-class marketed and investigational therapies in hemophilia, immune thrombocytopenia and acquired thrombotic thrombocytopenic purpura will be presented. Oral presentation. These data support the revised fitusiran dose and dosing regimen implemented in the ongoing adult and adolescent studies.

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The Pharma Data

MARCH 8, 2022

Also, the incidence of severe disease progression was observed to be significantly reduced. Proteins of SARS-CoV-2 can alter viral ribonucleic acids in a way, that they become indistinguishable from endogenous RNA. For example, viral RNAs are masked by the addition of a methyl group. Camouflage protects virus from immune system.

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Broad Institute

OCTOBER 4, 2023

Today, scientists use such reactions to produce the customizable DNA and RNA molecules that enable genetic sequencing, drug and vaccine development, pathogen tests, cancer diagnostics, and more. Wang concluded her presentation by expressing gratitude to Merkin. I’m really thankful for the support of the Merkin Fellowship,” she said. “It

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The Pharma Data

MARCH 28, 2022

We are incredibly grateful for the selfless commitment of the individuals with ALS who participated in the study, and the community’s dedication to advancing research for this devastating disease,” said Toby Ferguson, M.D., The companies will present the BIIB078 Phase 1 data at a future scientific forum. Biogen Inc.

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The Pharma Data

APRIL 22, 2023

Nasdaq: GILD) today announced positive results from several COVID-19 clinical and real-world evidence studies being presented at the 33rd European Congress of Clinical Microbiology & Infectious Diseases (ECCMID). Patients were randomized 2:1 to receive Veklury (n=163) or placebo (n=80), in addition to standard of care.

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The Pharma Data

MAY 31, 2022

Amgen (NASDAQ:AMGN) today announced positive topline data from the Phase 2 OCEAN(a)-DOSE clinical study, evaluating olpasiran (formerly AMG 890) in 281 adult patients with Lipoprotein(a), or Lp(a), levels over 150 nmol/L and evidence of atherosclerotic cardiovascular disease (ASCVD). About Lp(a). About OCEAN(a).

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The Pharma Data

AUGUST 30, 2021

Separate post hoc analyses of pooled Phase III ORION-9, -10 and -11 data show twice-yearly** Leqvio ® (inclisiran) consistently reduced low-density lipoprotein cholesterol (LDL-C) in patients with atherosclerotic cardiovascular disease (ASCVD) with established cerebrovascular disease (CeVD) 1 and polyvascular disease (PVD) 2.

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The Pharma Data

DECEMBER 8, 2020

The company’s commercial priorities are three-fold: (1) Initially focusing its commercial efforts on rare diseases within its prolific neurology and cardiology franchises (2) pioneer new markets where there are no available treatments (3) create new standards of care where there has been a lack of innovation to optimize patient care.

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The Pharma Data

DECEMBER 8, 2020

Overall, the vaccine protected against symptomatic disease in 70% of cases, according to a team led by researchers from Oxford University in England. Both employ a newer technology that uses messenger RNA to prime the immune system to attack the new coronavirus. TUESDAY, Dec. 8 in The Lancet.

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The Pharma Data

AUGUST 16, 2021

The CureVac-GSK COVID-19 collaboration announced in February 2021 extends the existing strategic mRNA technology partnership both companies started in July 2020, which focuses on the development of new products based on CureVac’s second-generation RNA-technology for different targets in the field of infectious diseases.

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Conversations in Drug Development Trends

MAY 30, 2024

The peak number of trials in the last five years and the regulatory approvals of three additional disease-modifying treatments for ALS in the previous eight years signal cause for hope that, as a community, we have the potential to transform this progressive and ultimately terminal neurodegenerative disorder into a treatable condition.

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The Pharma Data

DECEMBER 16, 2020

Throughout the day, various presentations will be given by medical experts pertaining to Emergency Use Authorization (EUA). The Moderna vaccine is similar to Pfizer and BioNTech’s vaccine in the sense that they both use an approach utilizing messenger RNA (mRNA). The meeting is slated to run from 9 a.m. ET to 5:15 p.m.

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The Pharma Data

JUNE 7, 2023

These late-breaking data are being presented for the first time today at 5:00 p.m. ET during an oral abstract session at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting (abstract #LBA9503). “We

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Advarra

MARCH 31, 2023

IBC review ensures compliance with biosafety guidelines, issued by the National Institutes of Health (NIH) as well as the Centers for Disease Control and Prevention (CDC) , intended to protect the health and safety of research personnel as well as the community and environment around the research site.

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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. In this regard, AI presents itself as a valuable ally, offering the potential to bolster our endeavours and ensure successful outcomes.

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