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. We have overcome many obstacles on this mission, inventing new concepts and methods where necessary and re-engineering known concepts and methods where possible. We will replace it.”

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The Open Targets Blog

AUGUST 15, 2023

Research in mice has shown that RBM3 expression is protective against misfolded protein damage, suggesting the protein as an interesting target in the development of therapies for neurodegenerative conditions such as Alzheimer’s disease. Curiously, these followed a theme in molecular function: the regulation of splicing*.

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The Pharma Data

DECEMBER 8, 2020

Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. The engineered cells are protected by Sigilon’s Afibromer biomaterials matrix, which shields them from immune rejection and fibrosis.

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Broad Institute

JANUARY 8, 2024

A team of researchers at the Broad Institute of MIT and Harvard has engineered virus-like particles to deliver prime editors to cells in mice at a high enough efficiency to rescue a genetic disorder. They also delivered prime editors to the mouse brain, and did not detect any off-target editing.

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Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. Credit: Neumann EN, Bertozzi TM, et al.

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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. Sanofi’s two marketed extended half-life factor replacement therapies shifted a two-decades-old treatment paradigm when launched in 2014. ePoster.

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The Pharma Data

MAY 31, 2022

Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, and Takeda ( TSE:4502/NYSE:TAK ) announced to transfer the marketing authorization in Japan for Moderna’s COVID-19 vaccine Spikevax TM (mRNA-1273) from Takeda to Moderna in Japan as of August 1, 2022. Moderna, Inc., About Moderna.

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Broad Institute

MAY 16, 2024

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

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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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The Pharma Data

OCTOBER 19, 2020

. “Innovation in cancer treatment is realized through collaboration, and the physicians and researchers at Emory’s Winship Cancer Institute have consistently shown their commitment to developing novel cancer therapies using this collaborative approach,” said Chadi Nabhan , M.D., Ramalingam , M.D.,

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Dark Matter Blog

APRIL 8, 2020

Most people, and in particular most investors we spoke to in those early days, thought we were nuts – that RNA lacked the structural and molecular complexity that medicinal chemistry exploits with such great effect for proteins. One is that molecular recognition is purely a matter of physics and RNA has to play by the same rules.

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The Pharma Data

JANUARY 12, 2021

is a clinical-stage immunotherapy company dedicated to transforming the treatment of cancer by developing therapies that enable the immune system to attack tumors and provide long-lasting benefits to patients through a biomarker-driven approach. About Jounce Therapeutics Jounce Therapeutics, Inc.

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The Pharma Data

JANUARY 10, 2021

FBX-101 is a first-in-human AAV gene therapy. ILP100 is a genetically engineered Lactobacillus that acts as a small bioreactor on site in the wound, continuously introducing a chemokine, CXCL12, into the wound tissue. The drug is an RNA interference (RNAi) therapeutic. Most Read Today. Source link.

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Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? But for a recent paper out of Nanjing, China, researchers engineered another enzyme and showed that, when mixed with FAST-PETase, the two worked together to break down plastic twice as fast as the Texas enzyme alone. LEGO robot to make sucrose gradients.

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Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? But for a recent paper out of Nanjing, China, researchers engineered another enzyme and showed that, when mixed with FAST-PETase, the two worked together to break down plastic twice as fast as the Texas enzyme alone. LEGO robot to make sucrose gradients.

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The Pharma Data

MARCH 23, 2022

In fact, antiviral therapies designed to treat viruses such as hepatitis C (Sofosbuvir/SOVALDI) work by blocking viral polymerase. Back in 2018, researchers tested a broad-spectrum antiviral candidate called remdesivir/VEKLURY, which acts as a nucleotide decoy to get incorporated into the viral RNA genome and stop viral polymerase.

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The Pharma Data

DECEMBER 10, 2020

ImmunityBio has engineered the vector to overcome this problem and has shown that its second-generation adenovirus vector can safely and effectively deliver its cargo even in patients with pre-existing adenovirus immunity. This can significantly limit the effectiveness of first-generation platforms. hAd5-COVID-19 Oral Formulation.

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Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. iPSC-derived lymphocytes, eg, T cells and natural killer (NK) cells, engineered to express targeting molecules such as chimeric antigen receptors (CARs) have shown clinical promise to treat haematological malignancies.

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The Pharma Data

JANUARY 17, 2021

That’s not the case with RNA-targeted drugs. Traditional companies have neither the technologies nor the financial incentives needed to develop ultra-rare therapies. Working closely with Ionis, “We connect the need to the drug development engine. Crooke, therefore, created n-Lorem Foundation to do that charitably.

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Codon

APRIL 9, 2023

” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. LanzaTech announced a partnership with H&M Move to convert factory carbon emissions into fabrics using engineered microbes. Support for $5 per month. Benz F & Hall A.R.

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Drug Target Review

JANUARY 2, 2024

Through a series of experiments, they successfully developed a personalised stem cell therapy using a data-driven, single-cell technique based on swift subcellular proteomic imaging. Our recent work proposes the use of an intracellular toolkit to map organelle bio-geography in stem cells that could lead to more precise therapies.”

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Drug Target Review

JULY 7, 2023

This gives epigenetic editing the unique ability to treat complex multigenic diseases and opens the door to enhancing cell and regenerative therapies, which require the manipulation of multiple genes for optimal results. She is currently focused on using genetic tuning to enhance and develop cell and gene therapy products for clinical impact.

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Codon

APRIL 30, 2023

Read more at Nature Biotechnology ( Similar paper at Nature Biomedical Engineering.) But now, their engineered brethren can live about 82 percent longer. Often, when we engineer a cell, we only mess with the magnitude of an effect; this study suggests that we should think more about the timing, too. From Zhang et al.

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Codon

APRIL 30, 2023

Read more at Nature Biotechnology ( Similar paper at Nature Biomedical Engineering.) But now, their engineered brethren can live about 82 percent longer. Often, when we engineer a cell, we only mess with the magnitude of an effect; this study suggests that we should think more about the timing, too. From Zhang et al.

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Broad Institute

MARCH 22, 2024

Messenger RNAs with multiple “tails” could lead to more effective therapeutics By Corie Lok March 22, 2024 Breadcrumb Home Messenger RNAs with multiple “tails” could lead to more effective therapeutics Scientists have engineered long lasting mRNAs that increased therapeutic protein production in cells and animals.

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Codon

APRIL 2, 2023

This is Codon Digest, a weekly roundup of research papers, news articles, and industry highlights about engineered biology. Read Biological Engineering mScarlet3: a brilliant and fast-maturing red fluorescent protein. Thanks for skimming. ❤️ Subscribe to support human writers. Gungabeesoon J. Nature Aging. Nature Methods.

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Codon

SEPTEMBER 29, 2024

But, regardless of which was first, they all operated with the same core data as their mechanism for understanding life: messenger RNA ( mRNA ). It suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life.

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The Pharma Data

MARCH 4, 2021

We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. For more information on Ovid, please visit www.ovidrx.com. .

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Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% As more therapies are moving out of the lab and into human clinical trials, this paradigm is changing.

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Codon

MARCH 20, 2023

🧪 Papers AI + Bio Protein-specific signal peptides for mammalian vector engineering. Signal Transduction and Targeted Therapy. Basic Science A trailing ribosome speeds up RNA polymerase at the expense of transcript fidelity via force and allostery. Metabolic Engineering. Metabolic Engineering. Szymczak P.

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Codon

MAY 30, 2023

Codon Digest is my weekly roundup of research, news, and industry highlights about engineered biology. An engineered version of this protein can convert DNA bases with efficiencies up to 92%. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome.

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DNA RNA Engineering Vaccine 52

Codon

MAY 30, 2023

Codon Digest is my weekly roundup of research, news, and industry highlights about engineered biology. An engineered version of this protein can convert DNA bases with efficiencies up to 92%. A particular variant, named IscB*-ωRNA*, had the highest editing efficiency across multiple different sites in the genome.

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DNA RNA Engineering Vaccine 52

Broad Institute

OCTOBER 11, 2023

But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way. These cells also express the Cas9 protein, which helps disable the genes targeted by the guide RNAs.

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Codon

JUNE 11, 2024

Their solution was to fuse hairpins, little loops made from RNA or DNA, at various positions along the DNA strand that was being sequenced. 3 Nanopore engineers decided to cleverly exploit the natural speed limit of DNA polymerase to slow down DNA translocation through the pores. Cite: Stephen Malina. “Driving Toward Nanopores.”

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The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.

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The Pharma Data

SEPTEMBER 8, 2020

About AstraZeneca AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology.

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Vial

DECEMBER 5, 2023

In 2017, the Food & Drug Administration (FDA) approved the first gene therapy for cancer and for inherited diseases, the first multiplex NGS panel for companion diagnostics (CDx), and the first drug targeting a genetic signature though not a disease. Fountzilas et al. Let’s Connect.

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