BioPharma Drive: Drug Pricing

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

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Science RNA Therapies Disease 59

Science Daily: Pharmacology News

JULY 5, 2023

An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome.

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Protein Production Production Therapies RNA 75

Drug Target Review

MARCH 21, 2024

There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. AAV-based therapies often can’t be dosed more than once due to concerns about an immune response. Of note, mRNA is redosable as opposed to AAV. Human molecular genetics.

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Therapies RNA Immune Response Treatment 64

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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Engineering Research Virus DNA 144

Broad Institute

JUNE 28, 2023

Today, scientists use these reactions to produce customizable DNA and RNA molecules that enable genetic sequencing, drug and vaccine development, pathogen tests, cancer diagnostics, and many aspects of basic biomedical research. “I Longer synthetic DNA and RNA molecules are also critical for modern biologic drugs.

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DNA RNA Science Therapies 98

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. and Virginia W.

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Drug Target Review

APRIL 26, 2023

Scientists from the Massachusetts Institute of Technology (MIT) and the University of Massachusetts Medical School (UMass), US, have collaborated to create a novel type of nanoparticle that can deliver messenger RNA that encodes for beneficial proteins to the lungs. The study appears in Nature Biotechnology.

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Science Daily: Pharmacology News

JULY 27, 2023

If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant.

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RNA Research Therapies Disease 72

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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DNA RNA Disease Hospitals 128

Codon

APRIL 9, 2023

“Computer science is to biology what calculus is to physics.” ” — Harold Morowitz 🔥 Ten Amazing Things (that happened this week…) A CAR-T therapy was tested in 27 children with neuroblastomas. The RNA strands shut down a specific gene in the insects, and killed half after 7 days. Shrock E.L.

Therapies 52

Therapies RNA Engineering Vaccine 52

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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Hospitals Treatment Medical Schools Science 121

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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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. Along the way, TIDE is showing how careful, systematic science at scale can quickly home in on promising drug targets.

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Research Immune Response Clinical Trials Therapies 137

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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Therapies Research Clinical Trials Trials 52

Codon

JULY 27, 2023

From an article in Science : “After 6 months, the most promising varieties had their lignin content reduced by 49.1% in Science. But what happens if you restore these cone cells, using gene therapy? approved a gene therapy for hemophilia A for the first time. of which had reduced lignin levels and extra cellulose.

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Therapies RNA DNA Engineering 52

Codon

JULY 27, 2023

From an article in Science : “After 6 months, the most promising varieties had their lignin content reduced by 49.1% in Science. But what happens if you restore these cone cells, using gene therapy? approved a gene therapy for hemophilia A for the first time. of which had reduced lignin levels and extra cellulose.

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Therapies RNA DNA Engineering 52

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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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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Licensing Licensing Therapies Disease 52

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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Small Molecule Therapies Regulations Treatment 105

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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Therapies Laboratories Molecular Biology RNA 52

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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Science Clinical Development Treatment DNA 105

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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RNA Drugs Research Science 52

Broad Institute

JANUARY 30, 2024

More bottles on the medicine shelf would help, but Bhattacharyya, who’s now also a research scientist, realized that science might lead to better tools for diagnosing and treating such challenging cases. FROM STARS TO CELLS Bhattacharyya’s interest in science began not in the lab, but in the sky.

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Hospitals Medical Schools Virus Science 137

Drug Target Review

SEPTEMBER 4, 2023

I had earlier proposed the use of Palmatine and Silver Nitrate in the efficacy against the RNA structure of the COVID-19 virus. 7,9 Dihydrofolate reductase-thymidylate synthase (DHFR) have been successfully exploited for the therapy of bacterial infections and other parasitic diseases such as malaria. References: Antunes Luisa (2023).

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Drugs RNA DNA Disease 111

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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RNA Clinical Trials Disease Science 98

KIF1A

AUGUST 20, 2024

Susannah is being treated with an Antisense Oligonucleotide (ASO) : To break down the term, this is a molecule designed with a sequence of 20 (oligo, “few”) letters of DNA (nucleotide) that match a sequence on the mutant KIF1A RNA (Antisense) and knock it down. Will this _ KAND? With gratitude, in the Year of Pioneers.

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Science Treatment Therapies Small Molecule 52

PLOS: DNA Science

MARCH 14, 2024

Biotechnology has solved many problems, from recombinant DNA and monoclonal antibody-derived drugs, to gene therapy and stem cell transplants, to RNA-based vaccines and genetically modified plants that resist diseases and pesticides. Let Them Eat Snake appeared first on DNA Science. And yes, snake meat tastes like chicken.

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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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Treatment RNA DNA Science 52

Broad Institute

MAY 2, 2024

But as an undergraduate at MIT, Martin found she could study biology and computer science and still row. In 2018, she graduated with a bachelor’s degree in computer science and biology and joined the lab of Gad Getz at the Broad Institute of MIT and Harvard to complete her master’s. The act of discovery always appealed to me.

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Science Medical Schools Disease Hospitals 124

Drug Target Review

AUGUST 9, 2023

14 TDP-43 is a DNA and RNA binding protein that regulates the expression and splicing of several target transcripts. Many potential toxic effects have been suggested, including the mis-splicing of the Stathmin-2 RNA transcript. ALS Therapy Development Institute, www.als.net/news/frontotemporal-dementia-vs-als/. Science vol.

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RNA Regulations Disease DNA 98

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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Small Molecule Targeted Protein Degradation Science Disease 115

The Pharma Data

JULY 29, 2021

Gilead Sciences, Inc. Our flagship HIV therapy, Biktarvy, saw continued growth and gains in market share, despite the ongoing impact of the pandemic,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences. Biktarvy Sales Increased 24% Year-Over-Year. Key Updates Since Our Last Quarterly Release.

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Science Production Treatment FDA 52

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.

Small Molecule 137

Small Molecule Trials Therapies Disease 137

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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DNA Virus Research Engineering 140

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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DNA Organic Chemistry Science Virus 116

Codon

MARCH 20, 2023

Signal Transduction and Targeted Therapy. Basic Science A trailing ribosome speeds up RNA polymerase at the expense of transcript fidelity via force and allostery. Science Advances. Science Advances. Ribozyme-mediated RNA synthesis and replication in a model Hadean microenvironment. O’Neill P.

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Virus Engineering RNA Small Molecule 52

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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Clinical Trials Science Molecular Biology Bioinformatics 59