Bio Pharma Dive

AUGUST 6, 2024

Through a new deal, Roche has exclusive rights to Sangamo molecules designed to repress the gene that makes “tau,” a protein many scientists view as a main driver of Alzheimer’s.

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Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.

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Bio Pharma Dive

FEBRUARY 16, 2023

The company’s platform technology is built on research from Zhang’s lab at the Broad Institute, which focuses on a proteins that could be used to package and deliver nucleic acids.

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Bio Pharma Dive

JUNE 23, 2022

After one year, researchers detected important protein changes in patients who received a low dose of the experimental treatment. Further testing and functional data are needed to assess its potential, however.

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Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

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Pharmaceutical Technology

MARCH 10, 2023

On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

JULY 7, 2022

For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases. A new frontier in cancer research. Cold chain logistics, meanwhile, have never been so important.

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pharmaphorum

AUGUST 5, 2021

Protein-protein interactions (PPIs) are becoming increasingly relevant in the pathology of many diseases, including cancer. PPIs are an integral part of the physiology of living organisms, as complexes which control biological pathways mediated by proteins. These regions are critical for optimal interactions between proteins.

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XTalks

JANUARY 6, 2025

CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. The CFTR gene itself is complex, with over 2,000 known mutations. Alyftrek targets the root cause of CF by enhancing CFTR protein function.

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Bio Pharma Dive

APRIL 27, 2023

Partner Regeneron said the data, which showed substantial reduction of a key protein, suggests hope for the “bold dream” of silencing genes in the brain.

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Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. The therapy is specifically aimed at treating FTD patients who have mutations in their granulin gene, which encodes for the protein progranulin (PGRN). This protein promotes lysosomal function.

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Pharmaceutical Technology

DECEMBER 22, 2023

Discover Amicus Therapeutics' groundbreaking gene therapy vectors and methods of use. Enhance protein expression and cellular targeting for effective treatment of genetic disorders. Explore the patent claim now!

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Scienmag

FEBRUARY 10, 2022

Soybeans outmatch all other legumes as the protein powerhouses of the plant kingdom, providing a key protein source for humans and livestock around the world. And now, after 30 years, University of Illinois scientists have identified the gene with the largest impact on seed protein in soybean. URBANA, Ill.

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pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. The exact mechanism depends on the disease in question.

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Scienmag

MARCH 4, 2022

By combining CRISPR technology with a protein designed with artificial intelligence, it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. Researchers from the University of Washington School of Medicine in Seattle describe this finding in the journal Cell Reports.

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Protein Gene Medicine Research 83

Pharmaceutical Technology

JUNE 15, 2023

Japan-based Astellas Pharma has entered a partnership with Cullgen to discover and develop multiple targeted protein degraders through the latter’s proprietary uSMITE targeted protein degradation platform, in a deal potentially worth $1.9bn. This method can target enzymes, transcription factors, scaffold proteins and more.

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Pharmaceutical Technology

JANUARY 19, 2023

AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4. This DUX4 protein abnormal expression leads to modifications in gene expression in muscle cells which are associated with progressive muscle function loss in FSHD patients.

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Pharmaceutical Technology

MAY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Oncolytic viral proteins. Sillajen and Advaxis are the other key patent filers of oncolytic viral proteins.

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Scienmag

NOVEMBER 11, 2020

Credit: Masato Kanemaki Researchers can now more accurately and precisely target specific proteins in yeast, mammalian cells and mice to study how knocking down specific protein traits can influence physical manifestation in a cell or organism. “Conditional gene knockout and small interfering RNA […].

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Pharmaceutical Technology

MAY 25, 2023

Pushing back an initial deadline, the US Food and Drug Administration (FDA) has proposed a new regulatory action date of 22 June, by which time the agency will assess the logistics of a possible approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy.

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Pharmaceutical Technology

OCTOBER 18, 2022

Last week, Nature magazine published a paper detailing an in-situ study of Parkinson’s disease (PD), showing protein structural changes in cerebrospinal fluid (CSF) between healthy individuals and Parkinson’s patients. So, the next logical step is to see what these genes do and try to correct deficit using drugs”, says Fasano.

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pharmaphorum

MARCH 19, 2021

Gain Therapeutics has priced its initial public offering, raising more than $40 million for its computational drug discovery platform and pipeline of drugs for diseases associated with protein misfolding. . The post Protein misfolding specialist Gain nets $40m from IPO appeared first on. and valuing the company at around $130 million.

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Pharmaceutical Technology

APRIL 13, 2023

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

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XTalks

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. For example, when T cells (a type of white blood cells that fight off infections and cancer) are activated in our immune systems, thousands of proteins in these cells change. Using CRISPR Technology to Disrupt Networks of Immune Genes.

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Pharmaceutical Technology

JUNE 8, 2023

GenScript ProBio has collaborated with the New York Blood Center Enterprises’ business unit, called Comprehensive Cell Solutions (CCS), to accelerate the development and manufacture of cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Pharmaceutical Technology

AUGUST 11, 2022

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

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pharmaphorum

AUGUST 23, 2022

France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease.

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Medical Xpress

DECEMBER 8, 2022

Researchers at the Johns Hopkins Kimmel Cancer Center have revealed how the gene HOXA5 may work to suppress formation of breast cancers. A new study indicates that it binds to another protein in an inflammatory cell pathway, boosting that protein's cancer-suppressing properties.

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pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. Regardless of cohort, eligible participants are scheduled to receive the investigational gene therapy, either at the start of the study or after one year following treatment with placebo.

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Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Gene splicing using nucleases. They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins.

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Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions.

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XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein.

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Gene Therapy Gene Protein Trials 110

Scienmag

JULY 7, 2022

A collaboration including researchers from Kobe University, University of Tokyo and Tohoku University has successfully identified and disrupted genes in the yeast Pichia pastoris (1) in order to increase its secretory production of useful proteins (2).

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pharmaphorum

DECEMBER 2, 2021

Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

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BioPharma Reporter

OCTOBER 22, 2020

Scientists have used gene therapy and a newly developed light-sensing protein to restore sight in mice: with the technology to start clinical trials in the US later this year.

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Scienmag

SEPTEMBER 22, 2020

Brazilian researchers observed that in uninfected adipocytes, the hormone irisin altered the expression of genes that regulate ACE-2, which encodes a protein to which the virus binds in order to invade human cells.

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