Clinical Research Informer

CRISPR tagging improves accuracy of model cells grown from stem cells

Scienmag

DECEMBER 1, 2020

Tagging produces detailed catalog of transcription factors key to making each cell type Credit: Gersbach Lab, Duke University DURHAM, N.C. – A team of biomedical engineers at Duke University has created a new way to turn stem cells into a desired cell type by mastering the language of gene regulatory networks.

Engineer

Engineer Gene Bioinformatics

#news #biotech CRISPR tagging improves accuracy of model cells grown from stem cells

BioTech 365

DECEMBER 1, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: CRISPR tagging improves accuracy of model cells grown from stem cells.A team of biomedical engineers at Duke University has created a new way to turn stem cells … Continue reading →

Engineer

Engineer Research

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

SEPTEMBER 30, 2022

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

Gene Therapy

Gene Therapy FDA Approval Gene Genetic Disease

First gene therapy trial for Hunter syndrome opens in Manchester, UK

Drug Discovery World

OCTOBER 31, 2023

In the combined Phase I and II clinical trial, five children under one year of age with the condition also known as mucopolysaccharidosis type II (MPS II) will be treated with autologous hematopoietic stem cell (HSC) gene therapy.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

DECEMBER 13, 2023

While Lyfgenia delivers a functional copy of hemoglobin A to replace the abnormal one in hematopoietic (blood) stem cells via a lentiviral vector, Casgevy edits the HBB gene using CRISPR technology to increase the production of fetal hemoglobin in blood stem cells. Both therapies will be available in early 2024.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

Epigenetics discovery could lead to new class of cancer drugs

Drug Discovery World

MARCH 2, 2023

New understanding of epigenetics It has been known for more than 20 years that the enzymes placing H3K4me3, a chemical tag added to DNA, are crucial for normal cell development, as well as being linked to leukaemia, breast, bowel and pancreatic cancers. The team found that H3K4me3 acts like a traffic light at a busy intersection.

Gene Expression

Gene Expression Gene DNA RNA

Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

Pharmaceutical Technology

APRIL 13, 2023

Although the findings in the report are only preliminary, they shed light at the considerations behind the high price tags of gene therapies. While stem cell transplants can cure the condition, their use is limited due to some of the risks that come with them.

Gene Therapy

Gene Therapy Gene FDA Approval Licensing

Scientists turn cancer cells into anticancer agents

Drug Discovery World

JANUARY 9, 2023

“Our team has pursued a simple idea: to take cancer cells and transform them into cancer killers and vaccines,” said corresponding author Shah, who is also Director of the Center for Stem Cell and Translational Immunotherapy and Vice Chair of research in the Department of Neurosurgery at Brigham and Women’s. .

Scientist

Scientist Gene Editing Gene Engineer

Second unanimous FDA adcomm vote boosts bluebird bio

pharmaphorum

JUNE 12, 2022

Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8 Haematopoietic stem cell transplantation (HSCT) offers a chance of a cure, but is only possible when a donor with a matching human leukocyte antigen (HLA) signature, within the correct age range, is available.

Gene Therapy

Gene Therapy Gene Marketing Trials

ICER says bluebird bio’s $2.1m gene therapy is cost-effective

pharmaphorum

APRIL 14, 2022

million price tag. Haematopoietic stem cell transplantation (HSCT) offers a chance of a cure, but is only possible when a donor with a matching human leukocyte antigen (HLA) signature, within the correct age range, is available.

Gene Therapy

Gene Therapy Gene Marketing Clinical Trials

AI-designed protein awakens silenced genes, one by one

The Pharma Data

MARCH 7, 2022

The approach will allow researchers to understand the role individual genes play in normal cell growth and development, in aging, and in such diseases as cancer, said Shiri Levy, a postdoctoral fellow in UW Institute for Stem Cell and Regenerative Medicine (ISCRM) and the lead author of the paper. Cas9 binds and uses RNA as an address-tag.

Protein

Protein Gene Genome Genomics

Beam makes USD 120M bet; GRAIL and Quest announce the collaboration; Brain organoids mimic infant’s brains; Improvement in T cells to kill cancer

Delveinsight

FEBRUARY 25, 2021

A new study from UCLA and Stanford University researchers observes that three-dimensional human stem cell-derived brain organoids can mature in a strikingly similar way to human brain development. Overall, this could result in a 26% overall reduction in all five-year cancer deaths. Brain organoids grown in the lab mimic infant’s brains.

DNA

DNA In-Vivo Genetics Medicine

CHMP backs J&J’s myeloma bispecific Tecvayli, ahead of US decision

pharmaphorum

JULY 25, 2022

The new drug is one of two bispecifics for multiple myeloma in the late-stage pipeline of J&J’s Janssen division stemming from its longstanding and fertile collaboration with Danish biotech Genmab, along with talquetamab which binds to CD3 and GPRC5D.

Antibody

Antibody Medicine Drugs Trials

Q&A: A decade on, what’s next for CAR-T therapies?

Pharmaceutical Technology

APRIL 5, 2023

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. Currently, all approved CAR-T therapies are autologous, where T cells taken from patients are modified and then re-infused.

Gene Therapy

Gene Therapy Gene Engineer Production

bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval

pharmaphorum

AUGUST 18, 2022

Zynteglo is a customised, one-time treatment created using a patient’s own bone marrow stem cells hat are genetically modified to produce functional beta-globin, which is mutated in the disease. million price tag attached to the therapy when it was first made available in Europe, and is also well above the proposed $2.1

Gene Therapy

Gene Therapy FDA Approval Gene Clinical Trials

Clover biopharmaceuticals raises $230 M; BrainStorm after ALS; BARDA grants $5.65 M LightDeck for SARS-CoV-2 antigen test; Libtayo for first-line advanced NSCLC subset

Delveinsight

FEBRUARY 23, 2021

The company plans to use the capital to expand its pipeline of protein-based vaccines and biologic cancer therapies utilizing its innovative and proprietary Trimer-Tag technology platform. The company develops transformative biologics including vaccines and therapeutics for the world’s most debilitating diseases.

In-Vitro

In-Vitro Vaccination Vaccine Licensing

A history of Pfizer

pharmaphorum

DECEMBER 2, 2020

With fingers in every pie, ranging from small molecules to biologics in every clinical area, to stem cells and consumer goods, Pfizer will surely celebrate its 200th anniversary in as strong a position as it spent the last 160 years. For all the latest Pfizer news follow pharmaphorum’s Pfizer tag.

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Sales Production Drugs Marketing

Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia

XTalks

AUGUST 19, 2022

Zynteglo is administered as a one-time treatment, with each dose consisting of a patient’s own bone marrow stem cells engineered to make functional beta-globin through insertion of working copies of the beta-globin gene. Zynteglo’s hefty price tag of $2.8 million is in line with it being a single, one-time administered gene therapy.

Gene Therapy

Gene Therapy FDA Approval Gene Clinical Trials

bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy

pharmaphorum

FEBRUARY 16, 2021

But haematopoietic stem cell transplantation is only possible when a donor with a matching human leukocyte antigen signature, within the correct age range, is available. million in Europe.

Gene Therapy

Gene Therapy Gene Medicine Development

Bluebird Bio’s Skysona Receives FDA Approval and Becomes World’s Most Expensive Drug

XTalks

SEPTEMBER 27, 2022

Treatment options prior to Skysona were limited to allogeneic hematopoietic stem cell transplant (allo-HSCT), which has a high risk of serious complications including death, particularly in patients without a human leukocyte antigen (HLA)-matched donor. Related: Bluebird’s $2.8M

FDA Approval

FDA Approval Gene Therapy Drugs Gene

Roche’s quantitative COVID-19 antibody test; Janssen buys a gene therapy asset; Zimmer Acquires A&E Medical; Glaucoma research study: Merck chooses TriNKET cancer immunotherapy program; Ovid fails Angelman phase 3 study

Delveinsight

DECEMBER 3, 2020

There are presently no medical treatments, which can regenerate retinal cells that have atrophied, though gene therapies and stem cell therapies are the leading R&D hopes in this arena. Shinya Yamanaka discovered that the factors could alter the epigenome, and in that way modify mature cells back to a stem cell-like state.

Gene Therapy

Gene Therapy Gene Antibody Research

Spade SB-66

The Pharma Data

MAY 13, 2021

Which is one of the only ingredients ever scientifically proven to lower cortisol and block Adamts1’s conversion of stem cells into fat cells. After all, the ingredients we’ve chosen to include in Spade SB-66 are premium ingredients and come with a hefty price tag. FREE SHIPPING. 365 DAY GUARANTEE. Free Shipping. 365 DAY GUARANTEE.

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What to expect from PEGS Europe 2023: Day 3

Drug Discovery World

NOVEMBER 13, 2023

Matthias Peipp, PhD, Research Head & Mildred Scheel Professor, Stem Cell Transplantation & Immunotherapy, University of Kiel, on: ‘Activating NK cell receptors as trigger molecules for bispecific antibodies to enhance anti-tumour NK cell responses’.

Protein

Protein Antibody Engineer Scientist

What to expect from PEGS Europe 2023: Day 2

Drug Discovery World

NOVEMBER 9, 2023

Thomas Valerius, MD, Professor, Stem Cell Transplantation & Immunotherapy, Christian Albrechts University of Kiel, on: ‘Neutrophil-activating therapy for the treatment of cancer’. Lei Shi, PhD, Senior Vice President, R&D, Biointron Biological, on: ‘Accelerating early discovery through htp and high-speed antibody production’.

Engineer

Engineer Antibody Protein In-Vivo

Women in Science Who Have Paved the Way Forward in Genetics

XTalks

FEBRUARY 11, 2021

They conducted two experiments, one with radioactive phosphorous to label DNA, and one with radioactive sulfur to tag protein (DNA does not contain sulfur and protein does not contain phosphate groups). Daly’s work on histones stemmed from her interest in nuclear proteins.

Genetics

Genetics DNA Genome Genomics

Leveraging flow cytometry in drug discovery

Drug Discovery World

OCTOBER 21, 2022

The main principle involves a well-defined light source such as that produced by lasers that excites fluorescent tags bound to the cells or particles present in a sample. A major breakthrough in the technology was the development of biologically active fluorescent reagents that could be detected by the instruments.

Reagent

Reagent Antibody Drugs Protein

Clinical Research Informer

CRISPR tagging improves accuracy of model cells grown from stem cells

#news #biotech CRISPR tagging improves accuracy of model cells grown from stem cells

Trending Sources

bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

First gene therapy trial for Hunter syndrome opens in Manchester, UK

Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

Epigenetics discovery could lead to new class of cancer drugs

Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

Scientists turn cancer cells into anticancer agents

Second unanimous FDA adcomm vote boosts bluebird bio

ICER says bluebird bio’s $2.1m gene therapy is cost-effective

AI-designed protein awakens silenced genes, one by one

Beam makes USD 120M bet; GRAIL and Quest announce the collaboration; Brain organoids mimic infant’s brains; Improvement in T cells to kill cancer

CHMP backs J&J’s myeloma bispecific Tecvayli, ahead of US decision

Q&A: A decade on, what’s next for CAR-T therapies?

bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval

Clover biopharmaceuticals raises $230 M; BrainStorm after ALS; BARDA grants $5.65 M LightDeck for SARS-CoV-2 antigen test; Libtayo for first-line advanced NSCLC subset

A history of Pfizer

Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia

bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy

Bluebird Bio’s Skysona Receives FDA Approval and Becomes World’s Most Expensive Drug

Roche’s quantitative COVID-19 antibody test; Janssen buys a gene therapy asset; Zimmer Acquires A&E Medical; Glaucoma research study: Merck chooses TriNKET cancer immunotherapy program; Ovid fails Angelman phase 3 study

Spade SB-66

What to expect from PEGS Europe 2023: Day 3

What to expect from PEGS Europe 2023: Day 2

Women in Science Who Have Paved the Way Forward in Genetics

Leveraging flow cytometry in drug discovery

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