Clinical Research Informer

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

Pharmaceutical Technology

SEPTEMBER 30, 2022

After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals. Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions.

Gene Therapy

Gene Therapy FDA Approval Gene Genetic Disease

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

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.

Gene Therapy

Gene Therapy Gene FDA Approval Licensing

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

XTalks

DECEMBER 13, 2023

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. It also affects Hispanic Americans, but at a lower prevalence.

Gene Therapy

Gene Therapy FDA Approval Gene In-Vivo

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

Drug Discovery World

OCTOBER 31, 2023

UK regulatory authorities have approved the first trial of a gene therapy for young children with Hunter syndrome. The drug was developed over eight years by Brian Bigger, Professor of Cell and Gene Therapy at The University of Manchester. The modified HSCs are then infused back into the patient to engraft in the bone marrow.

Gene Therapy

Gene Therapy Gene Trials Clinical Trials

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

Pharmaceutical Technology

APRIL 5, 2023

More than a decade after the first patient was treated with a CAR-T therapy, six therapies relying on the same principles have been approved by the US Food and Drug Administration (FDA) and marketed to thousands of patients. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023.

Gene Therapy

Gene Therapy Gene Engineer Production

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

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.

Gene Therapy

Gene Therapy FDA Approval Gene Clinical Trials

Scientists turn cancer cells into anticancer agents

Drug Discovery World

JANUARY 9, 2023

Harvard investigators have developed a new cell therapy approach to eliminate established tumours and induce long-term immunity. . Using gene engineering, we are repurposing cancer cells to develop a therapeutic that kills tumour cells and stimulates the immune system to both destroy primary tumors and prevent cancer.” .

Scientist

Scientist Gene Editing Gene Engineer

Second unanimous FDA adcomm vote boosts bluebird bio

pharmaphorum

JUNE 12, 2022

bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8

Gene Therapy

Gene Therapy Gene Marketing Trials

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

pharmaphorum

APRIL 14, 2022

US cost-effectiveness watchdog ICER has handed bluebird bio some good news ahead of its FDA advisory committee meeting for rare blood disorder gene therapy beti-cel in June, by endorsing its proposed $2.1 million price tag. gene therapy is cost-effective appeared first on. The post ICER says bluebird bio’s $2.1m

Gene Therapy

Gene Therapy Gene Marketing Clinical Trials

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

Beam employs three approaches to deliver its genetic medicines to cells. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. The company’s in vivo therapies reach the eye and central nervous system via adeno-associated viruses, while its liver therapies employ lipid nanoparticles (LNPs).

DNA

DNA In-Vivo Genetics Medicine

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

pharmaphorum

JULY 25, 2022

The EMA’s human medicines committee has recommended approval of Johnson & Johnson’s Tecvayli as a fourth-line therapy for multiple myeloma , joining a growing group of BCMA-targeted therapies for the blood cancer.

Antibody

Antibody Medicine Drugs Trials

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

pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. million for chelation therapy to remove excess iron from the body. million, which compares to the $1.7

Gene Therapy

Gene Therapy FDA Approval Gene Clinical Trials

AI-designed protein awakens silenced genes, one by one

The Pharma Data

MARCH 7, 2022

Researchers from the University of Washington School of Medicine in Seattle describe this finding in the journal Cell Reports. “The beauty of this approach is we can safely upregulate specific genes to affect cell activity without permanently changing the genome and cause unintended mistakes,” Levy said.

Protein

Protein Gene Genome Genomics

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

Roche has got an authorization from the FDA for a more accurate COVID-19 blood test capable of measuring the levels of specific antibodies, which target the cell-unlocking spike protein of coronavirus. Janssen buys a gene therapy asset. Janssen buys a new gene therapy aimed at reversing a severe disease, from Hemera Biosciences.

Gene Therapy

Gene Therapy Gene Antibody Research

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

XTalks

SEPTEMBER 27, 2022

With a list price of $3 million and a recent accelerated US Food and Drug Administration (FDA) approval, bluebird bio’s gene therapy Skysona (elivaldogene autotemcel, eli-cel), has officially become the world’s most expensive drug. Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia. Related: Bluebird’s $2.8M

FDA Approval

FDA Approval Gene Therapy Drugs Gene

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 now plans to study more about the biomarkers in ALS and hopes to come up with broadly effective therapy.

In-Vitro

In-Vitro Vaccination Vaccine Licensing

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

pharmaphorum

FEBRUARY 16, 2021

It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance. There is a curative treatment for people who rely on blood transfusions to survive and maintain their levels of red blood cells. million in Europe.

Gene Therapy

Gene Therapy Gene Medicine Development

What to expect from PEGS Europe 2023: Day 2

Drug Discovery World

NOVEMBER 9, 2023

Klaus Wagenbauer, PhD, Founder & CEO, Plectonic, on: ‘Programmable DNA-origami-based T cell engagers: PTE’. Gail Calvert, Field Application Scientist, Sartorius UK, on: ‘Leveraging integrated and advanced technologies for successful cell line development’.

Engineer

Engineer Antibody Protein In-Vivo

What to expect from PEGS Europe 2023: Day 3

Drug Discovery World

NOVEMBER 13, 2023

Alexander Yermanos, PhD, Lecturer, Systems & Synthetic Immunology, ETH Zurich, on: ‘Integrating single-cell immune repertoire sequencing, machine learning, and biophysical properties of antibodies’. He will discuss selection technologies for membrane targets using advances in whole cell panning and comparative deep sequencing.

Protein

Protein Antibody Engineer Scientist

Leveraging flow cytometry in drug discovery

Drug Discovery World

OCTOBER 21, 2022

Take, for example, its use for CD4 T cell counting to facilitate HIV monitoring. Flow cytometry is a technique used to rapidly analyse single cells in order to characterise their physical, chemical and biological properties. . Overview, evolution, trends .

Reagent

Reagent Antibody Drugs Protein

Clinical Research Informer

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

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

Trending Sources

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

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

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

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

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

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

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

AI-designed protein awakens silenced genes, one by one

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

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

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

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

What to expect from PEGS Europe 2023: Day 2

What to expect from PEGS Europe 2023: Day 3

Leveraging flow cytometry in drug discovery

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