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.

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

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.

Gene Therapy 102

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Pharmaceutical Technology

DECEMBER 8, 2022

According to the revised deal, Daiichi Sankyo and Kite will transfer the Marketing Authorization for Yescarta to Gilead’s Japan subsidiary, Gilead Sciences K.K., The therapy’s sales and promotion works in Japan following the transfer of marketing authorisation will be carried out by the Kite Cell Therapy Business Unit at Gilead Sciences K.K.

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pharmaphorum

AUGUST 26, 2022

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. The post Roctavian okay sets up another gene therapy test for Europe appeared first on. million spread over five years.

Gene Therapy 111

Gene Therapy Gene Antibody Marketing 111

Pharmaceutical Technology

DECEMBER 23, 2022

Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. By Cytiva Thematic.

Genetics 328

Genetics Medicine Development Gene Therapy 328

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. Though there are still few commercial treatments, in terms of therapeutic potential, Ben Hargreaves finds that companies and investment firms are pushing for this number, and the overall market, to grow rapidly in the coming years.

Gene Therapy 98

Gene Therapy Gene Gene Editing DNA 98

pharmaphorum

JANUARY 12, 2022

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file for regulator approval. point increase on the NSAA scale with the gene therapy, while the control group saw a 0.7-point

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Gene Therapy Gene Regulation Trials 105

pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023.

Gene Therapy 105

Gene Therapy Gene Genetics Trials 105

pharmaphorum

MAY 21, 2021

US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. The treatment restore the activity of a protein that breaks down the toxic metabolites.

Gene Therapy 96

Gene Therapy Gene FDA Approval Clinical Trials 96

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. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy? With a price tag of over $2.5

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

Pharmaceutical Technology

JUNE 29, 2022

Specialty enzymes are proteins that can act as very specific biocatalysts to accelerate reactions and produce the desired target molecule in pharmaceuticals,” explains Kristoffer Laursen, Head of R&D at Novo Nordisk Pharmatech. In the evolving biotechnology industry, enzymes are important process aids.

Drugs 295

Drugs Gene Therapy Production Manufacturing 295

XTalks

AUGUST 30, 2022

The hemophilia A gene therapy Roctavian delivers a functional gene to enable the body to make an essential blood clotting protein on its own. A lack of sufficient functioning factor VIII and factor IX blood clotting proteins would result in the development of hemophilia A and hemophilia B, respectively.

Gene Therapy 98

Gene Therapy Gene Clinical Trials Clinical Trials 98

pharmaphorum

NOVEMBER 5, 2020

An experimental gene therapy developed by Texas biotech Genprex will be paired with AstraZeneca’s Tagrisso and Merck & Co’s Keytruda – both leading their respective drug classes in the treatment of non-small cell lung cancer (NSCLC). Tagrisso is the top-selling EGFR inhibitor, with sales of almost $3.2

Gene Therapy 98

Gene Therapy Gene Drugs Contract Manufacturing 98

pharmaphorum

OCTOBER 4, 2020

Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review. CALD is caused by mutations in the ABCD1 gene located on the X chromosome, which provides instructions for the production of the ALD protein.

Gene Therapy 98

Gene Therapy Gene Genetic Disease Protein 98

pharmaphorum

SEPTEMBER 30, 2022

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the middle of 2023. Roche has exclusive rights to the therapy outside the US. Photo by Markus Spiske on Unsplash.

Gene Therapy 105

Gene Therapy Gene Genetics Protein 105

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 95

Gene Therapy FDA Approval Gene Clinical Trials 95

XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

Gene Therapy 98

Gene Therapy Gene FDA Approval Antibody 98

Pharmaceutical Technology

DECEMBER 11, 2022

5 Omicron sublineages spike proteins. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies.

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Vaccine Vaccination Gene Therapy Gene 289

pharmaphorum

DECEMBER 10, 2020

Shares in Rocket Pharmaceuticals have been living up to their name, shooting up following encouraging early-stage clinical trial results from a gene therapy for a serious inherited rare heart disease. RP-A501 could be the first gene therapy for the disease and the early data showed a positive increase in cardiac protein expression.

Gene Therapy 82

Gene Therapy Gene Protein Trials 82

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 98

Gene Therapy Gene FDA Approval In-Vivo 98

Pharmaceutical Technology

FEBRUARY 10, 2023

On November 22, 2022, the FDA approved CSL Behring’s Hemgenix (etranacogene dezaparvovec), the first gene therapy treatment for hemophilia B, with a staggering manufacturer price of $3.5 Undoubtedly, this has played a factor in the success of market-leading PD-1 inhibitors, Merck & Co.’s

Gene Therapy 263

Gene Therapy Marketing Gene Drugs 263

Pharmaceutical Technology

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

Gene Therapy 208

Gene Therapy Gene Gene Expression Regulation 208

Pharmaceutical Technology

DECEMBER 5, 2022

mRNA technology is projected to offer a new generation of vaccines that direct specific cells to generate proteins that are detected by the immune system to develop a defence. If Covid-19 taught us anything, it’s the importance of local capability rather than relying on global markets. “We By Cytiva Thematic.

Research 246

Research Gene Therapy Vaccine Vaccination 246

pharmaphorum

MARCH 8, 2021

million one-off gene therapy for Spinal Muscular Atrophy (SMA) NICE has said, the most expensive treatment ever approved for funding. This is the second gene therapy recommended for funding by NICE – in 2019 Novartis’ Luxturna (voretigene neparvovec) was approved for NHS funding for a rare inherited eye disease.

Gene Therapy 83

Gene Therapy Gene Marketing Genetics 83

Pharmaceutical Technology

SEPTEMBER 22, 2022

Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis. AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein.

Gene Therapy 147

Gene Therapy Gene Protein Clinical Trials 147

STAT News

NOVEMBER 23, 2022

Food and Drug Administration on Tuesday approved the first gene therapy to treat people with hemophilia B, an inherited bleeding disorder , STAT writes. The one-time treatment, called Hemgenix, was developed by the Dutch biotech company UniQure and will be marketed by CSL Behring, an Australian pharmaceutical company.

Gene Therapy 97

Gene Therapy FDA Approval Gene Genetics 97

BioPharma Reporter

MARCH 22, 2022

Fujifilm Irvine Scientific, Inc, has announced the acquisition of Shenandoah Biotechnology, a company supplying recombinant proteins to the drug discovery, life science research, and cell and gene therapy markets.

Gene Therapy 98

Gene Therapy Life Science Protein Gene 98

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. Common gene therapies use vehicles such as adeno-associated vectors (AAVs), which cannot carry the required payload in this case, says Carney.

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Research Gene Gene Therapy Genomics 264

Pharmaceutical Technology

DECEMBER 15, 2022

The congenital fibrinogen deficiency (CFD) market is expected to grow at a compound annual growth rate (CAGR) of 1.7% through 2031, reaching over $800m across the three major markets (3MM: US, Germany, and Japan), according to GlobalData’s recent report: Congenital Fibrinogen Deficiency: Opportunity Assessment and Forecast.

Marketing 130

Marketing Production Genotype Development 130

pharmaphorum

JULY 7, 2022

New data on Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. ” The post Myocarditis case mars Sarepta DMD gene therapy readout appeared first on.

Gene Therapy 59

Gene Therapy Gene Protein Trials 59

Roots Analysis

NOVEMBER 30, 2021

Bioassay – A Prerequisite for the Cell and Gene Therapy Development. Over the past few years, investigational new drug (IND) filings for cell and gene therapy product have significantly increased. Companies Offering Bioassay Services for Cell and Gene Therapies.

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Bioassay Gene Therapy Gene Development 52

pharmaphorum

JULY 22, 2021

Bluebird bio has its first approval for Skysona (formerly Lenti-D), its gene therapy for children with the rare disease cerebral adrenoleukodystrophy (CALD), after getting the nod from the EU regulatory authority. The modified cells are reinfused and can produce a functional version of the ALD protein that is lacking in CALD.

Gene Therapy 98

Gene Therapy Gene Trials Marketing 98

Pharmaceutical Technology

MAY 16, 2023

UniQure is selling a part of its royalty rights to the haemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) to HealthCare Royalty and Sagard Healthcare for a gross purchase price of up to $400 million dollars. Hemgenix is an adeno-associated virus (AAV) 5-based gene therapy.

Gene Therapy 130

Gene Therapy Gene Sales Development 130

XTalks

NOVEMBER 24, 2022

In a pivotal approval, the US Food and Drug Administration (FDA) has given the nod to a new gene therapy called Hemgenix (etranacogene dezaparvovecfor) for the treatment of adults with the genetic blood disorder hemophilia B (congenital Factor IX deficiency). Related: Roctavian, the First Hemophilia A Gene Therapy, Attains EC Approval.

Gene Therapy 52

Gene Therapy Gene FDA Approval Drugs 52

XTalks

APRIL 1, 2025

Qfitlia differs from traditional factor replacement therapies. Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. Qfitlia will be entering a small but competitive market. Among these are three gene therapies. Sanofi is not new to the hemophilia market.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

Pharmaceutical Technology

DECEMBER 16, 2022

Sosei Heptares and Eli Lilly and Company have signed a partnership for the discovery, development and commercialisation of small molecules that modulate new G protein-coupled receptor (GPCR) targets linked to diabetes and metabolic diseases.

Development 130

Development Gene Therapy Protein Sales 130

pharmaphorum

JANUARY 29, 2021

This growth reflects the continued and increasing interest in gene therapy – and with stock prices rising for editing companies across the board, Macrae says there has never been a more interesting time to be in genomic medicine. That is why the company is also working on gene therapy and ex vivo gene-edited cell therapy. “If

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Gene Editing Gene Gene Therapy In-Vivo 145