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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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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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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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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Pharmaceutical Technology

APRIL 20, 2023

In addition to new regulatory functionalities, this feature may result in stronger and more durable protein expression. Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy. Topic sponsors are not involved in the creation of editorial content.

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Pharmaceutical Technology

MARCH 28, 2024

Discover how 4D Molecular Therapeutics Inc's patented variant AAV capsid proteins enhance muscle cell infectivity for gene therapy, revolutionizing treatment for genetic disorders.

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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.

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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.

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Gene Therapy Gene Genetics Trials 105

pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

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pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

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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. Related: Is $2 Million Too Much For FDA-Approved SMA Gene Therapy?

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Gene Therapy Gene Protein Trials 110

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. The post FDA slaps clinical hold on BioMarin’s PKU gene therapy appeared first on.

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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.

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Gene Therapy Gene FDA Approval Dermatology 97

Medical Xpress

MARCH 18, 2023

University of Utah Health scientists have corrected abnormal heart rhythms in mice by restoring healthy levels of a protein that heart cells need to establish connections with one another.

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XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

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Gene Therapy Gene Protein FDA Approval 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. A genetic treatment for epilepsy Most treatments for epilepsy aim to treat seizures, which is the main symptom of this condition, says Ferraro.

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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. OVs can be attenuated natural viruses or recombinant viruses.

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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. The post Sarepta files Duchenne muscular dystrophy gene therapy with FDA appeared first on.

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Gene Therapy Gene Genetics Protein 105

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).

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Gene Therapy Gene FDA Approval Antibody 98

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.

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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.

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Gene Therapy Gene FDA Approval In-Vivo 98

Pharmaceutical Technology

JUNE 4, 2023

Innovation S-curve for the pharmaceutical industry Transcription factors for AAV is a key innovation area in pharmaceutical Adeno-associated virus (AAV) vectors are widely used for gene therapy. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

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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.

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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.

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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. And finally, no matter how full you get, there’s always room for dessert — and yes, for an extra helping of gratitude. Enjoy the holiday.

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Gene Therapy FDA Approval Gene Genetics 97

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.

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Gene Therapy Gene Marketing Genetics 83

XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

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Pharmaceutical Technology

APRIL 26, 2023

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The new deal excludes oncology and other therapeutic areas outside the scope of genetic disease.

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Scienmag

SEPTEMBER 7, 2020

Credit: Dr Jie Zheng An innovative genetic study of blood protein levels, led by researchers in the MRC Integrative Epidemiology Unit (MRC-IEU) at the University of Bristol, has demonstrated how genetic data can be used to support drug target prioritisation by identifying the causal effects of proteins on diseases.

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Genetics Protein Drugs Development 56

pharmaphorum

DECEMBER 22, 2021

Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient. AAV vectors are well-suited to gene therapies for DMD because they are effective at targeting muscle tissue.

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pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. Shares in bluebird ticked up following the announcement.

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pharmaphorum

OCTOBER 18, 2022

Eli Lilly has agreed to buy hearing loss gene therapy developer Akouos in a deal that could value the company at around $610 million – if its lead candidate progresses as hoped in the clinic. The buyout – for $12.50 The buyout – for $12.50 There are no approved drug treatments for this type of hearing loss.

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Gene Therapy Gene Genetics FDA Approval 52

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. And ultimately we will be able to add genetic influences to diseases that don’t have a genetic cause.

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Pharmaceutical Technology

AUGUST 11, 2022

In a Pompe mouse disease model, ABX1100 was demonstrated to greatly reduce the Gys1 mRNA and GYS1 protein, causing a meaningful decline in glycogen levels in the skeletal muscle. A biotechnology firm, Aro Biotherapeutics focuses on developing tissue-targeted genetic treatments.

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Drugs Clinical Trials Clinical Trials Gene Therapy 130

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.

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Scienmag

MARCH 19, 2021

Treatment involving a single injection has long-lasting effects BOSTON – Researchers have used a genetic engineering strategy to dramatically reduce levels of tau–a key protein that accumulates and becomes tangled in the brain during the development of Alzheimer’s disease–in an animal model of the condition.

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