Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. This includes Denali Therapeutics whose treatment TAK-594/DNL593 is in a Phase I/II trial. This protein promotes lysosomal function. The trial features four cohorts.

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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

FEBRUARY 21, 2023

The adeno-associated virus five (AAV5)-based gene therapy, Hemgenix, is intended to treat adult patients with moderately severe and severe haemophilia B without a Factor IX inhibitors history. Hemgenix is claimed to be the first gene therapy to receive approval for treating haemophilia B in the EEA and the EU.

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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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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.

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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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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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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

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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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.

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Pharmaceutical Technology

DECEMBER 11, 2022

In November, the companies announced plans for a Phase I clinical trial of the combined vaccine candidate in healthy adult subjects. The first subject was dosed in the trial, which is designed to evaluate the safety, tolerability and immunogenicity of a nucleoside-modified RNA-based combination vaccine approach. By Cytiva Thematic.

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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.

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FDA Law Blog

MARCH 19, 2023

s Mark Tobolowsky co-authored the peer-reviewed article “ Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials ” in the recently published edition of Human Gene Therapy. Hyman, Phelps & McNamara, P.C.’s

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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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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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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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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.

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Pharmaceutical Technology

JUNE 29, 2022

In September 2021, GlobalData figures revealed there to be 1,320 industry-sponsored regenerative medicine and advanced therapy trials ongoing worldwide. For cell and gene therapy applications, you need a variety of speciality enzymes of the highest purity, specificity, and consistency.

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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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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

DECEMBER 15, 2020

Eli Lilly has acquired Prevail, a biotech focusing on gene therapies for neurodegenerative diseases including Parkinson’s, in a deal potentially worth more than $1 billion. The CVR pays out if one of Prevail’s gene therapies is approved in one of several developed countries before December 31, 2024.

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BioSpace

DECEMBER 10, 2020

In a shining moment that signaled hope for hemophilia B patients, uniQure presented data on Tuesday showing that its gene therapy treatment, etranacogene dezaparvovec (AMT-061), substantially increased production of the blood-clotting protein factor IX in nearly all pivotal Phase III HOPE-B trial.

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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.

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The Pharma Data

AUGUST 20, 2020

The FDA has delayed Roctavin, BioMarin’s gene therapy for haemophilia A, by a year to wait for more extensive results, it has been revealed. . The FDA said it needs longer term clinical trial data, and complete results from BioMarin’s two-year follow-up of its Phase 3 GENEr8-1 trial. Conor Kavanagh. Source link.

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XTalks

NOVEMBER 6, 2020

Canadian clinical-stage biotech company Symvivo Corporation has developed an oral COVID-19 vaccine that entered clinical trials this week. The first healthy volunteer was dosed with the vaccine in Australia as part of the bacTRL-Spike COVID-19 Phase I clinical trial. BacTRL Gene Therapy Platform.

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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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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.

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Delveinsight

DECEMBER 22, 2020

Vorasidenib is an investigational, brain-penetrant, dual inhibitor of mutant IDH1 and IDH2 in a Phase III trial in patients with IDH-mutant low-grade glioma, AG-270 is an investigational first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor while AG-636 is a novel inhibitor of dihydroorotate dehydrogenase.

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XTalks

DECEMBER 27, 2022

Adstiladrin is an adenoviral vector-based gene therapy that is non-replicating, so it cannot multiply in human cells. Boorjian, MD, Carl Rosen Professor and Chair of the Department of Urology at Mayo Clinic, and lead investigator on the recent clinical trial of Adstiladrin, in the company’s press release.

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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. The company plans to advance ABX1100 into clinical trials in the middle of next year. Topic sponsors are not involved in the creation of editorial content.

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pharmaphorum

FEBRUARY 3, 2022

Al Sandrock’s shock departure from Biogen in November sparked speculation about where he would show up next, and the answer came in today – gene therapy specialist Voyager Therapeutics. The setbacks effectively sent Voyager back to being a preclinical-stage company however, setting it back years from bringing therapies to market.

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The Pharma Data

JUNE 28, 2021

Parkinson’s disease is most common neurodegenerative movement disorder / It impacts more than 10 million people worldwide/ No function-restoring therapy is currently available / Bayer is pursuing a two-pronged approach to deliver transformative therapies with one cell and one gene therapy candidate in clinical trials.

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Pharmaceutical Technology

AUGUST 23, 2022

It comprises messenger ribonucleic acid (mRNA) that encodes for the initial spike protein of SARS-CoV-2 along with mRNA that encodes for Omicron BA.4/BA.5 5 variant spike protein. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. 5 and the wild-type viral strain.

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Delveinsight

AUGUST 26, 2021

Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion. Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease.

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The Pharma Data

JANUARY 27, 2021

(Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. FTD is a debilitating form of early onset dementia that currently has no approved disease-modifying therapies. About Passage Bio.

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The Pharma Data

OCTOBER 27, 2020

Dry AMD is one of the leading causes of vision loss globally and gene therapies hold tremendous promise for this disease. Delivering gene therapy directly to the site of disease through a subretinal injection is a proven approach; however, the current method requires a vitrectomy. at Ophthalmic Consultants of Boston.

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pharmaphorum

APRIL 21, 2022

” Alpha-synuclein is a protein that gets misfolded and clumps together in the brains of patients with Parkinson’s and other diseases collectively known as synucleinopathies. AZ/Takeda are meanwhile running phase 2 trials of their MEDI 1342 antibody candidate.

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