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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Gene Therapy Gene FDA Approval Clinical Trials 96

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

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

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).

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Gene Therapy Gene FDA Approval Dermatology 97

XTalks

DECEMBER 27, 2022

Ferring Pharmaceuticals announced that the US Food and Drug Administration (FDA) approved Adstiladrin (nadofaragene firadenovec-vncg) for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

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Gene Therapy FDA Approval Gene Clinical Trials 52

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). With a list price of $3.5 With a list price of $3.5

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Gene Therapy Gene FDA Approval Drugs 52

pharmaphorum

AUGUST 10, 2020

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA) drug Evrysdi (risdiplam). The FDA approved Evrysdi for the treatment of spinal muscular atrophy (SMA) in adults and children two months of age and older. Zolgensma costs about $2.1m

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FDA Approval Drugs Gene Therapy Protein 59

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. There are no approved drug treatments for this type of hearing loss. The buyout – for $12.50 The buyout – for $12.50

Gene Therapy 52

Gene Therapy Gene Genetics FDA Approval 52

XTalks

NOVEMBER 30, 2022

In this episode, Ayesha shared news about the FDA approval of the first gene therapy for the rare blood disorder Hemophilia B. Read the full articles here: Hemgenix Approved as First Gene Therapy for Hemophilia B. World Antimicrobial Resistance (AMR) Awareness Week: Preventing AMR Together.

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Gene Therapy Life Science Gene FDA Approval 52

Pharmaceutical Technology

AUGUST 23, 2022

On obtaining the FDA EUA, the Omicron BA.4/BA.5-adapted 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.

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Vaccination Vaccine Gene Therapy Protein 130

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. A gene therapy, it permits the body to read over the DNA mutation and still continue to produce dystrophin. The post Translarna not approved by NICE for DMD on NHS appeared first on.

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Gene Therapy Gene FDA Approval DNA 98

XTalks

AUGUST 17, 2020

As DMD is caused by a lack of functional dystrophin protein, which supports and strengthens muscle fibers, therapies designed to treat this rare genetic disorder have focused on exon skipping to avoid the area of the dystrophin gene that is mutated and produce a nearly full-length dystrophin protein.

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Drugs Clinical Trials Clinical Trials Gene 105

XTalks

NOVEMBER 1, 2024

Why it sold so well: Botox is FDA-approved for the treatment of chronic migraines and that is currently the only chronic condition for which it has received approval. Related: Top 30 Drugs to Watch in 2024: Insights from 2023 Sales Data 1. billion in the US and $4.1 billion worldwide last year.

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FDA Approval Drugs Botox Sales 85

Pharmaceutical Technology

MAY 15, 2023

In a closely contested debate, a US Food and Drug Administration Advisory Committee (AdCom) panel voted eight to six in favour of the accelerated approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene transfer therapy delandistrogene moxeparvovec (SRP-9001).

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Gene Gene Therapy FDA Approval Drugs 130

XTalks

MAY 15, 2023

Chiesi Global Rare Diseases in collaboration with Protalix BioTherapeutics announced the US Food and Drug Administration (FDA) approval of their new medication Elfabrio (pegunigalsidase alfa-iwxj) for the treatment of Fabry disease.

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FDA Approval Gene Genetics Clinical Trials 98

Pharmaceutical Technology

MAY 23, 2023

The US Food and Drug Administration (FDA) has granted Centessa Pharmaceuticals’ activated Protein C (APC) inhibitor SerpinPC a fast track designation for treating haemophilia B. Centessa and other companies are investigating long-term disease-modifying therapies to reduce the disease burden for haemophilia B patients.

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Gene Therapy Gene FDA Approval Drugs 147

pharmaphorum

SEPTEMBER 12, 2022

Reddy says one of LabCorp’s groundbreaking tests, OmniSeq INSIGHT, is an “all-in-one test that includes comprehensive genomics and immune profiling from a single tissue sample to identify all FDA-approved therapeutics, confirm immunotherapy eligibility for a patient, and identify appropriate nearby clinical trials.”.

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Genome Genomics Clinical Trials Clinical Trials 105

XTalks

MARCH 1, 2024

While there is no cure for most types of tinnitus, treatments can help manage the condition, including sound therapy, counseling and hearing aids. Related: Interview with Neuromod COO Diarmuid Flavin About the FDA Approval of Tinnitus Treatment Lenire – Xtalks Life Science Podcast Ep.

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Gene Therapy Gene Life Science Genetics 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. Clinical Trials Backing Qfitlias Approval Two Phase III studies involving adult and pediatric male patients with hemophilia A or B supported Qfitlias FDA approval.

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FDA Approval RNA Clinical Trials Clinical Trials 59

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. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

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Gene Therapy Gene FDA Approval In-Vivo 98

XTalks

MAY 9, 2024

We also invented four proprietary antibody technologies, including our DuoBody® bispecific technology which is the backbone of four FDA-approved bispecifics and over ten in development. In turn, this advent in AI and machine learning has the potential to lead to the discovery and development of more effective therapies.

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Antibody Clinical Trials Clinical Trials Medicine 52

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. It was first approved in the US in 2019 to treat children below the age of two with SMA.

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Gene Therapy Gene Protein Trials 110

XTalks

AUGUST 30, 2022

We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways. What About Small Proteins and Antibodies Reaching Extracellular CNS Targets? Next Steps for Targeted CNS Delivery.

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Trials Drug Delivery Drugs Gene 83

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 caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells.

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Gene Therapy Gene Protein FDA Approval 98

XTalks

MARCH 28, 2024

Italy-based drugmaker Italfarmaco has won US Food and Drug Administration (FDA) approval for its oral medication Duvyzat (givinostat) for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. As one of the most expensive therapies in the world priced at $3.2 million last year, of which $131.3

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

XTalks

APRIL 12, 2021

Like other neurodegenerative disorders, such as Alzheimer’s disease and dementias, Parkinson’s disease research has been immensely challenging for biotechs, with a high rate of late-stage attrition in trials leading to a lack of disease-modifying therapies being approved. Axovant Gene Therapies. Prevail Therapeutics.

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Gene Therapy Gene Trials Development 96

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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Gene Therapy Gene Gene Editing Sales 111

Pharmaceutical Technology

FEBRUARY 13, 2023

Although recently approved drugs can treat seizures more safely, they do not treat the comorbidities that patients experience. 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.

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Research Gene Gene Therapy Genome 264

Pharmaceutical Technology

JUNE 9, 2023

Rocket Pharmaceuticals has received fast track and orphan drug designations for RP-A601 from the US Food and Drug Administration (FDA) to treat plakophilin-2-related arrhythmogenic cardiomyopathy (PKP2-ACM). In April 2023, the FDA approved the investigational new drug (IND) application for RP-A601 to begin a Phase I clinical trial.

Gene Therapy 147

Gene Therapy Gene FDA Approval Trials 147

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 million, according to GlobalData’s Price Intelligence (POLI), making it the most expensive drug in the world.

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Gene Therapy Marketing Gene Drugs 263

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.

FDA Approval 52

FDA Approval Gene Therapy Drugs Gene 52

Pharmaceutical Technology

FEBRUARY 1, 2023

RSV researchers at major pharmaceutical companies are currently working to develop new RSV drugs to beat future waves of RSV infection and gain the first RSV vaccine FDA approval. According to the Centers for Disease Control and Prevention (CDC) , 45.8 This is a significant increase compared to the 2021–20 season when the rate was 28.3

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Vaccination Vaccine Antibody Development 162

XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

FEBRUARY 27, 2024

Iovance Biotherapeutics’ Amtagvi (lifileucel) won US Food and Drug Administration (FDA) approval last week for the treatment of advanced melanoma, making it the first individualized tumor-infiltrating lymphocyte (TIL) therapy and the first T-cell therapy for a solid tumor to win US regulatory approval.

FDA Approval 52

FDA Approval Manufacturing In-Vivo Trials 52

XTalks

DECEMBER 4, 2023

In a statement outlining its investigation, the FDA said it received reports of the T-cell malignancies from clinical trials and post-marketing safety monitoring. The currently approved therapies target CD19 and BCMA, which are expressed in cancers like B-cell lymphoma and multiple myeloma.

Gene Therapy 69

Gene Therapy Gene Clinical Trials Clinical Trials 69

The Pharma Data

SEPTEMBER 28, 2021

The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to 6 weeks aged and data supporting the continued clinical investigation of gene therapy, SRP-9001, in Duchenne dystrophy (DMD).

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Protein Gene Therapy Gene Clinical Trials 52