Pharmaceutical Technology

MAY 22, 2023

The US Food and Drug Administration (FDA) has granted approval for Krystal Biotech’s Vyjuvek (beremagene geperpavec-svdt) to treat dystrophic epidermolysis bullosa (DEB) in patients aged six months and above. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials.

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FDA Approval Gene Therapy Genetic Disease Gene 130

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). It is indicated for patients who do not have a pre-existing medical reason preventing treatment with the therapy.

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Gene Therapy Gene Protein FDA Approval 98

Drug Discovery World

OCTOBER 12, 2023

Samulski’s graduate work demonstrated the first substrate for adeno-associated viruses (AAV) as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV. Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field.

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Gene Therapy Gene FDA Approval Clinical Trials 52

Drug Discovery World

OCTOBER 12, 2023

Samulski’s graduate work demonstrated the first substrate for adeno-associated viruses (AAV) as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV. Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field.

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Gene Therapy Gene FDA Approval Clinical Trials 52

Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). As a condition of the SKYSONA Accelerated Approval, the manufacturer will provide confirmatory long-term clinical data to the FDA.

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Gene Therapy FDA Approval Gene Clinical Trials 52

XTalks

AUGUST 31, 2022

In this episode, Ayesha discussed the FDA approval of Axsome Therapeutics’ rapid-acting oral treatment Auvelity for the treatment of major depressive disorder (MDD). The approval makes Auvelity the first and only rapid-acting oral medication for depression, and the first and only oral NMDA receptor antagonist approved for MDD.

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Gene Therapy FDA Approval Life Science Gene 52

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. An FDA advisory committee recommended approval of Zynteglo on the strength of that data in June.

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Gene Therapy FDA Approval Gene Clinical Trials 52

pharmaphorum

MARCH 6, 2022

As bluebird bio struggles to get its gene therapies over the finishing line, the company says its financial position raises “substantial doubt” about its ability to continue as a going concern. The post bluebird says future uncertain as delays to gene therapies bite appeared first on.

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Gene Therapy Gene FDA Approval Sales 101

pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. The first patient was recruited in The Newcastle upon Tyne Hospitals NHS Foundation Trust, one of three UK sites for the clinical trial and part of 55 globally, across 15 countries.

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Gene Therapy Gene Trials Protein 89

XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. The clinical study also highlighted the favorable tolerability profile of Filsuvez. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector. Specifically, 41.3

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FDA Approval Gene Therapy Gene Trials 111

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

Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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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.

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Gene Therapy Gene FDA Approval Trials 147

XTalks

NOVEMBER 10, 2023

cTTP is a very rare, inherited and life-threatening blood clotting disorder caused by a disease-causing mutation in the ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13) gene, which encodes the ADAMTS13 enzyme that regulates blood clotting by cleaving the von Willebrand factor (VWF) protease.

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FDA Approval Gene Therapy Gene Clinical Trials 59

Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Other biotechnologies coming to the fore this year were antibody-drug conjugates and CRISPR gene editing. Read the article – Expert view: What’s next for cell and gene therapy?

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Gene Therapy Gene Antibody FDA Approval 59

Drug Discovery World

FEBRUARY 19, 2024

“The approval of Amtagvi represents the culmination of scientific and clinical research efforts leading to a novel T cell immunotherapy for patients with limited treatment options.” Amtagvi is the first FDA-approved tumour-derived T cell immunotherapy.

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FDA Approval Contract Manufacturing Manufacturing Trials 52

XTalks

JUNE 12, 2023

Recent FDA Approvals of Cell Therapy Manufacturing Facilities in the US Successful commercialization of novel cell therapy requires exemplary performance at all stages, including upstream and downstream processing, process development, testing and manufacturing services. percent during the 2022 to 2030 forecast period.

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Manufacturing Gene Therapy FDA Approval Gene 95

Drug Discovery World

JUNE 12, 2023

The US Food and Drug Administration (FDA) has approved Minoryx Therapeutics’ Phase III clinical trial (CALYX) of leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).

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FDA Approval Trials Radiology In-Vivo 52

Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.

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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.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Pharmaceutical Technology

FEBRUARY 8, 2023

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.

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Gene Therapy Gene Trials Clinical Trials 264

XTalks

MAY 24, 2023

Eyecare giant Bausch + Lomb received its first prescription drug approval from the US Food and Drug Administration (FDA) for its dry eye disease med Miebo (perfluorohexyloctane ophthalmic solution). Last year, the FDA approved the first generic version of Restasis.

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FDA Approval Gene Therapy Gene Marketing 98

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

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Gene Editing Gene In-Vivo Clinical Trials 59

Pharmaceutical Technology

APRIL 5, 2023

But while companies continue studying allogeneic CAR-T therapies, including for their coveted use in solid tumours, such advancements remain challenging. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023. Bruce Levine [BL]: We now have six approved CAR-T therapies.

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Gene Therapy Gene Engineer Production 147

XTalks

DECEMBER 4, 2023

The FDA said it is investigating the identified risk of developing the T-cell malignancies as secondary cancers with serious outcomes, including hospitalization and death, and also looking at the need for regulatory action. However, the FDA’s investigation has now revealed actual reports of T-cell malignancies linked to the therapies.

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Gene Therapy Gene Clinical Trials Clinical Trials 69

XTalks

APRIL 11, 2024

After backing from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) last month, Johnson and Johnson (J&J) and Bristol Myers Squibb (BMS) scored FDA approvals for the use of their B-cell maturation antigen (BCMA)-targeting CAR T-cell therapies as earlier line treatment options for multiple myeloma.

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FDA Approval Manufacturing Trials Clinical Trials 52

XTalks

JULY 6, 2023

Pfizer has been going strong on the approval front, having secured its fifth US Food and Drug Administration (FDA) approval in the past five weeks, with the most recent being for the company’s once-weekly human growth hormone analog Ngenla (somatrogon-ghla).

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Hormones FDA Approval Clinical Trials Clinical Trials 98

XTalks

MAY 19, 2022

Mitsubishi Tanabe Pharma America last week announced US Food and Drug Administration (FDA) approval of RADICAVA ORS® (edaravone), a medication for patients with amyotrophic lateral sclerosis (ALS). RADICAVA ORS and RADICAVA are indicated to treat ALS, with clinical trials demonstrating that RADICAVA slows loss of physical function.

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FDA Approval Clinical Trials Clinical Trials Trials 52

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.

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FDA Approval Manufacturing In-Vivo Trials 52

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

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Gene Therapy Marketing Gene DNA 52

XTalks

NOVEMBER 27, 2023

Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

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FDA Approval Clinical Trials Clinical Trials Trials 104

XTalks

MARCH 21, 2023

It is estimated that Rett syndrome affects 6,000 to 9,000 patients in the US, with a diagnosed population of approximately 4,500 patients, according to a press release from Acadia announcing Daybue’s approval. However, in animal studies, trofinetide has been shown to increase dendritic branching and synaptic plasticity signals.

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FDA Approval Gene Therapy Gene Genetics 94

XTalks

JUNE 29, 2022

The new FDA approval gives Breyanzi (lisocabtagene maraleucel) the broadest patient eligibility of any CAR T-cell therapy in relapsed or refractory LBCL. XTALKS WEBINAR: Live Cell Metabolic Analysis — Paving the Way for Metabolic Research and Cell & Gene Therapy. Outcomes of Clinical Trials for Breyanzi.

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FDA Approval Gene Therapy Antibody Gene 52

pharmaphorum

NOVEMBER 7, 2022

The conference will explore advancements in ophthalmic treatments and variety of novel drugs, which are showing great clinical trial data. Further key areas will be gene therapy and Artificial Intelligence with developments on the latest drug pipelines and clinical trials.

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Drugs Clinical Trials Clinical Trials Gene Therapy 52

Pharmaceutical Technology

AUGUST 23, 2022

According to pre-clinical findings, a booster dose of this vaccine elicited a robust neutralising antibody response against the variants Omicron BA.1, A clinical trial of the vaccine to assess its safety, tolerability and immunogenicity in people aged 12 years and above is anticipated to commence this month.

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Vaccination Vaccine Gene Therapy Protein 130

The Pharma Data

AUGUST 28, 2020

The top 10 news stories this week are focused on coronavirus vaccines and treatments, as China is set to move ahead with human testing of a potential vaccine that has been created using insect cells, while AstraZeneca has started a clinical trial of its antibody treatment designed to prevent and also treat symptoms of coronavirus.

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Gene Therapy FDA Approval Gene Clinical Trials 52

Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

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