Drug Discovery World

JULY 21, 2023

The top stories: A third of clinical trials cancelled during Phase II Analysis of 13,490 clinical trials finds almost a third have been cancelled during Phase II amidst an overall decline in clinical development productivity.

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The Pharma Data

OCTOBER 25, 2020

NASDAQ: PTCT), today announced the initiation of a registration-directed Phase 2/3 clinical trial to evaluate vatiquinone (PTC743) in patients with mitochondrial epilepsy, the highly morbid condition of refractory seizures in children with inherited mitochondrial disease. . SOUTH PLAINFIELD, N.J. , Peltz , Ph.D.,

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Advarra

OCTOBER 26, 2023

Controlled Environment and IP Containment The potential for a release, and the risk associated with a genetically engineered IP, are part of the IBC’s assessment purview under National Institutes of Health (NIH) Guidelines. The containment must then prevent spills or leaks if the IP is dropped. Size matters.

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Pharmaceutical Technology

MAY 24, 2023

The regulator granted RMAT designation based on the data obtained from the ongoing Phase I RP-L301 clinical trial. It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene.

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pharmaphorum

JANUARY 27, 2023

Clinical trials design and patient input The definition of patient centricity, in fact, and its benefits are now – finally – being defined by patients themselves. “We We talk a lot in clinical trials and drug development about benefit,” Dr Mullen said. But who is benefitting?

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XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. The webinar included operational strategies for these trials from Laura Omoboni, MS, Executive Director of Clinical Trial Management. Rare diseases can often be progressive, chronic and fatal.

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The Pharma Data

OCTOBER 20, 2020

$1 million in research funding to support PD GENEration, a first-of-its-kind national study offering free genetic testing and counseling for people with Parkinson’s disease (PD). 10-15% of people with Parkinson’s disease have a genetic form of the disease. CAMBRIDGE, Mass.

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Drug Discovery World

JANUARY 11, 2024

The team, led by University College London (UCL), King’s College London and Moderna scientists, found that mRNA could be used to correct a rare genetic liver disease known as argininosuccinic aciduria in a mouse model. mRNA contains instructions that direct the cells to make proteins.

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The Pharma Data

DECEMBER 23, 2020

Eligible patients enrolled in the trial receive treatment with AIM’s flagship pipeline drug Ampligen. We hope that the treatment of ‘Long-Hauler’ patients in our AMP-511 clinical trial can help us to quickly determine whether Ampligen has potential as an important therapy for this post-COVID-19 syndrome. AIM ImmunoTech Inc.

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XTalks

JANUARY 3, 2024

Amvuttra: Alnylam’s Similar Treatment on the Market In 2022, Alnylam, a US-based biopharma company with expertise in RNA interference therapeutics for genetically defined diseases, received FDA approval for its drug Amvuttra (vutrisiran) for the treatment of hATTR.

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WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

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pharmaphorum

DECEMBER 2, 2021

In September, it reported a fourth patient death in a clinical trial of its AT132 (resamirigene bilparvovec) gene therapy for the rare disease X-linked myotubular myopathy (XLMTM), possibly linked to liver side effects. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

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The Pharma Data

NOVEMBER 27, 2020

Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. BOSTON, Nov.

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pharmaphorum

OCTOBER 2, 2020

Confirming press reports, the EMA said that large scale clinical trials from the vaccine codenamed AZD1222 involving several thousands of people are ongoing and results will become available over “the coming weeks and months”. AZD1222 was co-invented by the University of Oxford and its spin-out company, Vaccitech.

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Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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The Pharma Data

NOVEMBER 9, 2020

NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system disorders characterized by neuronal imbalance, today announced that it has received a response from the U.S. CAMBRIDGE, Mass., Cautionary Statement Regarding Forward-Looking Statements.

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XTalks

NOVEMBER 23, 2020

The global trials are assessing the safety and efficacy of the vaccine in individuals aged 18 years or over from diverse racial and geographic groups who are healthy or have stable underlying medical conditions. Oxford-AstraZeneca COVID-19 Vaccine Induces Immune Responses in Early Clinical Trials.

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The Pharma Data

SEPTEMBER 14, 2020

AstraZeneca has confirmed that clinical trials assessing its Oxford University partnered coronavirus vaccine AZD1222 have resumed in the UK, following a green light by the Medicines Health Regulatory Authority (MHRA). The move follows a green light by the UK's MHRA.

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pharmaphorum

SEPTEMBER 1, 2020

AstraZeneca has expanded development of COVID-19 vaccine AZD1222 into the US, beginning a phase 3 clinical trial across all adult age groups. The UK pharma said that the trial will recruit up to 30,000 adults aged 18 years or over to assess the safety, efficacy and immune response.

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XTalks

SEPTEMBER 8, 2020

AZD1222 is now in Phase III clinical trials which will enroll approximately 30,000 adult volunteers in 80 states in the US, to determine whether the vaccine can help prevent symptomatic coronavirus disease from spreading. The genetic material contains SARS-CoV-2 spike protein.

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XTalks

MAY 1, 2023

This makes Qalsody the first approved treatment to target a genetic cause of ALS. Since SOD1 mutations were first identified as a cause of ALS 30 years ago, the familial ALS community has been searching for genetically targeted treatments. To date, over 200 mutations in the SOD1 gene have been associated with the development of ALS.

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The Pharma Data

NOVEMBER 9, 2020

“While the regulatory review of this clinical program will be expedited, Novavax remains committed to a data-driven and scientifically rigorous approach in demonstrating safety and efficacy, which we believe will support confidence in the vaccine in the U.S. and globally.”. About NVX-CoV2373.

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The Pharma Data

AUGUST 16, 2021

If this genetic material is further replicated, defective RNA copies are produced and the pathogen can no longer spread. Molnupiravir is currently being tested in clinical trials. Based on preliminary clinical trials, the compound promises to be highly effective against Sars-CoV-2.

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Drug Discovery World

SEPTEMBER 4, 2023

The authors are currently recruiting participants for an NICHD-funded clinical trial called Fibroids and Unexplained Infertility Treatment With Epigallocatechin Gallate; A Natural Compound in Green Tea (FRIEND ) 1.

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XTalks

JANUARY 2, 2024

Filsuvez topical gel is a sterile botanical drug product designed for topical use, containing birch triterpenes within an oil base. The clinical study also highlighted the favorable tolerability profile of Filsuvez. Birch triterpenes constitute a botanical drug substance comprising a blend of pentacyclic triterpenes. Specifically, 41.3

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Trialfacts

NOVEMBER 23, 2022

Location: California Clinical Trials Medical Group 1560 East Chevy Chase Drive, Suite 140 Glendale, CA 91206. Achondroplasia (ACH) is a genetic condition that affects children’s growth. Must be willing to abstain or limit the use of nicotine-containing products to no more than 5 cigarettes/week until the end of the study.

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XTalks

MAY 7, 2021

Novavax published the latest results from a Phase IIb clinical trial conducted in South Africa evaluating the safety and efficacy of NVX-CoV2373, appearing in the NEJM ’s May 6, 2021 issue. Importantly, all five cases of severe disease observed in the trial occurred in the placebo group. 1.351 variant.

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The Pharma Data

NOVEMBER 30, 2020

CoV2373 is a stable, prefusion protein antigen derived from the genetic sequence of the SARS-CoV-2 coronavirus spike (S) protein and adjuvanted with Novavax’ proprietary Matrix?M. pivotal Phase 3 trial update. Novavax completed enrollment of 15,000 participants in a pivotal Phase 3 clinical trial being conducted in the U.K.

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XTalks

MARCH 10, 2023

Omaveloxolone Clinical Trials Skyclarys is an oral medication that contains omaveloxolone, an activator of the nuclear factor-erythroid 2 related factor 2 (Nrf2) pathway. Skyclarys was approved based on the results of the MOXIe Part 2 clinical trial.

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pharmaphorum

AUGUST 16, 2020

The order comes after the Commission put agreements in place for the supply of two other vaccines – from Johnson & Johnson and Sanofi/GlaxoSmithKline – that will be activated if the shots prove their worth in clinical trials. Those deals cover 200 million doses of J&J’s Ad26.COV2.S

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XTalks

MARCH 3, 2021

The US Food and Drug Administration (FDA) has granted approval to Nulibry (fosdenopterin) for reducing the risk of death associated with molybdenum cofactor deficiency type A (MoCD Type A), making it the first treatment for the ultra-rare, genetic metabolic disorder. Nulibry Trial Results.

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XTalks

SEPTEMBER 15, 2020

A vaccine developed by Swedish company Diamyd Medical has demonstrated significant treatment efficacy in a predefined genetic subgroup of individuals with type 1 diabetes in a Phase IIb clinical trial. So, this really validates what we already knew and now we have prospective results supporting that [from this trial].”.

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XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness. It is injected into a patient’s muscle tissue.

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The Pharma Data

DECEMBER 17, 2020

The Company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS. A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. Sarepta Therapeutics, Inc. www.lysogene.com.

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XTalks

DECEMBER 20, 2023

Novo Nordisk’s GLP-1 weight loss-specific drug Wegovy (semaglutide) has been facing similar shortages, as it contains the same active ingredient as Ozempic. The companies include a diverse range engaged in innovative approaches in areas like oncology, genetic medicines, inflammatory diseases, dermatology and cardiorenal therapy.

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The Pharma Data

DECEMBER 16, 2020

NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19. Additional terms of the agreement were not disclosed. About NVX-CoV2373.

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The Pharma Data

JANUARY 21, 2021

.–( BUSINESS WIRE )– Spectrum Pharmaceuticals (NasdaqGS: SPPI), a biopharmaceutical company focused on novel and targeted oncology therapies, today announced an oral presentation on updated efficacy, safety, and dosing management of poziotinib from Cohorts 1 and 2 of the ZENITH20 clinical trial.

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