The Pharma Data

NOVEMBER 29, 2020

Food and Drug Administration (FDA). Y-mAbs Therapeutics has a target action date of November 30 for its Biologics License Application (BLA) for Danyelza (naxitamab) for patients with relapsed/refractory high-risk neuroblastoma. The FDA approved the drug on November 24. Here’s a look.

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pharmaphorum

OCTOBER 16, 2020

The repurposing of drugs is becoming more common, especially in the field of rare diseases. Now, as part of lifecycle management, pharmaceutical companies are looking more closely at drugs they have on their shelves. In the quest to repurpose a drug for a rare condition, there is a need to look at any and all available data.

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The Pharma Data

NOVEMBER 2, 2020

Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. . PDUFA Action Date is Aug. 20, 2021.

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Drug Discovery World

JANUARY 30, 2024

Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Intellectual property: High patent royalties and licensing fees for key technologies can inflate treatment costs. appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 24, 2023

Israeli researchers have developed a drug that is effective in treating a rare genetic muscle disease. The team demonstrated that the disease is caused by a mutation in the gene encoding HMG CoA reductase, inhibiting the enzyme’s activity.

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The Pharma Data

DECEMBER 20, 2020

Food and Drug Administration (FDA) is plenty busy with COVID-19 vaccine Emergency Use Authorizations (EUAs) this month, but they’re also wrapping up the year with a few PDUFA dates for other therapies. The drug is a once-daily, beta-3 adrenergic agonist. Here’s a look. Urovant Sciences’ Vibegron for Overactive Bladder.

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Delveinsight

AUGUST 17, 2021

Food and Drug Administration gave a red flag to FibroGen’s anemia pill roxadustat, for the use in patients suffering from kidney diseases owing to safety concerns that arose after results, which in turn are concluded for the presentation of further clinical studies to assess safety for future medicinal use.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.

In-Vivo 52

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XTalks

DECEMBER 29, 2022

For example, on the pharma and biotech side, there has been growing demand for new drugs and biologics, from new classes of dual-action diabetes and weight loss drugs like Ozempic and Mounjaro to life-saving gene therapies. As of December 16, 2022, there are seven US Food and Drug Administration (FDA) approved gene therapies.

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Life Science Gene Therapy Gene Manufacturing 52

The Pharma Data

NOVEMBER 8, 2020

Food and Drug Administration (FDA) in terms of PDUFA dates. The drug is an investigational, novel, once-daily, oral atypical antipsychotic drug candidate designed to improve the efficacy of olanzapine while decreasing olanzapine-associated weight gain. November is promising to be a busy month for the U.S.

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The Pharma Data

DECEMBER 8, 2020

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Remix Therapeutics .

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RNA Antibody Life Science Protein 52

The Pharma Data

NOVEMBER 11, 2020

“ENPP1 deficiency is a systemic, progressive and continuous disease occurring over the course of a patient’s lifetime, starting as early as fetal development and spanning into adulthood. Received Rare Pediatric Disease Designation and Fast Track Designation from the U.S. Inozyme expects to share data from this study in 2021.

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XTalks

SEPTEMBER 17, 2020

Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012. For example, studies of heart attack prevention medications use the surrogate endpoint of low-density lipoprotein cholesterol (LDL-C) to assess the effectiveness of new drugs. Cushing’s Disease.

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XTalks

DECEMBER 13, 2023

Fulgent Genetics continues to make strides in its core genetics business, maintaining momentum in three primary areas: precision diagnostics, anatomic pathology and pharmaceutical services. This substantial investment is accelerating the growth of their new drug pipeline. million and $38.8 million and $33.9

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pharmaphorum

OCTOBER 26, 2020

The company’s most advanced programmes are in Pompe disease – a rare genetic disease causing buildup of a sugar molecule inside cells – Parkinson’s disease and congestive heart failure.

Gene Therapy 59

Gene Therapy Gene Genetic Disease Genetics 59

Drug Discovery World

MARCH 6, 2024

To find new ways of diagnosing and treating complex diseases we first must understand the mechanisms underpinning their key pathological drivers, how these relate to different patient subgroups, and which drugs might be useful in ameliorating their effects – this is the basis of precision neuroscience. This is wholly unsurprising.

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The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. Novartis today announced that the U.S.

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Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

Protein 52

Protein RNA Production Genetics 52

Drug Discovery World

FEBRUARY 22, 2024

Lu Rahman looks at neuroscience drug discovery – advances and challenges – and how breakthroughs in this field are helping address areas of unmet need. Recent advances in biomarkers, research into the central nervous system and breakthroughs in human iPSC cell models, have bolstered neuroscience drug discovery research.

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Drugs Gene Clinical Trials Clinical Trials 59

The Pharma Data

OCTOBER 28, 2020

These SAEs were assessed as mild or moderate in severity and have generally improved over a period of a few weeks while disease domain improvements have been sustained for three months. The administration method will be modified to help reduce local drug contact time. Food and Drug Administration (FDA). About Ultragenyx.

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Protein Production Development Drugs 40

The Pharma Data

OCTOBER 15, 2020

Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi. Source link.

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