FDA Law Blog

JANUARY 12, 2025

Since that time, it was formalized in FDA regulations (21 CFR 314 Subpart H) in 1992, codified in the Food, Drug, & Cosmetic Act by FDAMA (21 USC 356(c)) in 1997, revised by FDASIA in 2012, and described in guidance, most importantly, in the 2014 Expedited Programs for Serious Conditions Drugs and Biologics (2014 Guidance).

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Clinical Trials Clinical Trials Trials Drugs 64

pharmaphorum

JULY 21, 2021

Novartis has revealed that the FDA has accepted its resubmission for cholesterol-lowering drug Leqvio – rejected unexpectedly by the US regulator last year – and has set a new action date of 1 January 2022. Meanwhile, sales of gene therapy Zolgensma for spinal muscular atrophy shot up more than 50% to $315 million.

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Sales Gene Therapy RNA Production 52

Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome.

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Marketing Gene Drugs Protein 52

The Pharma Data

MARCH 5, 2021

This will be adapted to the needs of messenger RNA vaccine production for CureVac’s CVnCoV, the manufacturing of which is highly complex. Production will take place in a new high-tech production facility that was already under construction at the Novartis Kundl, Austria site.

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Manufacturing Vaccination Vaccine Production 52

pharmaphorum

APRIL 28, 2022

Additionally, regulators recognise the inherent difficulties of finding sufficient populations to study rare diseases, so requirements for clinical rigour can actually be lighter, which can also lead to savings. But they also don’t have to invest in mass market advertising.

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Pharma Companies Branding Development Marketing 59

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. However, a drawback is that the dose and neuronal specificity of gene therapy is still poorly understood even in translational models.

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Trials Drug Delivery Drugs Gene 83

Roots Analysis

AUGUST 31, 2023

Mechanism of Gene Switch During the transcription process, the promoter region, which is located near the upstream end of each gene, binds to transcription factor, which is a specific type of protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.

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Gene Gene Therapy Gene Expression Regulation 40

XTalks

MAY 4, 2021

These modifications regulate gene expression without changing the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Epigenetic Editing with CRISPR.

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DNA Gene Gene Silencing Genome 98

The Pharma Data

SEPTEMBER 22, 2020

Novartis Gene Therapies to initiate new pivotal confirmatory study to evaluate use of AVXS-101 intrathecal (I T ) formulation in older patients with SMA to further support registration. Novartis Gene Therapies remains confident in the overall benefit-risk profile for patients on treatment.

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Clinical Development Gene Therapy Development Gene 52

XTalks

MAY 4, 2021

These modifications regulate gene expression without altering the sequence or structure of DNA. The epigenome plays a key role in many diseases such as heart disease, viral infections and cancer, and the new CRISPRoff technology could lead to powerful epigenetic therapies. Epigenome Editing with CRISPR.

DNA 52

DNA Gene Gene Silencing Genome 52

Pharmaceutical Technology

DECEMBER 9, 2022

The move is particularly timely given the significant growth expected in global sales of cell and gene therapies. Ribonucleic acid (RNA) based technologies are among many innovative approaches explored to tackle the Covid-19 pandemic. However, messenger RNA (mRNA) based vaccines present particular analytical challenges.

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Development Production Manufacturing Vaccine 130

Delveinsight

SEPTEMBER 23, 2021

858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Now, they are ready for their next venture, which is launching RNA biotech 858 Therapeutics with a USD 60 million Series A fundraising. 858’s drug candidates include proteins, which regulate RNA and immune response. Jeffrey Stafford, Ph.D.,

RNA 61

RNA Gene Therapy Gene Protein 61

WCG Clinical

MAY 28, 2024

When properly regulated these cellular features—proliferative capacity and receptor-directed specificity—largely account for the effectiveness of our adaptive immune system in protecting us from infectious disease. Therapies that target autoreactive B cells are seen as hopeful approaches to treat lupus and similar autoimmune diseases.

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In-Vivo Clinical Trials Clinical Trials FDA Approval 40

XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. In September 2022, Roche acquired Good Therapeutics for an upfront payment of $250 million, and has access to their PD-1-regulated IL-2 program. But all three drugs have new competitors. Both are anti-CD20 agents.

Sales 98

Sales Manufacturing FDA Approval Life Science 98

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. Vaccine Considerations.

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Life Science Vaccination Vaccine Gene 111