Camargo

JANUARY 12, 2022

As we noted in a separate blog post , the forthcoming reauthorization of the FDA’s Prescription Drug User Fee Act (PDUFA VII) includes several provisions that are expected to help advance cell and gene therapy products, as well as therapies designed to treat rare diseases. Receipt of Background Package. Response Time.

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FDA Law Blog

NOVEMBER 2, 2023

While on the surface this may not seem like major advance for the field, this could be a game changer for the gene therapy space. The standards would, as stated previously, be voluntary, unless mandated by statute or regulation, and they cannot conflict with existing law or regulation.

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Camargo

AUGUST 11, 2021

However, global regulations are not one-size-fits-all, and sponsors aiming to market their products in multiple regions should be aware of the current standards and processes they may encounter during the development process. Meeting Package Due. Meeting Type. Meeting Purpose. Meeting Timing 1. from receipt of request).

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Camargo

JULY 30, 2021

But now it’s a warning in their package insert. That has to do a lot with the work in gene therapy and rare disease and the ultra-rare space because there are 7,000 rare diseases that have been identified. But I believe that the future of pediatrics is going to be in gene therapy, cell therapy.

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Drug Discovery World

MARCH 13, 2023

The cell and gene therapy (CGT) landscape has grown significantly in the past year. Between the 24 therapies already approved by the FDA 1 and a marked increase in clinical trials, widespread accessibility to precision medicine feels within reach. References “Approved Cellular and Gene Therapy Products.”

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Pharmaceutical Technology

DECEMBER 9, 2022

The move is particularly timely given the significant growth expected in global sales of cell and gene therapies. Our QC testing capabilities range from solids, semi-solids and liquids for oral, inhaled and injectable use to packaging materials, including rubber closures, glass and plastic containers.

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Cloudbyz

OCTOBER 19, 2023

Conclusion A unified clinical trial management platform is no longer a luxury but a necessity for pharmaceutical companies aiming to thrive in an increasingly competitive and regulated industry.

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Cloudbyz

AUGUST 9, 2023

As it becomes more complex with growing volumes of data, evolving regulations, and the pressure for faster drug development, traditional methods of clinical research management are no longer sufficient. Integrated Compliance and Governance With stringent regulations governing clinical trials, compliance is of utmost importance.

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The Pharma Data

NOVEMBER 22, 2020

As the Pfizer and BioNTech COVID-19 vaccine goes to regulators and the Moderna vaccine approaches the end of its Phase III trials, AstraZeneca and the University of Oxford announced high-level results from an interim analysis of their COVID-19 vaccine, AZD1222. Pascal Soriot, CEO of AstraZeneca, pictured above. Featured Jobs on BioSpace.

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Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more. 1] BioNews. Hemophilia News Today. link] [2] Tang Q, et al.

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pharmaphorum

APRIL 11, 2022

As part of this, innovative treatments such as CAR-T therapy and retinal gene therapy are emerging as ways to target rare diseases, multiple indications and underserved populations. According to EFPIA, there are over 7,000 medicines currently in development globally.

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Drug Discovery World

MARCH 29, 2023

What of the increased possibilities for ‘replacement pharmacy compounding’ for weekly hospital supplies, and for routine manufacturing of cell and gene therapies without a marketing authorisation? She also does not believe that the problems with Europe’s medicines can be solved with regulation.

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XTalks

FEBRUARY 10, 2023

It is important to have a comprehensive Product Development Plan (PDP) that is developed early and in collaboration with regulators and the non-clinical, manufacturing, clinical operations and logistics teams. Integrate clinical workflow considerations in the packaging and handling design of the investigational product (IP).

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The Pharma Data

AUGUST 3, 2021

We are very pleased that our comprehensive nonclinical data package has addressed all issues identified related to DRG toxicity and the FDA has reached the decision that we may proceed with our OAV-101 IT clinical trial program and initiate the STEER trial,” said Shephard Mpofu, M.D., SVP, Chief Medical Officer, Novartis Gene Therapies. “We

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Clinical Trials Clinical Trials Gene Therapy Trials 52

FDA Law Blog

SEPTEMBER 20, 2021

We have seen first-hand the difficult task faced by CBER review staff to keep up with the flood of innovative cell and gene therapy products, as well as the taxing influx of COVID-19 pandemic related applications requiring review resources in both Centers.

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Drug Discovery World

DECEMBER 15, 2022

Improve delivery: Clinical translation of gene therapy is dependent on the efficient and safe delivery of gene editing tools into cells, and this can occur either ex vivo or in vivo. In one example, Choi et al. In all cases, viral delivery vectors have limited payload capacity which can limit various use cases.

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Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. While AAVs show limited packaging capacity and may be costly and time-consuming, the delivery of LNPs to tissues other than the liver is particularly complicated.

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XTalks

AUGUST 2, 2023

2) Paxlovid (nirmatrelvir/ritonavir) Paxlovid is a dual-packaged oral medication for the treatment of COVID-19. Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. a month for either 150 mg or 300 mg dose strength packages, and $3,462.13 Both are anti-CD20 agents.

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The Pharma Data

MAY 5, 2021

Affinia Therapeutics raises $110 million for gene therapy treatments ( STAT ). Startup sets out to tackle the gene therapy manufacturing crisis ( Endpoints ). BD Taps Contract Packager for COVID-19 Test Kits ( MD+DI ). Medtech. Government & Regulatory.

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