Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Sonia Vallabh and Eric Minikel , two researchers at the Broad who are developing treatments for prion disease, are excited by the potential of the AAVs to deliver brain therapies in humans.

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The Pharma Data

OCTOBER 30, 2020

More recently, the FDA announced regulatory approval for the first treatment for Covid-19. FDA approves first treatment for Covid-19. On October 22, the FDA approved the antiviral drug Veklury for use in adult and pediatric patients for the treatment of Covid-19 requiring hospitalization. Source link.

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Fierce BioTech

JANUARY 3, 2025

This joint webinar presentation by West and Groninger, explores the unique challenges of Cell and Gene Therapies, mRNA LNP therapeutics particularly regarding cold storage requirements and their impact on packaging solutions. Click here to login. Listing Image WestPharma_ListingLogo_250x190.png On Demand Start Date Wed, 02/12/2025 - 11:00

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Fierce BioTech

JANUARY 3, 2025

Innovations in aseptic processing for container closure systems dwunderlin Fri, 01/03/2025 - 14:26 Wed, 02/12/2025 - 10:00 Resource Type Webinar Jim Thompson Julian Petersen Duration 60 Minutes The pharmaceutical landscape is shifting from blockbuster drugs to personalized treatments, driving the need for innovative container closure systems.

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Eye on FDA

APRIL 13, 2022

The medicine that was the subject of these communications was DUOBRII (halobetasol propionate and tazarotene) a topical lotion indicated for the treatment of plaque psoriasis in adults. Also of note, the treatment of pain, in particular migraine, was the subject of 3 of these 11 letters. The Context.

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The Pharma Data

MAY 2, 2022

The companies expect to complete submission of the NDA for treatment of MDD in the second half of 2022; associated filing for postpartum depression anticipated in the first half of 2023. Food and Drug Administration (FDA) for zuranolone in the treatment of major depressive disorder (MDD). Sage Therapeutics, Inc.

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The Open Targets Blog

OCTOBER 24, 2023

OMass Therapeutics is using a new technology platform to develop treatments for rare immunological and genetic diseases with high unmet patient needs. This is where our idea for an R package came from.  The package has three main functionalities: schema query functions, custom query functions, and plotting functions.

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Drug Target Review

AUGUST 7, 2024

Packaging is typically the patient’s first interaction with a product, and it is critical for communicating safety and benefit-risk information. High quality artwork ensures that the product packaging conveys reliability and professionalism to foster trust within the product.

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The Pharma Data

NOVEMBER 30, 2020

The Compact will bring together in one package all WHO materials for the prevention and management of diabetes, both existing and new. On the treatment side, emphasis will be on improving access to diabetes medicines and technologies, in particular in low- and middle-income countries.

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DrugBank

JULY 20, 2023

of the global population) suffer from rare conditions without effective treatments or cures. Contact our sales team to find out more about this research package. At any given time, millions of people ( 3.5–5.9% –5.9% Rare diseases, therefore, present compelling opportunities for Drug Development.

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The Pharma Data

AUGUST 5, 2020

Taken with previous clinical evidence showing that early treatment of axSpA with Cimzia provides improved clinical outcomes, axSpA patients have a treatment option that can help address their symptoms at every stage of their disease: from initiation of biologic therapy, to remission and maintenance.

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Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? In a roughly $14 billion deal, Bristol Myers Squibb acquired Karuna, a developer of treatments for schizophrenia and other forms of psychosis associated with neurodegenerative diseases.

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The Pharma Data

DECEMBER 14, 2020

Over half of cancer patients treated with chemotherapy – over two million patients in the US and Europe – develop CIPN and experience sensory symptoms and pain in the hands and feet: loss of sensitivity, tingling, burning, cold and intense pain can persist for months to years after treatment. 15, 2020 07:15 UTC.

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Olympian Clinical Research

JUNE 27, 2023

Typically, it’s wise to avoid: Dairy products and their alternatives (cow’s milk, cheese, butter) Gluten-containing grains (wheat, barley, rye) High-sugar foods and refined carbohydrates (sugary treats, sodas, processed snacks) Processed and packaged foods with additives, preservatives, and artificial ingredients.

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Broad Institute

SEPTEMBER 13, 2023

Developing diagnostics and treatments By Rose Circeo September 13, 2023 Breadcrumb Home Developing diagnostics and treatments At the Broad Institute, researchers are revealing deep biological insights about the causes of disease, which are leading to new ways to monitor, diagnose, and treat patients with greater precision.

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Altus Drug Development

MAY 31, 2022

However, many patients end up having to face the fact that many treatments are unfeasible, too expensive, or potentially counter-conducive. The purpose of value-added medicines is to create innovative treatments for patients while offering convenient solutions to healthcare professionals. What are value-added medicines?

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The Pharma Data

MARCH 15, 2022

Teva is donating medicines valued at over $11M, for acute therapies in support of Ukrainian refugees The donations include over 1 million packages of antibiotics and other essential medicines as well as hygienic products for Ukrainian infants and children. Teva Pharmaceutical Industries Ltd. The donations made by Teva include over 1.3

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The Pharma Data

NOVEMBER 2, 2020

billion cumulative treatments to more than 923 million people since 2000. The treatments target the parasites in the blood of infected people and prevent the risk of transmission in the community. The Global Programme to Eliminate Lymphatic Filariasis (GPELF) has delivered over 8.2

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The Pharma Data

MAY 17, 2021

These initiatives could support insurance coverage of previously off-label uses, prevent costly new drugs from inappropriately receiving preferential regulatory treatment that is intended for drugs with no FDA-approved alternatives, and better inform clinicians by providing evidence-based information about how drugs should be used,” they noted. .

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The Premier Consulting Blog

OCTOBER 11, 2022

In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinical development of treatments for rare diseases. The treatment had been granted breakthrough therapy designation, but FDA approval would ultimately rest on the sponsor’s ability to demonstrate clinically meaningful improvement.

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The Premier Consulting Blog

NOVEMBER 8, 2022

With the promise of simplified and improved treatments, the $118 billion combination-product market is projected to increase at a compound annual growth rate (CAGR) of 8.8%. e) reveals more detail — and gray areas: Two or more regulated components, in one package (e.g., The drug product is packaged and shipped as a solid powder.

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Advarra

JULY 19, 2022

Q: If an emergency use authorization (EUA) is granted, once there is an approved treatment, does that mean that the EUA is no longer valid? Q: What is the typical time period between the submission of the briefing package and the pre-IND meeting? A: Yes, the EUA is just temporary. is this acceptable to the FDA?

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The Pharma Data

JANUARY 18, 2021

Providers are encouraged to reference the package insert related to MRI safety for specific details regarding implant conditions and scanning procedures in their respective geographies. “We approval complements previous MR-conditional approvals in geographies outside of the United States.

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PLOS: DNA Science

OCTOBER 19, 2023

Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease. The only treatment for ALS is a drug, Riluzole , taken as a tablet or a film. The pace though varies quite a lot.

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The Pharma Data

MAY 26, 2023

The 6,000-square-meter Shiga site opened in October 2021 to support customers both locally and globally, providing flexible clinical supply solutions, including primary packaging, Catalent’s FastChain ® demand-led supply, white glove handling and logistics.

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The Pharma Data

JANUARY 17, 2021

It received FDA guidance January 5 th and is in the midst of preparing the first package of investigational new drug (IND) applications – usually with an n-of-1, for individual patients. The treatments all use the antisense technology and automation techniques developed at Ionis Pharmaceuticals , which Crooke founded.

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Drug Target Review

JULY 7, 2023

When targeted gene editing became a reality more than ten years ago, many people hoped for a revolutionary tsunami of new medical treatments. The molecular machinery of the epigenetic system can selectively package specific regions of DNA away, making them inaccessible and less active.

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The Pharma Data

MARCH 3, 2022

We are also actively working on solutions to ensure continued access to treatment for Ukrainian patients in ongoing clinical trials, including for those who have left Ukraine and moved to other countries.

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The Pharma Data

JULY 11, 2021

Eli Lilly and Company (NYSE: LLY) announced new Phase 2 data showing that gene expression changes induced by mirikizumab in patients with ulcerative colitis (UC) over a 12-week induction treatment were maintained for up to one year. 1 or an RB subscore of 0 or 1) continued onto maintenance mirikizumab treatment. 2 points and ?35%

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LifeSciVC

AUGUST 21, 2024

And importantly, breaks from treatment or treatment protocols can literally make or break trials. Instead, it was based on the large unmet need, the advantages of a unique ADC-based delivery platform, and a strong preclinical data package that supported this strategy. I can tell you that CDMO summer shutdowns are a real thing.

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Olympian Clinical Research

OCTOBER 13, 2022

It is a relatively common condition and will usually resolve on its own without treatment within a few days to weeks. Be sure to follow the instructions on the package and only use the medication as directed. These studies test new potential treatments in hopes of finding more effective ways to manage conditions like hives.

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KIF1A

APRIL 24, 2023

The 2023 KAND Conference webpage has information related to: – registration link, hotel room link, KOALA participatio n, and agenda topics The KAND Conference will bring together patients, family members, researchers, clinicians, treatment developers, and friends of the KIF1A.ORG community. We look forward to seeing everyone in August!

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Agency IQ

JULY 13, 2023

Each of these re-authorizations, along with provisions in other, related legislative packages such as the 21 st Century Cures Act , sought to add to, tweak and/or amend the U.S. The House PAHPRA reauthorization package does include some interesting provisions. public health emergency preparedness and response infrastructure.

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The Pharma Data

JUNE 28, 2021

Across all treatment arms, the most commonly reported adverse events were gastrointestinal-related. “These findings are significant as we continue to evaluate the comprehensive efficacy and safety profile of this potential new treatment option for people with type 2 diabetes.” years, a baseline A1C of 8.28

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The Pharma Data

APRIL 4, 2023

Biosimilars help patients to gain broader access to effective and high-quality treatments that improve their disease therapies,” said Rebecca Guntern, Head of Region Europe, Sandoz. 1 “Living with a chronic disease can take a significant toll on a patient’s quality of life.

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Agency IQ

APRIL 12, 2024

Additionally, reform package included revisions of the regulations for pediatric medicines and orphan drugs. Heated debates on the most contentious issues resulted in a compromise text for each legislation in March The proposal package , consisting of one directive and one regulation, was finally released in April 2023. citizens; 2.

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Agency IQ

SEPTEMBER 15, 2023

Most commonly, PFAS enter the environment through fire training and fire response sites via PFAS-containing aqueous film-forming foams (AFFFs), industrial sites, landfills, and wastewater treatment plants. Since that time, the EPA has published additional drafts of the PHGs. On October 5, 2021, Assembly Bill (AB) 1200 was signed into law.

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