Codon

JULY 27, 2023

But what happens if you restore these cone cells, using gene therapy? approved a gene therapy for hemophilia A for the first time. It’s called Roctavian and it’s only available for people who don’t have antibodies against AAV5, which is the viral vector used to deliver the gene therapy. From Moazami et al.

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Drug Target Review

AUGUST 9, 2023

14 TDP-43 is a DNA and RNA binding protein that regulates the expression and splicing of several target transcripts. Stathmin-2 is one of many proteins that appear critical for the growth and stabilisation of axons, most likely by regulating the dynamics of axonal microtubules.

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Drug Target Review

OCTOBER 12, 2023

Although non-coding variants have been increasingly appreciated for their essential role in controlling gene expression across cell types and tissues, scientists find it difficult to match them to the genes they regulate, the effects on AD-related functions and thus the individual risk of contracting AD. Frontiers [Internet].

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Elrig

APRIL 11, 2024

From 2015 to 2019, he was a postdoctoral researcher in Juergen Knoblich’s lab at the Institute of Molecular Biotechnology (IMBA), Vienna, Austria where he designed advanced human brain organoids aiming to recapitulate the architecture and function of the developing brain. He is an outdoor aficionado and loves to climb the highest summits.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

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Common Sense for Drug Policy Blog

JUNE 6, 2024

2018 ; Llewellyn and Tober 2010; Denham 2019 ). 2019 ), who found that their sample’s consumption ‘transcended IPEDs to encompass a much broader cocktail of substances’ (Salinas et al. 2019 : 49) including recreational drugs like cannabis and cocaine (see also Turnock 2021b ). 2014 ; Begley et al. Fincoeur et al.

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The Pharma Data

JANUARY 19, 2021

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. The authors suggest natural tracr-L regulates autoimmunity. And removing tracr-L altogether greatly increased CRISPR-Cas9 expression.

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Drug Target Review

OCTOBER 25, 2024

Biomarkers are an essential advancement that will help determine risk for developing Alzheimer’s disease, diagnose the disease before symptoms have progressed significantly, predict how quickly patients may experience cognitive decline and evaluate how well new therapies are working. 2019, June 11). Molecular neurodegeneration.

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Advarra

DECEMBER 15, 2022

As the research community continues to seek clinical trial efficiencies designed to bring therapies to patients faster, more governing bodies are pushing for single institutional review board (single IRB) review as one way to streamline efforts. Policies and Regulations Affecting Single IRB Review. Nonexempt human subjects research.

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Agency IQ

AUGUST 2, 2024

What We Expect the FDA to do in August and September 2024 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the months ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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Drug Target Review

SEPTEMBER 4, 2023

7,9 Dihydrofolate reductase-thymidylate synthase (DHFR) have been successfully exploited for the therapy of bacterial infections and other parasitic diseases such as malaria. 2019): CRISPR in Parasitology: Not Exactly Cut and Dried! 2019): Biological plasticity rescues target activity in CRISPR knockouts, Nature Methods.

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Agency IQ

JUNE 7, 2024

The devil is in the details: a deep dive into the state of Notified Body designations While the European Commission has been laser-focused on the number of medical device and IVD certificates, AgencyIQ is taking a look at progress toward designating Notified Bodies to the European medical device and diagnostics regulations.

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Drug Target Review

MAY 18, 2023

Specifically, the study will assess the effects of Panobinostat on non-histone proteins such as NFkB, a regulator of innate immunity, and p53, a protein that plays a critical role in controlling cell division and apoptosis. Kutlar says the possibilities of this additional therapy are exciting.

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Drug Target Review

AUGUST 24, 2023

Neutralising GDF-15 with CatalYm’s anti-GDF-15 antibody visugromab was shown to reverse its inhibitory effects and to re-sensitise tumours to anti-PD-1 treatment, achieving commensurate survival benefit of anti-GDF-15-anti-PD-1 combination therapy in vivo. 2019 May 22;10:1078. Cancer Immunology Research. 2021 Dec 1;9(12):1425-38.

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The Pharma Data

NOVEMBER 2, 2020

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders. The communication of this press release in certain countries may constitute a violation of local laws and regulations. About Sensorion.

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The Pharma Data

JANUARY 12, 2021

million for the fourth quarter of 2019. million for the full year 2019. Due to COVID-19, we saw customer demand increase significantly in both new and existing accounts, resulting in increased awareness of our high velocity therapy for treating patients with respiratory distress. 2020 was a transformational year for Vapotherm.

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Drug Target Review

SEPTEMBER 26, 2023

Early detection of neurotoxicity induced by potential new therapies is a major challenge, and hiPSC-neuronal cells may provide a solution. Ther Innov Regul Sci. 2019 Mar 1;55:93–100. 2019 Nov 1;8(6):784–8. Regul Toxicol Pharmacol RTP. 2019 Jun;78:130–41. 2018;53(4):519–25. Drug Discov Today. Toxicol Res.

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Drug Target Review

MAY 23, 2023

Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. However, many patients respond poorly to corticosteroid treatment, or their immune system responds unfavourably to biological therapies, such as the development of autoimmune diseases.

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The Pharma Data

MARCH 9, 2022

The key secondary endpoint was also met, demonstrating once-weekly efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy, showing a statistically significant reduction in ABR based on intra-patient comparison. The median annualized bleeding rate (ABR) was 0 with a mean ABR of 0.71.

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Agency IQ

MAY 17, 2024

As the legislation explained, the FDA could require the sponsor of an approved “molecularly targeted” medicine to complete a pediatric assessment if the target is “germane” to a pediatric cancer, either in terms of the number of patients or the therapeutic benefit over existing therapies.

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FDA Law Blog: Biosimilars

FEBRUARY 27, 2024

NDA 212161 and NDA 211996 were approved concurrently on May 3, 2019, at 6:13 pm, which makes the submission of the patent term extension applications on June 27, 2019, timely within the meaning of 35 U.S.C The first multiple PTE case up-to-bat concerns Gilead Sciences Inc.’s In September 2014—nearly ten years ago!

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The Pharma Data

JANUARY 6, 2021

Jefferies and Kempen join Chardan and Bryan Garnier & Co, whose analysts also initiated coverage on the company with a “Buy” rating in 2020 and 2019 respectively. The communication of this press release in certain countries may constitute a violation of local laws and regulations. About Sensorion. Source link.

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The Pharma Data

NOVEMBER 3, 2020

m (-), including US launch of the scientifically proven digital therapies deprexis® and vorvida®. 2019 Jul-Sep. 2019 Jan-Sep. 2019 Jan-Dec. 2019-2020 Jul-Sep ?. . m (90.9).

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The Pharma Data

DECEMBER 12, 2020

It plays a crucial role in many inflammatory and autoimmune diseases across multiple therapy areas, including haematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care. Alexion achieved impressive revenue growth over the last few years, with revenues of $5.0bn in 2019 (21% year-on-year growth).

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The Pharma Data

NOVEMBER 29, 2020

MDM2 is a key regulator of the tumor suppressor p53 and one of the most potent inhibitors of apoptosis discovered thus far. 1 Meanwhile, these MDM2 inhibitors present some challenges, including dose-limiting hematological toxicities, thus the urgent need to develop new generation of MDM2-targeting therapies in the treatment of cancer.

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The Pharma Data

OCTOBER 19, 2020

New Guidance: The FDA issued and implemented new guidance, Enforcement Policy for Modifications to FDA-Cleared Molecular Influenza and RSV Tests During the Coronavirus Disease 2019 (COVID-19) Public Health Emergency. Testing Therapies, Antivirals and Vaccines. Please read more here. . Please read more here. .

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The Pharma Data

AUGUST 6, 2020

Otezla® Divestiture) occurred on January 1, 2019 and exclude foreign currency hedge gains and losses. All comparisons are made versus the same period in 2019 unless otherwise stated. The increase was driven primarily by the impact of the Celgene Acquisition, which was completed on November 20, 2019. Second Quarter. . .

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The Pharma Data

JANUARY 18, 2021

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders. The communication of this press release in certain countries may constitute a violation of local laws and regulations. About Sensorion.

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The Pharma Data

NOVEMBER 19, 2020

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders. The communication of this press release in certain countries may constitute a violation of local laws and regulations. About Sensorion.

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Agency IQ

SEPTEMBER 29, 2023

What We Expect the FDA to do in October 2023 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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The Pharma Data

SEPTEMBER 9, 2020

The announcement was made during the virtual European Society of Minimally Invasive Neurological Therapy (ESMINT). Good Trackability to MCA: (1) Competitive Test Report 103618595,October 2019 (2) Competitive Test Report 103628641, October 2019. [v] v] Variable Braid Density: (1) Competitive Test Report 103628641, October 2019.

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The Pharma Data

AUGUST 1, 2021

AstraZeneca’s Saphnelo (anifrolumab-fnia) has been approved in the US for the treatment of adult patients with moderate to severe systemic lupus erythematosus (SLE) who are receiving standard therapy. 2,3 TULIP-2 demonstrated superiority across multiple efficacy endpoints versus placebo with both arms receiving standard therapy.

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Agency IQ

JUNE 2, 2023

What We Expect the FDA to do in June 2023 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods and more. An updated Unified Agenda: The Unified Agenda is the U.S.

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The Pharma Data

NOVEMBER 10, 2020

While the world is facing a severe shortage of specific antiviral drugs, a new treatment developed by Asclepius Meditec highlights the potential of hydrogen-oxygen therapy in tackling COVID-19. The paper has been contributed and under review. Effectiveness of molecular hydrogen in treating COVID-19 symptoms.

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The Pharma Data

NOVEMBER 8, 2020

Following the data releases in 2018 and 2019, this will be the third time that Ascentage Pharma has presented updated clinical data of HQP1351 at the ASH Annual Meeting. In July 2019 , HQP1351 was cleared by the US Food and Drug Administration (FDA) to enter a Phase Ib clinical study. SUZHOU, China and ROCKVILLE, Md. ,

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DECEMBER 29, 2020

HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the company was officially included into the Shenzhen-Hong Kong Stock Connect program (the “Hong Kong Stock Connect”). Ascentage Pharma (6855.HK)

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The Pharma Data

JANUARY 23, 2021

On a larger scale, the tool may greatly aid existing and future TNBC studies that are currently exploring new therapies for targeted treatments. Collagen density regulates the activity of tumor-infiltrating T cells Kuczek, D.E., immunotherapy cancer 7, 68 (2019). J Transl Med 17, 309 (2019). Larsen, A.M.H.,

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