Drug Discovery World

SEPTEMBER 4, 2024

The drug was approved by the FDA for Expanded Access, allowing trial participants to continue CS1 treatment when no comparable or satisfactory alternative therapy options are available. The EU OMPD complements the orphan drug designation (ODD) granted by the US FDA in 2020, enhancing the protection and value of CS1 in these major markets.

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Drug Discovery World

AUGUST 2, 2022

Biotechnology company Calidi Biotherapeutics has announced that a Phase I US clinical trial will use the company’s licensed oncolytic virotherapy platform NeuroNova to deliver an oncolytic adenovirus selectively to tumor sites in patients with recurrent high-grade glioma. . Official comments . Worldwide,?an less than one year. Camaisa.

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PPD

AUGUST 16, 2023

The COVID-19 pandemic rapidly accelerated the adoption of hybrid and decentralized clinical trial (DCT) models. However, as the world settles into its post-pandemic state and returns to pre-pandemic paradigms in many areas, the pharmaceutical industry remains dedicated to moving beyond traditional, centralized clinical trial constructs.

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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Trial results for Casgevy The authorisation is based on positive clinical trial data.

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Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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DrugBank

JULY 20, 2023

Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. We're excited to expand our data offering by launching a new Rare Disease add-on to complement our Clinical Trial dataset.

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Drug Discovery World

MAY 3, 2024

TGCT is a type of tumour that affects the soft tissue lining of joints and tendons and is a highly debilitating disease often impacting large, important joints such as the knee, hip and ankle. It is a chronic disease, which often impacts patients throughout their lives. References Lin F, et. JHEOR, 2022 Cassier , et al.

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The Pharma Data

JULY 21, 2021

Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company focused on the development and commercialization of prophylactic vaccines for infectious diseases with significant unmet medical need, and Pfizer Inc. If successful, this trial could enable the inclusion of a pediatric population in the Phase 3 trial.

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Drug Discovery World

JANUARY 24, 2023

Virtual clinical trials ‘Virtual’ and ‘technology’ are concepts which are readily exchanged in this industry, but these are more than just buzzwords. The overall virtual clinical trials market is currently valued at $7.8 In early November 2022, two Alzheimer’s drugs were tested head-to-head in a first-ever virtual clinical trial 3.

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FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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The Pharma Data

NOVEMBER 12, 2020

13, 2020 10:00 UTC. The DETOUR1 trial is the first-in-man trial of the FDA-designated Breakthrough Device, The Detour System, evaluating the safety and effectiveness of the novel procedure and device system for treating long, complex lesions in the superficial femoral artery (SFA). MILPITAS, Calif.–(

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Drug Discovery World

NOVEMBER 2, 2022

“We need to expand the arsenal of available interventions to prevent malaria infection and accelerate efforts to eliminate the disease,” said Anthony S Fauci, Director of the National Institute of Allergy and Infectious Diseases (NIAID), part of NIH. . Trials in more potent antibody.

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The Pharma Data

OCTOBER 22, 2020

The randomized, placebo-controlled trial is being conducted in partnership with the NIH’s National Institute of Allergy and Infectious Diseases and HHS’ Biomedical Advanced Research and Development Authority.

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Drug Discovery World

OCTOBER 12, 2023

Dr Samulski has advanced therapeutics into human clinical trials for haemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease and heart failure. He co-founded AskBio in 2001 and in October 2020, Bayer acquired AskBio for $4 billion. He holds more than 200 patents related to AAV technology.

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Drug Discovery World

SEPTEMBER 8, 2022

Cytel has expanded its operations into the Asia-Pacific region (APAC), with hopes of optimising clinical trial design in the area. But APAC-based developers’ efforts to adopt trial-optimising innovation have been stifled by the services currently on offer in the region.”.

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The Pharma Data

NOVEMBER 13, 2020

Centers for Disease Control and Prevention analysis finds. “While health systems are strained by the COVID-19 pandemic, we must not allow our fight against one deadly disease to come at the expense of our fight against another. © 2020 HealthDay. Posted: November 2020. All rights reserved. Source link.

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The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler. Source link.

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Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

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Drug Discovery World

DECEMBER 6, 2022

1 Vaccine development was conducted at great pace and within a year of the first infection being reported, the first Covid-19 vaccine to report phase III clinical trial data demonstrated unexpectedly high efficacy and cleared regulatory efficacy criteria with ease. Indeed, few randomised trials of NPIs have been undertaken.

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The Pharma Data

OCTOBER 26, 2020

The NIH’s National Institute of Allergy and Infectious Diseases (NIAID) has halted a clinical trial evaluating Eli Lilly’s investigational monoclonal antibody, LY-CoV555 (bamlanivimab), in combination with remdesivir for the treatment of COVID-19 patients. The trial was paused earlier this month due to a safety issue.

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Drug Discovery World

OCTOBER 18, 2023

Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas. 4 Unfortunately, many AAV-based gene therapies fail during clinical trials, despite them appearing safe and efficacious in preclinical studies.

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Drug Discovery World

AUGUST 16, 2022

1) and the original 2020 strain 1. . No serious safety issues were reported during the trial and most side – effects were mild and self-resolving. . 1 variant as well as the original 2020 strain. . “mRNA-1273.214 has consistently shown superior breadth of immune response over mRNA-1273 in clinical trials.

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The Pharma Data

DECEMBER 17, 2020

COVID-19 vaccine developers are faced with an ethical quandary — whether to let phase 3 trial participants become “unblinded” and to receive authorized vaccines as they become available, which could hinder the collection of meaningful trial data. Pfizer, which has received emergency approvals in the U.S.

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The Pharma Data

OCTOBER 26, 2020

27, 2020 /PRNewswire/ — Synlogic, Inc. Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced the Company will release its third quarter 2020 financial results before the market opens on Thursday, November 5, 2020. CAMBRIDGE, Mass. ,

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Drug Discovery World

OCTOBER 9, 2023

The reported MOD framework advances the mechanistic rationale of the clinical benefits on fatigue observed in our clinical trial with AXA1125 in the context of the combination’s mechanism of action. This is a new dynamic and efforts on research, clinical trials regulatory, and financial support are required.

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Drug Discovery World

FEBRUARY 28, 2024

Using IgE to treat cancer In the UK, a Cancer Research UK-funded clinical trial has shown, for the first time, that a new class of antibody could benefit cancer patients whose existing treatments have stopped working. Research into new cancer drugs remain strong.

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Drug Discovery World

OCTOBER 12, 2023

Dr Samulski has advanced therapeutics into human clinical trials for haemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease and heart failure. He co-founded AskBio in 2001 and in October 2020, Bayer acquired AskBio for $4 billion. He holds more than 200 patents related to AAV technology.

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Quanticate

MAY 3, 2020

This article has been updated in May 2020 to reflect the recent guidance by European Medicines Agency and other regulatory bodies on remote monitoring due to the recent coronavirus disease (COVID-19) pandemic. With this increase comes the enhanced pressure of effectively monitoring these trials.

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The Pharma Data

NOVEMBER 29, 2020

AstraZeneca’s COVID-19 vaccine candidate is facing another potential delay as the company plans to undertake a new global clinical trial to confirm the vaccine’s 90 percent efficacy based on a one-and-a-half-dose regimen. An additional trial could further delay AZ’s efforts to earn an EUA in the U.S., James Miessler. Source link.

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Drug Discovery World

JULY 18, 2023

of all new cancer cases in Europe in 2020, with around 136,000 new cases diagnosed. The MAA is based on results from the Phase III SPOTLIGHT and GLOW clinical trials. In both SPOTLIGHT and GLOW, approximately 38% of patients screened for the trials had tumours that were CLDN18.2-positive Gastric cancer accounted for 3.1%

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The Pharma Data

DECEMBER 8, 2020

8, 2020 — AstraZeneca’s COVID-19 vaccine is safe and effective, new data from late-stage trials shows. Overall, the vaccine protected against symptomatic disease in 70% of cases, according to a team led by researchers from Oxford University in England. TUESDAY, Dec. 8 in The Lancet.

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The Pharma Data

NOVEMBER 12, 2020

12, 2020 — Hydroxychloroquine is not beneficial for adults hospitalized with COVID-19 , according to the formal conclusion of a study funded by the U.S. Patients were randomly assigned to either hydroxychloroquine or placebo (242 and 237 patients, respectively); the trial was stopped for futility at the fourth interim analysis.

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The Pharma Data

NOVEMBER 4, 2020

5, 2020 11:00 UTC. NASDAQ: CNCE) today reported financial results for the third quarter of 2020. Our team has diligently and effectively executed on our plan, and I’m pleased that we have achieved our stated 2020 pipeline milestones. Initiated CTP-543 THRIVE-AA1 Phase 3 Trial in November 2020.

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Agency IQ

JUNE 28, 2024

New guidance provides definition for orphan device, offers alternative trial designs New guidance from the European Commission outlines alternatives for full pre-market clinical trials for orphan devices, defined by the Commission for the first time. MDCG 2020-10 provides guidance on safety reporting during clinical investigations.

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Drug Discovery World

MAY 29, 2023

“When it comes to published scientific research, the UK is ranked third in the world with nearly 200,000 citable publications in 2020.

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The Pharma Data

NOVEMBER 10, 2020

Innovent has established a robust pipeline of 23 valuable assets, including four launched products, four products in pivotal trials, and multiple prioritized assets such as CD47, PDL1/CD47, TIGIT, VEGF/complement protein etc. that are fast progressing clinical trials. SAN FRANCISCO and SUZHOU, China , Nov.

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The Pharma Data

NOVEMBER 11, 2020

. Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency. Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021. 12, 2020 (GLOBE NEWSWIRE) — Inozyme Pharma, Inc. BOSTON, Nov.

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