Pharmaceutical Technology

APRIL 26, 2023

This marks the first-ever designation for genetically modified gamma-delta T cell therapies. INB-400, an autologous, genetically engineered gamma-delta T cell therapy, is the company’s DeltEx chemotherapy-resistant autologous and allogeneic drug-resistant immunotherapy (DRI) technology.

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Genetic Engineering Drugs Genetics Engineer 130

pharmaphorum

APRIL 26, 2022

Making sure clinical trials are diverse and representative of the populations they’re studying isn’t just the right thing to do to promote inclusivity and equity; it’s also just good science/clinical scientific practice. Solutions for improving diversity, equity and inclusion in trials.

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Clinical Trials Clinical Trials Trials Regulation 96

Drug Discovery World

DECEMBER 21, 2023

The company also plans to conduct global, multicentre Phase Ib trials in patients with ulcerative colitis following the Phase Ia study. IBD impacts a large patient population who currently have limited therapeutic options,” said Sujata Rao, Chief Medical Officer at Insilico Medicine who is leading the clinical trial. “We

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Trials Clinical Trials Clinical Trials Medicine 52

Pharmaceutical Technology

OCTOBER 25, 2022

There are options in the future to possibly apply the worldwide research and development (R&D), manufacturing and marketing expertise of Astellas in gene therapy to AAV gene therapy development programmes of Taysha for genetic ailments of the central nervous system (CNS).

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Gene Therapy Gene Development Genetics 264

Pharmaceutical Technology

MARCH 2, 2023

There are three more drug candidates with major trial readouts that are expected in 2023. We are grateful to Friedreich’s ataxia patients, investigators, US regulators, and our scientists and employees who made this approval possible. “As

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FDA Approval Trials Genetics Drugs 264

Pharmaceutical Technology

JUNE 22, 2022

It works by down-regulating fibroblast growth factor receptor 3 (FGFR3) signalling and subsequently boosting endochondral bone formation. The latest approval is based on findings from an international, placebo-controlled, randomised, double-blind Phase III clinical trial assessing the efficacy and safety of Voxzogo.

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Regulation Trials Clinical Trials Clinical Trials 162

Pharmaceutical Technology

MAY 4, 2023

The approval allows KALYDECO to be used in infants who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the therapy, on the basis of clinical and/or in vitro assay results.

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In-Vitro Genetic Disease Medicine Genetics 246

Drug Discovery World

JULY 6, 2023

The drug candidate, a monoclonal antibody (mAb) targeting the protein Semaphorin-3A (Sema3A), is being developed as a potential first-to-market treatment for Alport syndrome, a rare genetic kidney disease. Semaphorin-3A is an extracellular guidance protein and a well-known regulator of the actin cytoskeleton.

Antibody 52

Antibody Trials Genetics Protein 52

Drug Discovery World

JANUARY 23, 2024

Sano Genetics, a software company working to accelerate precision medicine research, has raised $11.4 Access and referrals for patients to join trials often depend on the hospital or location where they’re receiving treatment – which is why recruiting for clinical trials is one of the biggest challenges in research.

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. The webinar included operational strategies for these trials from Laura Omoboni, MS, Executive Director of Clinical Trial Management. Rare diseases can often be progressive, chronic and fatal.

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Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

AUGUST 16, 2022

According to the deal, Horizon will provide the funding to develop the therapy until the conclusion of its two Phase II clinical trials. Under the agreement, Horizon will hold an option for the acquisition of the ADX-914 programme, which is exercisable through a period after concluding the Phase II trials.

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Development Clinical Trials Clinical Trials FDA Approval 130

pharmaphorum

AUGUST 23, 2022

France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease.

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Gene Therapy Gene Trials Protein 105

pharmaphorum

SEPTEMBER 14, 2020

AstraZeneca has resumed UK trials for its coronavirus vaccine, after the country’s medicines regulator gave the all-clear following a safety scare. A UK safety committee has concluded its investigations and recommended to the country’s Medicines and Healthcare products Regulatory Agency (MHRA) that trials are safe to resume.

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Vaccination Vaccine Trials Protein 83

FDA Law Blog

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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Gene Therapy Gene Trials Clinical Trials 98

Cloudbyz

MARCH 30, 2023

These therapies are often based on personalized treatments that leverage the unique characteristics of each patient’s genetic makeup, and they hold enormous promise for treating a wide range of conditions, from cancer to rare genetic disorders.

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Gene Therapy Gene Trials Clinical Trials 52

Pharmaceutical Technology

MAY 22, 2023

It is claimed to be both the first re-dosable gene therapy and the first and only FDA-approved treatment for both recessive and dominant types of DEB, a rare and serious genetic disease affecting the skin and mucosal tissues. The regulatory approval was supported by data from the GEM-1/2 and GEM-3 clinical trials.

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FDA Approval Gene Therapy Genetic Disease Gene 130

Drug Discovery World

APRIL 14, 2023

Clinical trials are, undoubtedly, an essential part of the drug development process, providing us with valuable information about the safety and efficacy of new treatments and therapies. of participants 2 Covid-19 vaccination trials. By Lucy Radley , Senior Vice President, Head of European Regulatory Development at ProPharma Group.

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Clinical Trials Clinical Trials Trials Drugs 52

The Pharma Data

SEPTEMBER 14, 2020

. AstraZeneca has confirmed that clinical trials assessing its Oxford University partnered coronavirus vaccine AZD1222 have resumed in the UK, following a green light by the Medicines Health Regulatory Authority (MHRA). Source link.

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Vaccination Vaccine Trials Immune Response 52

Drug Discovery World

FEBRUARY 8, 2023

A diversity study has shown that clinical trials should use real world diseases epidemiology data, rather than US Census Bureau race and ethnicity data, to ensure enrolment reflects the populations affected by different diseases. It also showed that GSK trial enrolment for each condition differed by race and ethnicity.

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Clinical Research Research Clinical Trials Clinical Trials 97

pharmaphorum

DECEMBER 22, 2021

Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient. In an update posted today the pharma giant said “we do not yet have complete information and are actively working with the trial site investigator to understand what happened.”

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Gene Therapy Gene Trials Clinical Trials 59

XTalks

SEPTEMBER 17, 2021

After asking for a placebo-controlled Phase III study for the drug in April that upset the ALS Association, the FDA dialed back on the request this week, agreeing to accept only Phase II trial data for evaluation of Amylyx’s drug application. People with ALS and their loved ones cannot wait.”.

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Trials Drugs FDA Approval Clinical Trials 98

pharmaphorum

JUNE 11, 2021

Abel Archundia discusses how COVID-19 has highlighted the importance of securing clinical trials against cyber threats. However, data-intensive, decentralised clinical trials also present a major risk to healthcare participants through cybercrime and data privacy vulnerability.

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Clinical Trials Clinical Trials Trials Compliance 111

Cloudbyz

NOVEMBER 30, 2021

As a relatively young industry, it’s no wonder that the clinical trial industry tends to undergo rapid change. Decentralized Clinical Trials. Decentralized Clinical Trials (DCTs) are clinical trials that remove reliance on central research sites for clinical studies. Let’s look at the trends.

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Clinical Trials Clinical Trials Trials Clinical Research 52

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. The post bluebird seeks gene therapy trial restart after cancer scare appeared first on.

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Gene Therapy Gene Trials Gene Expression 53

pharmaphorum

MARCH 3, 2021

Novavax hopes its COVID-19 vaccine could be filed in the US in the second quarter, following a potential approval from the UK regulator in the coming weeks. The timetable for approval from the FDA depends on whether the regulator accepts data from the company’s trial in the UK, which could set up a potential Emergency Use Authorisation.

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Vaccination Vaccine Regulation Immune Response 116

XTalks

APRIL 3, 2024

Related: Novartis’ Fabhalta Gets FDA Approval for Rare Complement Blood Disorder The FDA’s green light for Voydeya comes three months after the Japanese Ministry of Health, Labour and Welfare (JHLW) became the first regulator in the world to back the therapy. AstraZeneca is also venturing into cell therapy and genetic medicine.

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FDA Approval Sales Trials Development 102

XTalks

NOVEMBER 7, 2022

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Regulators may ask at some point to provide that documentation, so it’s important to track that across the study.

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Clinical Trials Clinical Trials Trials FDA Approval 98

XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Surrogate endpoints were used as the basis for approval of 45 percent of new drugs reviewed by the FDA between 2010 and 2012.

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Trials Gene Hormones Clinical Trials 98

Drug Discovery World

OCTOBER 3, 2023

Participants in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot programme will be able to obtain frequent advice and regular ad-hoc communication with FDA staff to address product development issues. The FDA will be accepting applications to the START programme between 2 January 2024 and 1 March 2024.

Clinical Trials 52

Clinical Trials Clinical Trials Production Regulation 52

Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. Many monoclonal antibodies are used to treat cancer by targeting specific cell structures regulating cell activity, causing the death of tumor cells.

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Vaccination Vaccine Protein Antibody 52

XTalks

OCTOBER 7, 2020

A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. One chapter discusses how to form early partnerships with regulators.

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Development Gene Therapy Clinical Trials Clinical Trials 98

Pharmaceutical Technology

MAY 24, 2023

The regulator granted RMAT designation based on the data obtained from the ongoing Phase I RP-L301 clinical trial. It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene.

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Gene Therapy Gene Licensing Licensing 130

Pharmaceutical Technology

DECEMBER 14, 2022

Bacteria naturally develop resistance to antibiotics over time, through genetic mutation, horizontal gene transfer between bacteria, or because of selective pressure. Local UK pharmacies are already reporting and forecasting future shortages of antibiotics to treat Strep A infections. Secondly, antibiotics are not a permanent solution.

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Bacteria DNA Clinical Trials Clinical Trials 130

Drug Discovery World

SEPTEMBER 9, 2022

Notably, it was a mixed week for PARP inhibitor olaparib, which was approved by regulator the MHRA for patients with early-stage breast cancer, but rejected by NICE for use on the NHS in metastatic prostate cancer. NICE rejects prostate cancer drug due to cost of genetic testing .

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Drugs Genetic Engineering Genetics Engineer 52

Drug Discovery World

JANUARY 11, 2024

The team, led by University College London (UCL), King’s College London and Moderna scientists, found that mRNA could be used to correct a rare genetic liver disease known as argininosuccinic aciduria in a mouse model. The researchers also noted that, mRNA-treated organs were very similar to those in the unaffected, control mice.

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Research Gene Therapy RNA Gene 59

The Pharma Data

OCTOBER 25, 2020

NASDAQ: PTCT), today announced the initiation of a registration-directed Phase 2/3 clinical trial to evaluate vatiquinone (PTC743) in patients with mitochondrial epilepsy, the highly morbid condition of refractory seizures in children with inherited mitochondrial disease. . SOUTH PLAINFIELD, N.J. , Peltz , Ph.D.,

Clinical Trials 52

Clinical Trials Clinical Trials Trials Regulation 52

pharmaphorum

NOVEMBER 19, 2020

The FDA will review data from the phase 3 COMET trial in patients with late-onset Pompe disease. Also in the file will be data from the phase 2 mini-COMET trial testing safety and efficacy in patients with infantile-onset Pompe disease previously treated with standard enzyme replacement therapy, alglucosidase alfa.

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Development Drugs Trials Clinical Trials 105