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.

Clinical Trials 111

Clinical Trials Clinical Trials Trials Compliance 111

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.

Genetic Engineering 130

Genetic Engineering Drugs Genetics Engineer 130

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.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Clinical Research 52

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. percent show less reliability). Optimal methods for bioinformatic analysis are still needed.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

Drug Discovery World

JANUARY 23, 2024

Sano Genetics, a software company working to accelerate precision medicine research, has raised $11.4 When it comes to precision medicine, which requires understanding the genetics of patients and tailoring drugs to suit them, it can be even more difficult for researchers to find the right patients for a trial.

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

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.

Development 130

Development Clinical Trials Clinical Trials FDA Approval 130

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.

Clinical Research 97

Clinical Research Research Clinical Trials Clinical Trials 97

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.

Regulation 162

Regulation Trials Clinical Trials Clinical Trials 162

XTalks

DECEMBER 28, 2023

BillionToOne, a company specializing in molecular diagnostics, has partnered with Janssen Research & Development, LLC, a Johnson & Johnson division, to conduct the AZALEA Phase III clinical trial globally. The trial is set to occur across the US, the European Union (EU) and selected global locations.

Trials 97

Trials Clinical Trials Clinical Trials Antibody 97

Drug Discovery World

MAY 17, 2024

Deepika Khedekar , Associate Centralized Clinical Trial Lead at IQVIA Inc, looks at the challenges of utilising artificial intelligence (AI) in oncology clinical research. MIT research 4 tells us a hard truth: about 95% of these trials fall short, leaving behind a trail of broken hopes for cancer patients each year.

Trials 59

Trials Clinical Trials Clinical Trials Protein 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

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.

FDA Approval 130

FDA Approval Gene Therapy Genetic Disease Gene 130

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

Trials 52

Trials Clinical Trials Clinical Trials Medicine 52

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

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.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Advarra

JANUARY 11, 2024

Personalized medicine using genetic resequencing techniques, such as cell and gene therapies, enables researchers to create more customized therapies for combatting cancer. Many monoclonal antibodies are used to treat cancer by targeting specific cell structures regulating cell activity, causing the death of tumor cells.

Vaccination 52

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.

Development 98

Development Gene Therapy Clinical Trials Clinical Trials 98

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?

Gene Therapy 98

Gene Therapy Gene Trials Clinical Trials 98

Worldwide Clinical Trials

DECEMBER 12, 2022

Genetic testing provides patients with a diagnosis for their illness, helps patients and family members to understand risks of developing new diseases, and can be used to support clinical trial advancement. What are the benefits and limitations to using genetic testing?

Clinical Trials 130

Clinical Trials Clinical Trials Genetics Trials 130

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.

Gene Therapy 130

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.

Bacteria 130

Bacteria DNA Clinical Trials Clinical Trials 130

Drug Discovery World

JUNE 2, 2023

Health data generated by hospitals, healthcare providers, research organisations and clinical trials hold the potential to advance research and innovation, improve patient care and accelerate drug development. The post Partnership hopes to accelerate cancer data research appeared first on Drug Discovery World (DDW).

Research 52

Research Compliance Clinical Trials Clinical Trials 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.

Research 59

Research Gene Therapy RNA Gene 59

Drug Discovery World

OCTOBER 18, 2023

4 Unfortunately, many AAV-based gene therapies fail during clinical trials, despite them appearing safe and efficacious in preclinical studies. 3 The world’s confidence in the future of these therapies is also clear, as the global AAV vector manufacturing market has a projected compound annual growth rate (CAGR) of 22.5%

Gene Therapy 64

Gene Therapy Gene Clinical Trials Clinical Trials 64

WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. WCG has many ways to support cell and gene therapy clinical trials for lupus.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials FDA Approval 52

pharmaphorum

DECEMBER 22, 2021

In August, we spoke to Alastair Kent, former director of the Genetic Alliance UK and independent patient advocate. Tackling the ‘disease of systemic racism’ in clinical trials. True patient involvement is inclusive, but we know that diversity within clinical trials is a problem. Under EU regulation No.

Clinical Trials 105

Clinical Trials Clinical Trials Trials Medicine 105

Drug Discovery World

JANUARY 30, 2024

“Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Joel Eichmann, Co-Founder and Managing Director, Green Elephant Biotech, agrees that prices need to come down significantly for CGT to be widely available.

Gene Therapy 71

Gene Therapy Manufacturing Gene Clinical Trials 71

Drug Discovery World

APRIL 5, 2023

The drug, which is now called NXP800 and has already entered a Phase I clinical trial, was discovered by scientists at The Institute of Cancer Research, London. We hope that our drug goes on to show effectiveness in clinical trials.” It also supports the drug’s potential to treat other cancers.

Drugs 52

Drugs Scientist Clinical Trials Clinical Trials 52

Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Drug Discovery World

JANUARY 23, 2023

Armed with this understanding, the researchers further demonstrated that the genetic inhibition of DGAT1 suppresses radioresistance. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1.

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

pharmaphorum

NOVEMBER 19, 2020

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. European regulators began their review of the data last month.

Development 105

Development Drugs Trials Clinical Trials 105

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.

Gene Therapy 52

Gene Therapy Gene Trials Clinical Trials 52

XTalks

JANUARY 26, 2023

The US Food and Drug Administration (FDA) recently granted approval for the Bruton tyrosine kinase inhibitor (BTKi) Brukinsa (zanubrutinib) based on the results from two Phase III clinical trials. Diagnosis is confirmed by a differential complete blood count and genetic testing.

FDA Approval 97

FDA Approval Life Science Clinical Trials Clinical Trials 97

pharmaphorum

OCTOBER 26, 2022

Less than a week before Ipsen’s palovarotene was due to go in front of an FDA advisory committee, the meeting has been called off following a request by the regulator for more data. The drug failed a futility test in a pivotal trial and was also placed under a partial clinical hold by the FDA while it probed the drug’s safety.

Drugs 59

Drugs Genetics Marketing Trials 59

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). The move follows a green light by the UK's MHRA.

Vaccination 52

Vaccination Vaccine Trials Immune Response 52

pharmaphorum

OCTOBER 7, 2020

There is enormous power in this genetic tool, which affects us all. Together, they recreated the process in a test tube, refined the molecular components so they were easier to use, and in a landmark study showed it could be reprogrammed to rewrite genetic sequences. What started as a curiosity?driven, million) Nobel Prize award. “In

Gene Editing 105

Gene Editing Bacteria DNA Gene 105