XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. For rare disease studies where there is little or no clinical trial experience, outcome assessments would have to be validated within a clinical development program.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

APRIL 5, 2023

It’s a multifaceted issue and we perhaps need to go beyond our comfort zone in science and clinical development to understand what barriers patients are really facing. But I think we’ve never had more tools than we have now in synthetic biology, genetic engineering, enhanced potency design switches and CRISPR.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

pharmaphorum

OCTOBER 6, 2020

The European Medicines Agency has begun a second “rolling review” of a potential coronavirus vaccine jointly developed by BioNTech and Pfizer, setting up a race with a rival from AstraZeneca and Oxford University. According to latest figures from the World Health Organization, there are 42 potential COVID-19 vaccines in clinical development.

Vaccination 59

Vaccination Vaccine RNA Immune Response 59

pharmaphorum

NOVEMBER 18, 2021

RNAi uses small RNA molecules to bind to messenger RNA, which is responsible for synthesising proteins, and either destroy it or recruit other cellular factors that regulate its activity. If approved, it could become a $500 million-a-year product, according to the company.

Gene Silencing 52

Gene Silencing RNA Protein Gene 52

pharmaphorum

NOVEMBER 9, 2020

Results will be discussed with regulators worldwide and the European Medicines Agency has already begun its rolling review of data to hasten a decision on the clinical trial dossier. The independent data monitoring committee that conducted the interim analysis said that there had been no serious safety concerns identified.

Vaccination 98

Vaccination Vaccine Clinical Trials Clinical Trials 98

Advarra

NOVEMBER 29, 2022

However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines. Many companies are unable to develop these niche therapies because of the associated costs and limited financial return.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

pharmaphorum

SEPTEMBER 8, 2021

The orally-active drug can be used in patients aged 12 and over after two other systemic therapies have been tried, and is thought to work by downregulating inflammatory immune cells and boosting the levels of T cells that help to regulate the immune system. KD033 is an anti-PD-L1/IL-15 fusion protein and is currently in a phase 1 trial.

Drugs 52

Drugs Sales Immune Response Marketing 52

Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Source: Medicines in Development | 2020 Update: Cell and Gene Therapy , PhRMA.

Gene Therapy 155

Gene Therapy Gene Packaging Packaging 155

Drug Discovery World

MARCH 2, 2023

Positive early signs for precision cancer therapies In the first presentation, Dr Fiona McLaughlin, CSO, provided an update on Avacta’s oncology candidates that are currently in clinical development. She explained the mechanism by which AVA6000 can provide more precise treatment than current chemotherapies.

Trials 52

Trials Development Genetic Analysis Genetics 52

The Pharma Data

DECEMBER 17, 2020

Cadavid will be responsible for the strategy, direction and execution of the company’s clinical development programs. Prior to Fulcrum, Cadavid held several leadership positions at Biogen, including Senior Medical Director of the multiple sclerosis clinical development group. Immune Regulation – U.K.-based

Development 52

Development Gene Therapy Engineer Life Science 52

Delveinsight

AUGUST 20, 2020

regulator that had earlier told there was no requirement of an AdComm for the drug, aka, valrox, has issued a complete response (CRL) letter, which could cause a significant delay for any future approval, and canceled what would have been the first-ever approval for gene therapy in the bleeding disorder. J&J has agreed to pay USD 52.50

Gene Therapy 52

Gene Therapy Research Gene Antibody 52

Drug Discovery World

SEPTEMBER 14, 2023

joined Pfizer’s Executive Leadership Team as Chief Oncology Research and Development Officer and Executive Vice President reporting to Chairman and Chief Executive Officer, Albert Bourla. In July 2023, AstraZeneca and Ionis Pharmaceuticals expanded their existing collaboration on eplontersen to also include Latin America. Chris Boshoff, M.D.,

Drugs 59

Drugs Vaccination Vaccine Medicine 59

The Pharma Data

DECEMBER 9, 2020

Using state-of-the-art genetic approaches and imaging methods, the team has provided a comprehensive insight into defective neuro-glia interactions and their consequences for neurotransmitter homeostasis. This work has led to the identification of molecular components regulating crucial processes impaired in depression.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

Drug Discovery World

JULY 11, 2022

As the field matures and the risks are better understood, regulators continue to streamline duplicative and burdensome oversight efforts 5. Regulatory frameworks need to be established to reduce these barriers and ensure patient safety while also facilitating expeditious development of these complex and innovative products 6.

Gene Therapy 52

Gene Therapy Gene Development Production 52

The Pharma Data

JULY 6, 2021

GlaxoSmithKline plc (LSE/NYSE: GSK) and Alector (Nasdaq: ALEC), today announced a strategic global collaboration for the development and commercialisation of two clinical-stage, potential first-in-class monoclonal antibodies (AL001 and AL101) designed to elevate progranulin (PGRN) levels. ” Arnon Rosenthal, Ph.D.,

Genetics 52

Genetics Gene Antibody Development 52

XTalks

NOVEMBER 20, 2023

The newly approved Truqap and Faslodex combo is restricted to patients with a genetic alteration in one or more of the following PI3K/AKT pathway genes: PIK3CA , AKT1 or PTEN. PI3K/AKT is a key intracellular signaling pathway that regulates cell survival and proliferation. It was also given FDA priority review.

HR 98

HR FDA Approval Gene Trials 98

Drug Discovery World

MARCH 6, 2024

The development of effective new disease-modifying treatments in neurodegenerative and neuropsychiatric disorders has been hindered by their inherent genetic complexity, environmental influences, and clinical variability. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.

Genome 65

Genome Genomics Clinical Trials Clinical Trials 65

The Pharma Data

DECEMBER 15, 2020

Contribution of these robust data sets from industry led trials is critical to TOMI-T1D’s work in developing innovative and quantitative tools that can facilitate clinical development efforts and be endorsed by regulators for future use by the pharmaceutical industry to optimize the design of future clinical trials.

Development 40

Development Insulin Drugs Clinical Trials 40

Advarra

AUGUST 10, 2023

“I am very excited for the field because I feel like we’re beginning to get to a critical mass, where a single method or product can be deemed safe and then adapted for many uses,” said Dr. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

Gene Therapy 52

Gene Therapy Development Gene Drugs 52

pharmaphorum

APRIL 22, 2021

Anne Kerber, VP, Head of Clinical Development, Europe at Kite, a Gilead Company, explains how the company works with researchers and physicians across Europe to expand patient access to cell therapy. This thought leadership series has been paid and developed by Kite, a Gilead Company. But we can’t – and don’t – do this alone.

Research 98

Research Clinical Trials Clinical Trials Trials 98

XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. There are around 300 million people worldwide living with a rare disease.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials Gene 52

The Pharma Data

SEPTEMBER 8, 2020

Together, these nine companies have collectively developed more than 70 novel vaccines that have helped to eradicate some of the world’s most complex and deadly public health threats, underscoring their experience in clinical development and regulatory rigor, as well as their longstanding commitments to patient safety and public health.

Vaccination 52

Vaccination Vaccine Containment Clinical Trials 52

The Pharma Data

SEPTEMBER 8, 2020

Together, these nine companies have collectively developed more than 70 novel vaccines that have helped to eradicate some of the world’s most complex and deadly public health threats, underscoring their experience in clinical development and regulatory rigor, as well as their longstanding commitments to patient safety and public health.

Vaccination 52

Vaccination Vaccine Containment Clinical Trials 52

XTalks

APRIL 12, 2021

The gene therapy company was focused on applying adeno-associated virus (AAV) vectors to developing a one-time treatment for patients with Parkinson’s disease who have specific genetic mutations. But just three months later, Voyager faced another stumbling block in the form of an FDA-mandated clinical hold on the trial.

Gene Therapy 96

Gene Therapy Gene Trials Development 96

Drug Discovery World

FEBRUARY 15, 2023

However, over the last few decades, it has been widely accepted that interaction between the tumour and host immune response plays a critical role in regulating tumour biology and hence its progression. The long-term objective is to offer bespoke and personalised therapy for individuals based on their genetic profile and tumour signature.

Research 52

Research In-Vitro In-Vivo Development 52

The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

Roots Analysis

OCTOBER 30, 2023

These RNA based therapeutics play a crucial role in protein production and regulation of gene functions. It is worth highlighting that one such therapeutic, ATRY1923 is currently in phase III of clinical development and has received fast track and orphan drug designations from the US Food and Drug Administration.

RNA 40

RNA Vaccination Vaccine Development 40

The Pharma Data

APRIL 8, 2021

Roche will present data from five studies from the EVRYSDI clinical development programme, which was designed to represent a broad spectrum of people living with SMA. The full range of data from Roche’s clinical development programme in neuroscience being presented at 2021 AAN include: Medicine and/or Therapeutic Area.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Antibody 52

The Pharma Data

SEPTEMBER 20, 2020

Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C ?inhibitor To learn more about Amgen ‘s innovative pipeline with diverse modalities and genetically validated targets, please visit AmgenOncology.com.

Medicine 52

Medicine Production Sales Clinical Trials 52

The Pharma Data

SEPTEMBER 16, 2020

Amgen’s clinical development team and clinical investigators, will discuss Phase 1 data being presented on the Company’s investigational KRAS G12C ?inhibitor To learn more about Amgen’s innovative pipeline with diverse modalities and genetically validated targets, please visit www.AmgenOncology.com.

Production 52

Production Sales Clinical Trials Clinical Trials 52

The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. About Spinal Muscular Atrophy SMA is the leading genetic cause of infant death.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

The Pharma Data

OCTOBER 28, 2020

Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. About Ultragenyx.

Protein 40

Protein Production Development Drugs 40

XTalks

DECEMBER 24, 2020

However, for virologists like Lori Frappier, PhD, professor in the department of molecular genetics at the University of Toronto, the outbreak of SARS-CoV-2 was less of a surprise and more of an inevitability. Omer maintains that the solution to this challenge is something that’s already in practice by developers of the mRNA vaccines.

Life Science 111

Life Science Vaccination Vaccine Gene 111

The Pharma Data

MARCH 30, 2021

Basel, 30 March 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission (EC) has approved Evrysdi (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients two months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.

Protein 52

Protein Medicine Trials Clinical Trials 52

Pharmaceutical Technology

MARCH 6, 2023

However, struggles to prove to regulators the validity of or need for drugs in this realm still serve as a major barrier to success. However, Kaeberlein explains, “The challenge with that is I think we’re pretty far away from the FDA recognizing those biomarkers as true surrogate endpoints for clinical trial.”

Drugs 363

Drugs Regulation Clinical Trials Clinical Trials 363

XTalks

SEPTEMBER 14, 2020

Unlike other candidate vaccines, CoronaVac is an inactivated whole virus vaccine developed through the same process as has long been used to make influenza vaccines, among many others. Several laboratories have already shown that they can detect genetic material from the virus in wastewater.

Vaccination 92

Vaccination Vaccine Antibody Life Science 92