Drug Discovery World

APRIL 18, 2023

AVA3996 is in pre-clinical development with the aim of submitting an Investigational New Drug (IND) application to allow clinical development to begin during 2024. “We are focused on accelerating AVA3996 through to IND filing as soon as possible so that it can follow AVA6000 into the clinic next year.”

Therapies 242

Therapies Clinical Development Treatment Marketing 242

Drug Discovery World

AUGUST 30, 2022

The Phacilitate Advanced Therapies Europe 2022 conference will take place from 30 August to 1 September at the Royal Lancaster in London, UK. The conference is the European twin to Phacilitate Advanced Therapies Week, an event held in January in Florida, US. Improving patient experience within clinical trials and long term follow up.

Therapies 246

Therapies Hospitals Clinical Trials Clinical Development 246

Drug Discovery World

FEBRUARY 14, 2024

AlphaMedix is a targeted alpha therapy currently in Phase II clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) that serves as an in vivo generator of alpha particles. In the Phase I study, treatment was well-tolerated, with a response rate of 62.5%

Therapies 130

Therapies FDA Approval FDA Clinical Trials 130

Drug Discovery World

DECEMBER 8, 2023

ONK Therapeutics and NAYA Biosciences have agreed to develop a combination therapy of ONK’s optimally-engineered natural killer (NK) cell therapies and NAYA’s FLEX-NK bispecific antibodies. The post Partners explore NK cell therapy and bispecific antibodies combination appeared first on Drug Discovery World (DDW).

Therapies 130

Therapies Clinical Trials Engineering Clinical Development 130

Drug Discovery World

MARCH 25, 2024

Cardior is focused on the discovery and development of therapies that target RNA to prevent, repair and reverse diseases of the heart. We have been impressed by the scientific work carried out by the Cardior team, especially on CDR132L, which has a distinctive mode of action and potential to become a first-in-class therapy.”

Therapies 130

Therapies RNA Disease Trials 130

Drug Discovery World

JANUARY 22, 2024

The UK is cementing its position as a global leader in cell and gene therapy (CGT), according to a new report released today by the UK BioIndustry Association (BIA), in collaboration with Citeline. The UK has 84 drugs in clinical development, surpassing all other European countries, with 23 CGTs are already approved for patient use in the UK.

Therapies 130

Therapies Clinical Trials Trials Clinical Development 130

Drug Discovery World

FEBRUARY 1, 2024

Vivet is a clinical stage biotech company developing gene therapies for rare inherited metabolic disorders. CrisTauX will enable accelerated clinical development of VTX-806 upon completion of the programme. Vivet Therapeutics has received €4.9

Therapies 130

Therapies Treatment Disease Virus 130

Drug Discovery World

JUNE 19, 2024

DDW Editor Reece Armstrong speaks to Jasminka Taleska , Director, RLT HCS Readiness and Policy at Novartis, about the opportunities radioligand therapies (RLTs) represent in treating cancer patients. The post How ready are we for radioligand therapies? RA: What are the potentials of RLTs across cancer? JT: This is very important 6.

Therapies 148

Therapies Treatment Regulations Production 148

Drug Discovery World

JANUARY 5, 2023

CANbridge Pharmaceuticals has exercised its option to secure the exclusive global rights to develop, manufacture and commercialise a novel gene therapy to treat spinal muscular atrophy (SMA) from UMass Chan Medical School. . The benchmark therapy uses a vector similar to that used in the only gene therapy approved for SMA.

Therapies 130

Therapies Medical Schools Clinical Development Pharmaceuticals 130

Cytel

AUGUST 28, 2023

A clinical development plan — a comprehensive strategy for developing an investigational product through regulatory submission — is a critical component of drug development and helps ensure that new therapies are safe, effective, and of high quality.

Clinical Development 52

Clinical Development Drug Development Therapies Production 52

Drug Discovery World

DECEMBER 4, 2023

Qnovia has announced positive results from its first in-human study of QN-01, an inhaled smoking cessation therapy. QN-01 is designed to meet the urgent need for new safe and effective pharmacotherapies that address the shortcomings of currently available nicotine replacement therapies (NRTs).

Therapies 130

Therapies Treatment Pharmacokinetics Clinical Development 130

Drug Target Review

AUGUST 28, 2024

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

Therapies 114

Therapies Disease Engineering Medical Schools 114

Drug Discovery World

OCTOBER 3, 2023

The agency is inviting a limited number of sponsors to participate in the programme, which allows for more frequent communication with FDA staff to address clinical development issues. “We The post FDA pilot programme to boost rare disease therapies appeared first on Drug Discovery World (DDW).

FDA 130

FDA Disease Therapies Clinical Trials 130

Drug Discovery World

DECEMBER 19, 2022

Kite and Arcellx have entered a global strategic collaboration to co-develop and co-commercialise CART-ddBCMA for the treatment of patients with relapsed or refractory multiple myeloma. . The post Partnership to co-develop cell therapy for multiple myeloma appeared first on Drug Discovery World (DDW).

Therapies 130

Therapies Clinical Trials Engineering Clinical Development 130

thought leadership

AUGUST 26, 2024

In the dynamic world of pharmaceuticals and biotechnology, small Sponsor companies are often the unsung heroes driving innovative therapies through the complex journey from concept to clinic.

Clinical Development 40

Clinical Development Pharmaceuticals Therapies Compliance 40

Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). The post FDA approves gene therapy for rare neurological disorder appeared first on Drug Discovery World (DDW).

FDA Approval 130

FDA Approval FDA Therapies Hospitals 130

Drug Discovery World

MARCH 2, 2023

With our pre|CISION and Affimer platforms we have an unparalleled opportunity to develop really meaningful new cancer therapies, by developing targeted therapies, multi-specific immunotherapies, difficult to drug targets, and next-generation cell therapies.” The post Precision cancer therapies: Where are we?

Therapies 130

Therapies Treatment Trials Disease 130

Drug Discovery World

DECEMBER 20, 2022

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). . The therapy was also recently approved by the FDA for patients in the US. .

Therapies 130

Therapies Clinical Trials Licensing Licensing 130

Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? What opportunities and challenges are emerging and can therapies get to market faster? The deeper the understanding of product and process from a characterisation standpoint, the smoother the regulatory conversations and development pathway will be.” The solution?

Therapies 130

Therapies Clinical Trials Marketing Production 130

Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. Record breaking investments in 2021 fuel an industry on fire . billion raised.

Therapies 130

Therapies Clinical Trials Production Disease 130

Drug Discovery World

DECEMBER 14, 2023

“We are thrilled to see this level of clinical activity in such a heavily pre-treated patient population,” said John Houston, Chairperson, Chief Executive Officer and President at Arvinas. Vepdegestrant is the only PROTAC ER degrader in late-stage clinical development. The results show a 25.1%

Therapies 130

Therapies Trials Pharmacokinetics Treatment 130

Drug Discovery World

DECEMBER 21, 2022

Biopharmaceutical company Rezolute has initiated a Phase II proof-of-concept study for RZ402, a plasma kallikrein inhibitor (PKI) being developed as an oral therapy for the treatment of diabetic macular oedema (DMO). .

Therapies 130

Therapies Pharmacokinetics Clinical Development Treatment 130

Drug Discovery World

SEPTEMBER 4, 2024

EVO756 is a highly selective small molecule antagonist of mas-related G-protein coupled receptor X2 (MRGPRX2) and could offer the potential for once-daily oral administration without the side effects observed with other therapies.

Clinical Trials 130

Clinical Trials Trials Small Molecule Clinical Development 130

Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. The global cell and gene therapy (CGT) market reached a value of ~$4 billion in 2020 and is expected to grow to ~$34 billion by 2030 1.

Therapies 130

Therapies Clinical Trials Production Trials 130

Drug Target Review

JULY 31, 2023

Our inaugural report is a groundbreaking exploration of the remarkable advancements in cell and gene therapy that are revolutionising the field of drug discovery. Expert Insights We are honoured to have collaborated with renowned experts in the field of cell and gene therapy, who have generously shared their invaluable insights.

Therapies 98

Therapies Disease Clinical Development Hospitals 98

Drug Discovery World

APRIL 3, 2024

million from the Spanish Research Agency (AEI), under the call for grants for Public-Private Collaboration Projects, to accelerate the development of an innovative treatment aimed at controlling disabling and lethal haemorrhage.

Therapies 130

Therapies Laboratories Pharmacokinetics Treatment 130

Drug Discovery World

JULY 12, 2024

Following the success of therapies like Ozempic, it’s no surprise that type 2 diabetes research is being prioritised by biopharmaceutical companies. High levels of cancellations at Phase II not only increase the costs of clinical development, but also have a knock-on effect on the rate at which new therapies reach patients.

Research 148

Research Clinical Trials Clinical Development Trials 148

Broad Institute

NOVEMBER 2, 2023

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

Therapies 105

Therapies Research Small Molecule Clinical Trials 105

Drug Target Review

FEBRUARY 1, 2024

Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? Investors have historically taken a ‘guilty until proven innocent’ approach to investing in neuroscience therapies. All stakeholders have benefited from these innovations.

Therapies 104

Therapies Drug Development Disease Clinical Development 104

Drug Target Review

MAY 22, 2024

How does Circio’s circVec technology aim to enhance the potency and reduce the cost of current gold-standard gene therapy? There are three major reasons for why Circio has selected AATD as its lead gene therapy program. Firstly, there have been previous AAV gene therapy clinical studies in this indication that have shown promise.

Therapies 59

Therapies RNA Protein Expression DNA 59

Drug Discovery World

JULY 5, 2024

Long-term, 25% of treated patients treated with evenamide as an add-on therapy met the criteria for remission, a result that Stephen Marder, Distinguished Professor of Psychiatry at the Semel Institute of Neuroscience & Human Behavior, has described as “remarkable and unprecedented”.

Pharmaceuticals 265

Pharmaceuticals Clinical Development International Therapies 265

Drug Discovery World

JANUARY 23, 2024

What has been especially notable is the increase in solid tumour trials – these are likely to be the foundation of innovative cancer therapies, and investment in this area is a promising sign for patients,” explained Dr Gen Li, President, Phesi. Read the full report.

Disease 130

Disease Clinical Trials Research Trials 130

Drug Discovery World

JANUARY 11, 2024

Vice President, Clinical Development, Infectious Diseases at Moderna examines the data on cytomegalovirus (CMV), why attempts at vaccines for CMV have so far proved elusive and the emerging options to address this unmet need. In the first article, Allison August, M.D.,

Vaccine 148

Vaccine Clinical Development Drugs Marketing 148

The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.

Clinical Development 52

Clinical Development Therapies Clinical Trials FDA Approval 52

The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

Production 52

Production Therapies DNA Virus 52

DS in Pharmatics

JANUARY 7, 2023

Cell and gene therapies for treating multiple myeloma, such as CAR-T, have resulted in breakthrough health outcomes for patients. In the last decade, approval of cell and gene therapy (CGT) products has increased significantly.

Production 52

Production Therapies Clinical Development 52

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. 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.

Therapies 52

Therapies FDA Clinical Trials Production 52