LifeSciVC

APRIL 24, 2024

Additionally, for illustrative reasons this is geared towards a single target / product focus vs. broader platform diligence, though many of these mental models will apply for selecting targets and indications for a platform. with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g., in liver, in CNS)?

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Drug Target Review

SEPTEMBER 1, 2023

Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.

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Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

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FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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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.

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PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.

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Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Operationalizing these trials requires proactive and flawless management at every stage.

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PPD

OCTOBER 8, 2024

With FSO, all clinical trial tasks (e.g., across protocols, medicinal products, a portfolio of studies or the entire company. In a hybrid model, one or more FSP offerings are added to a new or existing FSO arrangement to optimize clinical trial operations or address unforeseen circumstances and changing demands.

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thought leadership

JULY 2, 2024

Change in the Submission of the Summary Notification Information Format The Summary Notification Information Format Form As of January 31, 2023, Sponsors are required to submit a Clinical Trial Application (CTA) in the EU through the Clinical Trial Information System (CTIS).

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PPD

JANUARY 15, 2025

As clinical trials become increasingly complex, particularly in decentralized trials and rare disease studies, sponsors experience increased challenges in site selection, forecasting and resourcing, and patient recruitment and enrollment.

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Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects. 3D rendering of Antibody Drug Conjugate Molecules.

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Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

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Codon

JULY 14, 2024

receives a blood transfusion or a blood product every two seconds. But currently, traditional blood transfusions face significant challenges, including a dwindling pool of donors and a limited shelf life for stored blood products. However, the subsequent death of another patient thrust Denys into a contentious trial.

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FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations.

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Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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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.

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The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.

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Therapies Clinical Trials Small Molecule Immune Response 52

Advarra

MARCH 20, 2023

The Food and Drug Administration (FDA) recently released new guidance regarding cellular and gene therapy products, one of which may significantly impact early-phase clinical trials of such products. The Benefits of the Umbrella Trial There are many potential benefits to the umbrella trial.

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FDA Law Blog: Drug Discovery

MARCH 26, 2024

Sasinowski — On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here ). The primary basis for approval, like other drugs for DMD, was based on a single placebo-controlled randomized trial. By Charles G. Raver & James E.

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Drug Target Review

AUGUST 6, 2024

Liver Fibrosis : Targeted therapies using ADCs could help reduce liver fibrosis and prevent the progression to cirrhosis. Expedited programs like the FDA’s Breakthrough Therapy Designation and Fast Track can shorten development timelines and reduce risk.

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PPD

MARCH 13, 2024

In every early phase clinical trial, the transportation, chain of custody, environmental monitoring and subsequent analysis of biological samples are the cornerstones of a successful, robust and quality outcome. This significance and focus are ever more apparent when the starting cellular material is imperative to drug product success.

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Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

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Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. Additional trials (e.g., Join the club.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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FDA Law Blog: Drug Discovery

OCTOBER 11, 2023

Snow — On September 18, 2023, FDA published an updated, final iteration of guidance for immediate implementation entitled, “ Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies.” hurricane) or public health emergency (e.g.,

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Clinical Trials Trials FDA Drug Development 64

Advarra

JUNE 13, 2023

Research in gene therapies and genetically engineered drugs and vaccines are growing exponentially, and will only continue to become more popular. The accelerating gene therapy market is expected to grow globally by 16.6% An IRB tends to get the most attention since all human trials need IRB approval. between 2020-2027.

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Therapies Research Clinical Trials Trials 52

Drug Target Review

SEPTEMBER 6, 2024

What trends are driving the increased use of in vivo delivery methods in gene therapy? Finally, the market demand has increased as clinical trials are advancing towards more advanced phases and more vectors are being approved for commercial use. How are new therapeutic areas emerging as potential targets for in vivo gene therapy?

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The Premier Consulting Blog

APRIL 18, 2024

Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. The cell or gene therapy should be pharmacologically active in the animal model. Biodistribution of the product (transgene/gene or cells) should be evaluated. How long does the study need to be?

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Therapies Small Molecule Production Clinical Trials 52

Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.

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Fierce BioTech

OCTOBER 6, 2023

Bringing a New Heart Failure Therapy to the Clinic swheeler Fri, 10/06/2023 - 14:56 Tue, 11/28/2023 - 11:00 Resource Type Webinar Phil Morton, MD Dr. Takehiko Kaneko, MD Duration 60 minutes Commercializing biopharmaceuticals and advanced therapies requires robust, standardized manufacturing processes.

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The Premier Consulting Blog

OCTOBER 21, 2024

Accelerated approval is an expedited regulatory pathway designed to hasten the availability of drugs (including biologics) that treat serious conditions, offer advantages over existing therapies, and address unmet medical needs. In essence, these trials are the final step that turns provisional approval into full approval.

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Trials FDA Clinical Trials Disease 52

Advarra

JUNE 20, 2023

years , with biotechnology-derived products potentially adding another year or more. Innovation Organizations conducting oncology clinical trials face challenges distinct from the rest of the research community. cell and gene therapies), with other therapeutic areas then pushing it further.

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Conversations in Drug Development Trends

MAY 23, 2024

By: Sarah Bly, Regulatory Science and Innovation and Matt Cooper, Executive Director, Therapeutic Strategy Lead, Oncology The European Union (EU) presents a unique set of regulatory challenges and opportunities for clinical trials in oncology. Increased Transparency : Ensures that information on clinical trials is more accessible.

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Trials Clinical Trials Therapies Treatment 52

DrugBank

SEPTEMBER 26, 2024

Due to the less extensive clinical trial requirements and the competitive nature of the biosimilar market, these biologics can be produced and marketed at significantly lower prices than their reference counterparts. The primary advantage is cost-effectiveness.

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Advarra

JUNE 13, 2024

Recombinant DNA technologies and genetically modified biological agents are being adapted for a wide scope of therapeutic applications, and their use is becoming increasingly common in clinical trials. How can I protect myself from exposure? What should I do if I’m exposed?

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Drug Target Review

JANUARY 8, 2024

Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.

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