Drug Target Review

MARCH 21, 2024

Dystrophin is mutated in Duchenne muscular dystrophy (DMD), a rare disease that causes progressive muscle weakness and atrophy. There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin. Of note, mRNA is redosable as opposed to AAV.

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Drug Target Review

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing. We believe we are at a breakthrough moment in Alzheimer’s disease research.

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Disease Clinical Trials Therapies Science 59

Drug Target Review

FEBRUARY 2, 2024

What specific limitations in mirroring diseases in humans are commonly observed when using animal models for preclinical research? Our cells are fully differentiated, non-tumour, primary cells, which have a more natural growth pattern and gene expression profile, and the disease signature is often carried over epigenetically.

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Broad Institute

OCTOBER 11, 2023

He also read everything he could about her disease, including emerging evidence that suggested that the immune system could recognize and kill Merkel cell carcinoma. His mother had a presentation of the disease that suggested her immune system was already on the job. This is what we need for patients,” Manguso said.

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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Therapies Treatment Engineering Molecular Biology 59

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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Therapies Immune Response DNA Virus 69

The Pharma Data

APRIL 7, 2022

This unprecedented CMS decision effectively denies all Medicare beneficiaries access to ADUHELM ® (aducanumab-avwa), the first and only FDA-approved therapy in a new class of Alzheimer’s drugs. ADUHELM is indicated for the treatment of Alzheimer’s disease. It may also limit coverage for any future approved treatment in the class.

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Treatment Therapies Disease FDA Approval 52

The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc.

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Therapies Virus Disease Licensing 52

Drug Target Review

OCTOBER 26, 2023

Genome engineering and gene therapies that manipulate DNA sequences in cells have driven a biotechnological revolution over the past decade. 1 Adeno-associated virus (AAV) vectors are the leading platform for in vivo gene delivery for the treatment of various human diseases. Molecular Therapy 20 , 1831-1832 (2012).

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Engineering Virus Therapies Immune Response 105

The Pharma Data

JANUARY 11, 2021

Pfizer has started dosing in a phase 3 study evaluating its investigational gene therapy, PF-06939926, in young male patients with Duchenne muscular dystrophy (DMD). The agency previously granted the gene therapy Orphan Drug and Rare Pediatric Disease designations in 2017. Source link.

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Drug Target Review

AUGUST 16, 2023

Furthermore, it successfully distinguished early-stage cancer samples from those with common diseases, laying the foundation for a robust data platform for further in-depth analysis. This in-depth understanding of the disease could aid in selecting targeted therapies for individual patients, further enhancing treatment outcomes.

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DNA Disease Therapies Treatment 98

Drug Target Review

JANUARY 15, 2024

Cancer is a disease driven by variable genetic mutations. Immunotherapy has become a cornerstone of cancer therapy and significantly improved patient outcomes. However, an unmet need persists in patient populations that do not respond to these therapies where ~50-70 percent relapse or become refractory to them.

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Treatment Immune Response Therapies Clinical Trials 105

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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Therapies Virus DNA Compliance 52

Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

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Disease Clinical Trials FDA Approval FDA 52

KIF1A

JUNE 17, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. #ScienceSaturday posts share exciting scientific developments and educational resources with the KAND community. What’s a pre-print?

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Laboratories Therapies Disease Research 98

Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Now, FDA’s CBER is setting its sights on making ambitious strides toward tackling rare diseases.

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Drug Development Disease Therapies Engineering 52

Drug Target Review

OCTOBER 17, 2023

Metabolic changes and tumourigenicity Cancer is a multi-faceted disease which has previously been seen as a disorder in cell proliferation, the increase in cell numbers resulting from cell division. Diagnosis and therapy The study’s findings have promising implications for liver cancer therapy. Available from: [link]

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The Pharma Data

DECEMBER 18, 2020

18 December 2020 — The US Food and Drug Administration (FDA) has requested further clarifying analyses of clinical data, to complete its review of the New Drug Application (NDA) for roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor for patients with anemia of chronic kidney disease (CKD).

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Disease FDA FDA Approval Treatment 52

The Pharma Data

JANUARY 12, 2021

In December, life sciences giant Bayer launched a cell and gene therapy platform within its pharmaceutical division in order to become a leading company within a rapidly emerging and evolving field that offers the potential of life-saving therapies. Photo courtesy of Bayer.

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Therapies Licensing Licensing Pharmaceuticals 52

The Pharma Data

JUNE 1, 2022

The United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A, a rare and life-threatening bleeding disorder, based on data from the pivotal XTEND-1 P hase 3 stud y.

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Therapies FDA Science Treatment 52

The Pharma Data

MARCH 22, 2021

Basel, 22 March 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced the decision to discontinue dosing in the Phase III GENERATION HD1 study of tominersen in manifest Huntington’s disease (HD). In December 2017, Roche licensed the investigational molecule from Ionis Pharmaceuticals. About Huntington’s disease.

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Disease Clinical Trials Pharmacokinetics Treatment 52

Advarra

JUNE 12, 2023

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What Does an IBC Review? The post IBC vs. IRB: What’s the Difference? appeared first on Advarra.

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Engineering Therapies Regulations Research 98

Drug Target Review

NOVEMBER 7, 2024

Introduction Therapeutic antibodies have proven to be indispensable medicines for addressing the most debilitating diseases. Agonist antibodies in immunological diseases Agonist antibodies targeting immune checkpoint pathways are increasingly recognised and investigated for their potential in immunological diseases.

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Disease Immune Response Regulations Engineering 52

The Pharma Data

DECEMBER 15, 2020

A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.

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Disease Clinical Trials Small Molecule Trials 52

Drug Target Review

AUGUST 9, 2023

Published on bit.bio’s website, this article authored by Dr Tony Oosterveen dives into the close relationship between FTD and ALS with a focus on mechanisms underlying the neuropathology of both diseases. ALS is the most common motor neuron disease and is estimated to affect more than 80,000 people worldwide.

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RNA Regulations Disease DNA 98

Drug Target Review

SEPTEMBER 26, 2023

Human derived induced pluripotent stem cells (hiPSCs) have revolutionised research and are increasingly used for toxicology screening and disease modelling. Early detection of neurotoxicity induced by potential new therapies is a major challenge, and hiPSC-neuronal cells may provide a solution. 2017 Jan;155(1):234–47.

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The Pharma Data

JANUARY 17, 2021

The companies will evaluate ViGeneron’s proprietary, intravitreally injected vgAAV vectors for delivering a novel therapeutic protein to develop a gene therapy treatment for a highly prevalent eye disease. There is significant unmet medical need for a sustained therapy to treat eye diseases. MUNICH, Germany, Jan.

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Therapies Research Virus Disease 40

The Pharma Data

MAY 27, 2022

In December 2017, Janssen Biotech, Inc. “With today’s approval of cilta-cel in Europe, we are pleased to be delivering a new approach to the way in which healthcare professionals can tackle, and hopefully one day overcome, this complex disease.”. of patients with RRMM responding to therapy (98 percent overall response rate ORR ).1,6

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Therapies Treatment Pharmaceutical Companies Nurses 52

Drug Target Review

SEPTEMBER 22, 2023

Organoid technologies are becoming an invaluable solution for preclinical research, with the ability to augment the development of personalised medicine, drug discovery and gene therapies. Organoids for cell therapy and drug discovery. CRISPR engineering in organoids for gene repair and disease modelling. 2023 May;30(5):571–91.

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Clinical Research Research Drug Development Molecular Biology 116

Drug Target Review

DECEMBER 16, 2024

Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.

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Therapies Clinical Trials Treatment Trials 52

Drug Target Review

OCTOBER 4, 2023

Antibody-drug conjugates (ADCs) have been a groundbreaking approach to cancer treatment with their ability to deliver cytotoxic drugs directly to diseased cells while sparing healthy tissues. To overcome these obstacles, combination therapy, which delivers multiple small molecules, has emerged as a potential solution. 1 , 33–43 (2018).

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Small Molecule Drugs Immune Response Targeted Protein Degradation 126

New Drug Approvals

NOVEMBER 13, 2023

PATENT WO2023214314 VODOBATINIB FOR REDUCING PROGRESSION OF PARKINSON’S DISEASE (wipo.int) Vodobatinib (N’-(2-chloro-6-methylbenzoyl)-4-methyl-3-[2-(3-quinolyl) ethynyl]-benzohydrazide), a c-Abl inhibitor is represented by Formula I (referred hereinafter interchangeably as vodobatinib or compound of Formula International Publication Nos.

Treatment 62

Treatment Disease Therapies Production 62

The Pharma Data

MARCH 16, 2022

(Nasdaq: BIIB) announced that The Journal of Prevention of Alzheimer’s Disease (JPAD) today published a peer-reviewed manuscript detailing data from the pivotal Phase 3 EMERGE and ENGAGE trials for ADUHELM ® (aducanumab-avwa) 100 mg/mL injection for intravenous use in early Alzheimer’s disease.

Disease 52

Disease Clinical Trials Trials Treatment 52

The Pharma Data

SEPTEMBER 29, 2021

In 2017, an alarming 1.2 to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. 1 in 3 adults is at risk for the disease.

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Clinical Development Disease Clinical Trials Trials 52

The Pharma Data

JULY 21, 2021

Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company focused on the development and commercialization of prophylactic vaccines for infectious diseases with significant unmet medical need, and Pfizer Inc. Food and Drug Administration (FDA) in July 2017 3. About Lyme Disease. Juan Carlos Jaramillo M.D.,

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Vaccine Disease Trials Protein Expression 52

Drug Target Review

SEPTEMBER 4, 2023

There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.

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Drug Target Review

MAY 23, 2023

Inflammatory bowel disease (IBD) is an umbrella term that describes complex disorders that cause chronic inflammation in the digestive tract with alternating periods of relapse and remission. Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. More than 6.8

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