Drug Target Review

SEPTEMBER 6, 2024

What trends are driving the increased use of in vivo delivery methods in gene therapy? Being a specialist lentiviral vector CDMO, at VIVEbiotech, this annual growth rate has been higher with a CAGR from 2020 to 2023 of almost a 50 percent. How are new therapeutic areas emerging as potential targets for in vivo gene therapy?

Therapies 59

Therapies Vaccine Marketing Disease 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.

Drugs 116

Drugs Clinical Trials Disease Science 116

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.

Treatment 52

Treatment Therapies Disease FDA Approval 52

Drug Target Review

NOVEMBER 7, 2023

To target proteins involved in causing or spreading disease, typically a drug will obstruct a protein’s active site so it cannot function. Proteins can become part of a cancerous tumour and allow for its unrestricted growth, cause diseases like Alzheimer’s , and build up in the heart to impact how it pumps blood around the body.

Therapies 59

Therapies Disease Science Treatment 59

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 Clinical Trials FDA Production 52

The Pharma Data

FEBRUARY 24, 2022

Bayer announced today the initiation of the CONFIDENCE study, a Phase II, three-arm study that will investigate simultaneous initial combination therapy with finerenone and the SGLT2 inhibitor empagliflozin, compared with finerenone alone and empagliflozin alone respectively in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D).

Clinical Development 52

Clinical Development Therapies Disease Treatment 52

FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

Disease 59

Disease Production FDA Drug Development 59

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

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. recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Join the club.

Small Molecule 137

Small Molecule Trials Therapies Disease 137

Drug Target Review

JANUARY 15, 2024

Chronic obstructive pulmonary disease Chronic obstructive pulmonary disease (COPD) is the third leading cause of global deaths. The Pathology of Chronic Obstructive Pulmonary Disease. Annual Review of Pathology: Mechanisms of Disease. Pluripotent Stem Cell-Based Cell Therapy – Promise and Challenges.

Immune Response 113

Immune Response Disease Science Therapies 113

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.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

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. Nature 578 , 229-236 (2020). References Doudna JA.

Engineering 105

Engineering Virus Therapies Immune Response 105

The Pharma Data

JULY 28, 2021

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for avalglucosidase alfa, a long-term enzyme replacement therapy for the treatment of people with Pompe disease. Avalglucosidase alfa is also currently undergoing review in other countries including the U.S.,

Disease 52

Disease Therapies FDA Clinical Development 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.

Disease 52

Disease Clinical Trials FDA Approval FDA 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.

Disease 52

Disease FDA Treatment Clinical Trials 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. Elsevier; 2020. 2020 Oct 7;21(10):571–84. 2020 Jun 8;4(9):863–74.

Clinical Research 116

Clinical Research Research Drug Development Molecular Biology 116

Broad Institute

OCTOBER 23, 2024

They knew that discovering the genetic cause of her disorder would help them find other people like her, help get the condition formally recognized as a new disease, and help them better advocate for research into new treatments. This is the first human disease caused by loss of one copy of a lncRNA gene.

DNA 128

DNA RNA Disease Hospitals 128

The Pharma Data

AUGUST 5, 2021

The acquisition strengthens BioNTech’s cell therapy pipeline by accelerating the individualized solid tumor Neoantigen TCR cell therapy research and development program. It also expands the Company’s cell therapy capabilities and manufacturing footprint in North America, building on its acquisition of Neon Therapeutics in 2020.

Therapies 52

Therapies Small Molecule Vaccine Science 52

The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

Drug Development 52

Drug Development Disease Clinical Trials Drugs 52

Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

Small Molecule 105

Small Molecule Therapies Regulations Treatment 105

The Pharma Data

OCTOBER 27, 2021

Small molecule candidate Anle138b targets disease modification for multiple system atrophy and other neurological disorders. Anle138b targets pathological alpha-synuclein oligomers and is being evaluated in patients with neurodegenerative diseases for potential disease modification. Teva Pharmaceutical Industries Ltd.

Licensing 52

Licensing Licensing Disease Small Molecule 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.

RNA 98

RNA Regulations Disease DNA 98

The Pharma Data

SEPTEMBER 11, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. o Completion: 2020. o Number of patients: 3,730. 20,21,22.

Therapies 52

Therapies Hospitals Clinical Trials Trials 52

The Pharma Data

MAY 31, 2022

BIIB122 is an inhibitor of LRRK2, a potential novel target intended to impact the underlying biology and slow the progression of Parkinson’s disease Phase 2b LUMA to enroll approximately 640 participants with early-stage Parkinson’s disease; most advanced clinical study of a LRRK2 inhibitor. Denali Therapeutics Inc.

Disease 52

Disease Small Molecule Engineering Biosimilars 52

DrugBank

AUGUST 9, 2024

Humira , AbbVie's monoclonal antibody for autoimmune diseases, generated over $20 billion in annual revenue at its peak before losing patent exclusivity in 2023.  This exponential growth is primarily fueled by the escalating global burden of chronic diseases. trillion dollars, and it is forecasted to exceed 1.4

Pharmaceuticals 76

Pharmaceuticals Research Pharmaceutical Companies Clinical Trials 76

The Pharma Data

JULY 12, 2021

Finerenone 10 mg or 20 mg is indicated to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, cardiovascular death, non-fatal myocardial infarction, and hospitalization for heart failure in adult patients with chronic kidney disease (CKD) associated with type 2 diabetes (T2D).

FDA Approval 52

FDA Approval Disease FDA Treatment 52

The Pharma Data

APRIL 27, 2022

Based on DESTINY-Breast04 results where AstraZeneca and Daiichi Sankyo’s Enhertu demonstrated a significant improvement in both progression-free survival and overall survival Enhertu has now been granted five Breakthrough Therapy Designations, including three in breast cancer and one in both lung and gastric cancers.

Therapies 52

Therapies Trials Protein Expression Treatment 52

The Pharma Data

SEPTEMBER 27, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for empagliflozin as an investigational treatment for adults with coronary failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced. 2,3 a number one explanation for hospitalization within the U.S.

Therapies 52

Therapies Clinical Trials Hospitals Trials 52

The Pharma Data

MARCH 25, 2021

this week announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Breyanzi (lisocabtagene maraleucel: liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy for the treatment of patients with relapsed or refractory (R/R) large B-cell lymphoma 1 and R/R follicular lymphoma. Bristol Myers Squibb K.K.

Therapies 52

Therapies Trials Treatment Disease 52

The Pharma Data

MAY 27, 2022

“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

Therapies 52

Therapies Treatment Pharmaceutical Companies Nurses 52

New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. 2020-01-02. SCHEME PATENT PTC Therapeutics Inc., Audiol Res. J Pers Med.

Small Molecule 57

Small Molecule RNA DNA Disease 57

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. Valneva and Pfizer entered into a collaboration agreement in April 2020 to co-develop VLA15 2. About VLA15.

Vaccine 52

Vaccine Disease Trials Protein Expression 52

The Pharma Data

FEBRUARY 4, 2021

3, 2021 /PRNewswire/ — AbbVie (NYSE: ABBV) announced financial results for the fourth quarter and full year ended December 31, 2020. AbbVie hosted an Immunology Strategic Update event on December 14, 2020 for members of the investment community. NORTH CHICAGO, Ill., Gonzalez , chairman and chief executive officer, AbbVie.

Production 52

Production Clinical Trials International Small Molecule 52

The Pharma Data

JANUARY 11, 2021

a San Diego-based biotechnology company with an array of technology platforms for antibody discovery and optimization, and novel NK and T cell engager generation, today announced licensing of a panel of its anti-SARS-CoV-2 antibody clones to IGM Biosciences for COVID-19 therapy development.

Licensing 52

Licensing Licensing Therapies Engineering 52

Drug Target Review

JUNE 8, 2023

An important research area at AbbVie involves blood cancers, where our ultimate goal is to transform standards of care across these diseases and advance a dynamic cancer research and treatment pipeline. Chronic lymphocytic leukemia in 2020: a surfeit of riches? 2020 Aug;34(8):1979-1983. Epub 2020 May 11.

Treatment 105

Treatment Organic Chemistry Research Disease 105

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).

Small Molecule 126

Small Molecule Drugs Immune Response Targeted Protein Degradation 126

The Pharma Data

APRIL 22, 2023

Multiple sclerosis (MS) is a devastating autoimmune disease that destroys the protective myelin covering around nerves, disrupting communication between the brain and body, and causing patients’ ability to move and function to progressively decline. million individuals that currently have to live with the disease.

Therapies 40

Therapies Immune Response Small Molecule Engineering 40