The Pharma Data

NOVEMBER 29, 2020

Prior to joining Gannex, Dr. Palmer was Head of Liver Disease Clinical Development at Takeda Pharmaceuticals. She has also held senior leadership positions at Shire Pharmaceuticals and Kadmon Corporation, where she was in charge of the global development of NASH and other liver disease programs. Source link.

Clinical Development 40

Clinical Development Disease Clinical Trials Therapies 40

Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

Disease 59

Disease Clinical Trials Clinical Pharmacology Trials 59

The Pharma Data

MAY 8, 2021

Sandoz, a Novartis division, today announced progress in the late-stage clinical development program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. About aflibercept. About Sandoz.

Biosimilars 52

Biosimilars Clinical Development Production Government 52

The Pharma Data

DECEMBER 21, 2020

UBX1325 is developed from BM-962, a Bcl-xL inhibiting compound licensed to UNITY by Ascentage Pharma for the treatment of age-related diseases. This progress in clinical development qualifies Ascentage Pharma for a milestone payment according to the terms of the licensing agreement. SUZHOU, China and ROCKVILLE, Md. ,

Clinical Development 40

Clinical Development Disease Treatment Licensing 40

The Pharma Data

NOVEMBER 30, 2020

Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today provided an update on its COVID-19 vaccine program. About NVX-CoV2373 NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease.

Clinical Development 52

Clinical Development Vaccine Clinical Trials Immune Response 52

The Pharma Data

JULY 8, 2021

ADUHELM should be initiated in patients with mild cognitive impairment due to Alzheimer’s disease or mild Alzheimer’s dementia. The update includes an addition to the Indications and Usage section of the label (Section 1) to emphasize the disease stages studied in the clinical trials, as seen below ( italics to note updated language).

Clinical Trials 52

Clinical Trials FDA Approval Trials FDA 52

Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

Disease 52

Disease Clinical Trials Trials Pharmaceutical Companies 52

Drug Target Review

JULY 31, 2023

Disease-Specific Applications Each chapter of the report focuses on specific therapeutic areas, such as cancer, Alzheimer’s disease, HIV infection, and immunotherapy. This targeted approach provides readers interested diseases or applications with tailored information and insights that align with their specific interests.

Therapies 98

Therapies Disease Clinical Development Hospitals 98

The Premier Consulting Blog

JUNE 18, 2023

This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and to integrate information in useful ways. One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases.

Drug Development 52

Drug Development Disease Clinical Trials Drugs 52

Drug Target Review

SEPTEMBER 5, 2024

I was part of building up this small biopharma company and learn a lot about rare diseases, regulatory interactions and challenges related to that, challenges related to out licensing drug candidates and having new people joining the company every month as we were growing so fast.

Pharma Companies 111

Pharma Companies Licensing Licensing Disease 111

The Pharma Data

DECEMBER 10, 2020

Spending several years with Sanofi in Paris, France as a Global safety officer, he then relocated to New Jersey, USA to work in Early and Late Stage Clinical Development in Immunology, when he became the Global Project Head of Dupixent, a medicine that has revolutionized the field of allergic and atopic diseases.

Disease 52

Disease Research Doctors Clinical Development 52

The Pharma Data

JANUARY 26, 2021

Novel chronic kidney disease (CKD) target from the partnership enters AstraZeneca’s portfolio. CKD is a complex, and debilitating disease which affects around 10% of the world’s population. LONDON , Jan. LONDON , Jan.

Disease 52

Disease Clinical Development Treatment Drug Development 52

Conversations in Drug Development Trends

AUGUST 23, 2023

Back in 2019, he was working in full-service ophthalmology when he learned he was going blind due to a rare and orphan disease called Wolfram syndrome. Earlier this year, McNary participated in a panel discussion at a rare disease conference that focused on the importance of including patients during FDA engagement.

FDA 96

FDA Trials Clinical Trials Disease 96

Drug Target Review

SEPTEMBER 19, 2024

Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). The third project in clinical phase, IRL757, is in Phase I and is being developed for the treatment of apathy.

Disease 52

Disease Drugs Treatment Drug Development 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

The Pharma Data

MAY 31, 2022

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of of BRT-DA01 in patients with Parkinson’s disease.?. The purpose of the?Ph1

Trials 52

Trials Disease Clinical Trials Engineering 52

The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. However, acceptance of that data depends on a sponsor’s understanding of the FDA criteria for historical control groups, study data standards, and clinical meaningfulness.

Disease 52

Disease FDA FDA Approval Treatment 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

The Pharma Data

MAY 2, 2021

Holzkirchen, May 3, 2021 – Sandoz, a Novartis division, today announced progress in the late-stage clinical development program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. About aflibercept.

Biosimilars 40

Biosimilars Clinical Development Production Government 40

The Pharma Data

JANUARY 25, 2021

“The establishment of an Advisory Board focusing on Alzheimer’s disease, and the recruitment of such an impressive group of scientific and clinical leaders in this field is an extremely important step forward for the Company,” stated Paul Brennan, NervGen’s President & CEO. Dr.

Disease 40

Disease Clinical Development Molecular Biology Treatment 40

The Pharma Data

JULY 23, 2021

Nasdaq: BIIB) today announced it will share multiple oral and poster presentations from its Alzheimer’s disease clinical development portfolio at the Alzheimer’s Association International Conference (AAIC), which will be held in Denver, Colorado and online July 26-30, 2021. Biogen Inc.

International 52

International Disease Clinical Trials Trials 52

The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

Disease 52

Disease Trials Treatment Clinical Trials 52

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.

Drug Development 52

Drug Development Disease Therapies Engineering 52

The Pharma Data

NOVEMBER 9, 2021

– More than 30 Abstracts Across HDV, HCV, HBV, NASH and PSC Reflect Gilead’s Ongoing Commitment to Addressing Challenges Facing People Living With Liver Diseases –. Important Safety Information. About Gilead Sciences in Liver Disease. Gilead Lores,Inc. See below forU.S. Long- term Treatment With Vemlidy in HBV.

Disease 52

Disease Clinical Trials Treatment Pharmacokinetics 52

The Pharma Data

JULY 27, 2021

AbbVie (NYSE: ABBV) and Calico Life Sciences today announced an extension of their leading-edge collaboration to discover, develop and bring to market new therapies for patients with age-related diseases, including neurodegeneration and cancer. For more information about AbbVie, please visit us at www.abbvie.com. Levinson, Ph.D.,

Disease 52

Disease Science Therapies Production 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. VLA15 has demonstrated strong immunogenicity and safety data in pre-clinical and clinical studies so far.

Vaccine 52

Vaccine Disease Trials Protein Expression 52

The Open Targets Blog

JUNE 26, 2023

This new target-disease association evidence is presented in a “CRISPR Screens” widget, contributing to our Pathways and Systems Biology data type. Thanks to the expertise of our colleagues at the Wellcome Sanger Institute, 23 screens included in CRISPRbrain were mapped to disease or diseases in the Open Targets Platform.

Disease 59

Disease Clinical Development Clinical Trials Drugs 59

The Pharma Data

DECEMBER 29, 2020

(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A.,

Clinical Trials 52

Clinical Trials Disease Treatment Trials 52

The Pharma Data

OCTOBER 27, 2020

Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). About Krabbe Disease. Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021.

Disease 40

Disease FDA Treatment Clinical Trials 40

Drug Target Review

JULY 20, 2023

Additionally, they compared their long COVID analysis with known genetic associations in various chronic diseases, particularly neurological and metabolic disorders. The combinatorial analysis revealed that 73 genes were significantly associated with long COVID development.

Clinical Development 75

Clinical Development Regulations Disease Treatment 75

The Pharma Data

JANUARY 10, 2021

The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Enrolled patients will receive a single administration of EDIT-301.

Clinical Trials 52

Clinical Trials Disease FDA Trials 52

LifeSciVC

APRIL 24, 2024

Target validation : Do we believe the target(s) plays a central role in disease biology and that modulation will modify disease? I will note that “de-risked” is never fully risk-free in therapeutics investing, as even precedented mechanisms hit stumbling blocks in preclinical or clinical development.

Production 111

Production Small Molecule Regulations Trials 111

The Pharma Data

DECEMBER 13, 2020

2] Pre-clinical studies have shown that these differentiated properties of HMBD-001 lead to robust and sustained tumor growth inhibition in multiple HER3 cancer models, including those with NRG1-fusions. Our highly experienced teams in Singapore and the US span antibody discovery, pharmacology, production and clinical development.

Clinical Development 40

Clinical Development Clinical Trials Therapies Trials 40

The Pharma Data

OCTOBER 1, 2021

Deucravacitinib demonstrated efficacy no matter baseline characteristics, including weight , disease severity and former treatment with biologic or non-biologic therapies. Deucravacitinib is being studied in multiple immune-mediated diseases, including psoriasis, rheumatoid arthritis , lupus and inflammatory bowel disease.

Disease 52

Disease Laboratories Clinical Pharmacology Treatment 52

Drug Target Review

APRIL 3, 2024

Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. The transition from a scientist to a manager often requires to make impactful decisions with limited information.

Science 105

Science Clinical Development Treatment DNA 105

Drug Target Review

JANUARY 15, 2024

CD8+ T cells are a type of lymphocyte that specialises in scanning information presented by HLA Class I. As T cells develop, chromosomal DNA encoding the TCR of the CD8 T cells of each individual is rearranged and ‘educated’ to respond only to peptides presented in the context of a specific HLA Class I molecule present in that individual.

Engineering 110

Engineering DNA Virus Immune Response 110

The Pharma Data

JANUARY 18, 2021

Potential first-in-class disease-modifying biologic in late-stage clinical studies. Experienced executive development team from Gilead Sciences. Therefore, atacicept may be uniquely positioned to be disease-modifying for these patients with no current treatment options.”

Disease 40

Disease Science Clinical Development Treatment 40