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

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America. SATURDAY, Dec.

Therapies 52

Therapies Disease Medical Schools Virus 52

Broad Institute

NOVEMBER 6, 2024

We are incredibly grateful to each and every patient who partnered with us, all those involved in their clinical care, and the exceptional team in our lab who made this research possible.” “This work provides a biological foundation for testing more targeted therapies for myocarditis due to an immune checkpoint inhibitor.

Immune Response 118

Immune Response Medical Schools Clinical Trials Treatment 118

Drug Target Review

APRIL 26, 2023

Scientists from the Massachusetts Institute of Technology (MIT) and the University of Massachusetts Medical School (UMass), US, have collaborated to create a novel type of nanoparticle that can deliver messenger RNA that encodes for beneficial proteins to the lungs.

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RNA Medical Schools Engineering DNA 98

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.

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Therapies Immune Response Small Molecule Engineering 40

Drug Target Review

FEBRUARY 29, 2024

During my academic career, I had the opportunity to examine the underlying causes of neurodegenerative diseases like Parkinson’s disease and develop novel animal models to investigate potential disease-modifying therapeutics. AskBio) I continue to work to bring innovative gene therapies to patients in need.

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Therapies Disease Clinical Trials Science 111

KIF1A

APRIL 6, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. As our genetic analyses advance, studies like this highlight the value in reassessing established disease populations. She graduated from UC San Diego with a B.S.

DNA 111

DNA Medical Schools Therapies Research 111

Broad Institute

NOVEMBER 21, 2024

Bernstein is also chair of the Department of Cancer Biology at Dana-Farber Cancer Institute, a professor in cell biology and pathology at Harvard Medical School, and holds the Richard and Nancy Lubin Family Chair. Improving therapy Moreover, the researchers discovered a new effect of the excessive methylation.

Research 64

Research Medical Schools DNA Hospitals 64

Broad Institute

MARCH 8, 2024

“Type 2 diabetes is a disease of elevated blood sugar impacting approximately one in 10 people in the United States that can cause devastating health complications and is usually not cured. Udler and her colleagues assessed genetic findings from more than 1.4 Adapted from an MGH press release. Paper cited: Smith K, Deutsch AJ, McGrail C.,

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Medical Schools Hospitals Disease Therapies 127

Broad Institute

NOVEMBER 6, 2024

Earlier this year, the researchers shared their first translational results, describing the immune cell response underlying colon inflammation in patients undergoing ICI therapy. These are home-run, breakthrough therapies, and quite an amazing success story. How did you go about launching this effort?

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Research Immune Response Hospitals Therapies 124

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.

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DNA RNA Disease Hospitals 128

The Pharma Data

JUNE 2, 2022

Novartis today announced a partnership with the American Society of Hematology (ASH) to provide six additional African nations with technology that is already being used in Ghana to document and share the diagnosis of babies with sickle cell disease.

Disease 52

Disease Medical Schools Treatment Therapies 52

Drug Target Review

MAY 16, 2024

The body’s ability to maintain homeostasis in chronic inflammatory and autoimmune diseases is a defining feature of good health, and a goal is largely neglected by modern medicine. This suggests that the field of drug discovery is primed for a paradigm shift in the fundamental strategy of how diseases are treated.

Disease 64

Disease Therapies Medical Schools Treatment 64

Broad Institute

AUGUST 19, 2024

This study is an important example of how understanding core metabolic requirements and functions of key bacteria can lead directly to new therapies that allow us to modify the microbiome for improved health,” said co-senior author Seth Bloom, an infectious diseases instructor at the Massachusetts General Hospital.

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Hospitals Treatment Medical Schools Science 121

Nvidia Developer: Drug Discovery

JULY 14, 2022

Drug discovery startup Insilico Medicine—alongside researchers from Harvard Medical School, Johns Hopkins School of Medicine, the Mayo Clinic, and. ALS is a debilitating disease. Today, patients typically face an average life expectancy of between two and five years after symptom onset. This is only the beginning.”

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Medical Schools Clinical Trials Engineering FDA Approval 52

Broad Institute

JUNE 21, 2023

With further development, the findings could inform a potential treatment that lowers oxygen levels in certain tissues in humans to reverse advanced disease. If we accept that disease stems from the interaction of genes and environment, then why not manipulate the environment?” The study appeared recently in Human Molecular Genetics.

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Treatment Disease Molecular Biology Medical Schools 98

The Pharma Data

JUNE 2, 2023

Zouboulis, President of the European Hidradenitis Suppurativa Foundation, Director of the Departments of Dermatology, Venereology, Allergology and Immunology, Städtisches Klinikum Dessau, and Founding Professor of Dermatology and Venereology at the Brandenburg Medical School, Germany.

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Medical Schools Treatment Disease Therapies 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.

Disease 52

Disease Clinical Trials Small Molecule Trials 52

Broad Institute

NOVEMBER 13, 2023

The initial focus of the Gerstner Center is to develop an ultrasensitive liquid biopsy test to detect minimal residual disease (MRD) — the presence of tumor cells in the body after treatment. Doing so would guide treatment decisions, such as whether to continue therapy. "We

DNA 116

DNA Medical Schools Treatment Therapies 116

Drug Target Review

DECEMBER 28, 2023

In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. However, if the therapy targets only the subclonal (branch) mutations, this will result in the mere ‘pruning’ of specific branches rather than elimination of the whole cancer.

Bioinformatics 105

Bioinformatics Immune Response DNA Vaccine 105

Drug Target Review

FEBRUARY 26, 2024

Regarding therapy resistant melanomas, why is the alteration of the epigenetic landscape considered significant, and what has been lacking in terms of validated targetable epigenetic mechanisms? What is remarkable here is that we show that the addition of corin to BRAFi-resistant tumour cells resensitises them to BRAFi therapies.

Therapies 59

Therapies Molecular Biology Regulations Medical Schools 59

Drug Target Review

JULY 1, 2024

As a non-NSAID, it reduces adverse effects associated with these pain relief drugs, including kidney injury, GI gastritis/ulceration and blood pressure elevation, which can affect patients with cardiovascular disease and the elderly. was founded in 2016 to develop safer, non-opioid therapies for acute and chronic pain.

Treatment 59

Treatment Clinical Trials Therapies Molecular Biology 59

Broad Institute

JUNE 30, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

Small Molecule 105

Small Molecule Cell Based Assays Disease Assay Development 105

Broad Institute

JULY 26, 2022

By Stephanie Dutchen, Harvard Medical School Office of Communications and External Relations July 26, 2022 Credit: Susanna Hamilton, Broad Communications From immunity to metabolism to mental health, it seems like the gut microbiome has been linked to every aspect of human health and disease. muciniphila ’s cell membrane.

Research 52

Research Immune Response Medical Schools Disease 52

Drug Target Review

JANUARY 15, 2024

This produces a great advantage in infectious disease, as it becomes difficult for a virus to adapt and spread from person to person, because each person’s HLA array is genetically distinct. Engineering soluble T-cell receptors for therapy. Cancer Therapy With TCR‑Engineered T Cells. The FEBS Journal 288:6159–6173 (2021).

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Engineering DNA Virus Immune Response 110

The Pharma Data

SEPTEMBER 18, 2020

Promising Phase 2 data has been released at the European Society for Medical Oncology (ESMO) Virtual Congress 2020 for Sanofi and Regeneron’s jointly-developed PD-1 inhibitor Libtayo (cemiplimab) in the treatment of locally advanced basal cell carcinoma (BCC) in patients for whom hedgehog inhibitor (HHI) therapy is not applicable.

Medical Schools 52

Medical Schools Trials Therapies Hospitals 52

The Pharma Data

JUNE 7, 2023

These longer-term results show the durable overall survival benefit of sacituzumab govitecan over traditional chemotherapy in pre-treated HR+/HER2- metastatic breast cancer,” said Sara Tolaney, MD, MPH, Chief of the Division of Breast Oncology at the Dana-Farber Cancer Institute, and Associate Professor of Medicine, Harvard Medical School. “At

Medical Schools 40

Medical Schools Therapies Treatment Clinical Development 40

The Pharma Data

JANUARY 17, 2021

Optometrists provide comprehensive eye care, including evaluations for glasses and contact lenses and common eye diseases. Ophthalmologists are physicians who have completed four years of medical school and four years of residency training. They can also treat acute eye problems such as pink eye or sties.

Medical Schools 52

Medical Schools Doctors Treatment Disease 52

Broad Institute

JANUARY 30, 2024

Bhattacharyya wears many coats today, splitting his time between the Broad and the infectious disease ward at MGH, where a diagnostic method he developed at Broad is now being tested to see if it helps patients quickly receive the best antibiotics for their infections.

Hospitals 137

Hospitals Medical Schools Virus Science 137

The Pharma Data

MAY 15, 2023

For people living with HDV with advanced disease progression, including cirrhosis, mortality rates can be as high as 50% within five years, presenting significant disease burden and high healthcare-related costs. bulevirtide was granted Breakthrough Therapy and Orphan Drug designations by the Food and Drug Administration (FDA).

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Virus Marketing Treatment Science 40

The Pharma Data

MARCH 13, 2021

Further, prespecified exploratory analyses showed donanemab slowed the accumulation of tau across key brain regions in patients affected by Alzheimer’s disease. “This is the first late-stage study in Alzheimer’s disease to meet its primary endpoint at the primary analysis. Apostolova, M.D.,

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Disease Trials Treatment Medical Schools 40

On Medicine

APRIL 6, 2023

I have a background in Biomedical Engineering prior to medical school and a particular interest in global health and healthcare in resource-limited settings. The chronic cardiovascular diseases are frequently insidious but pervasive and progressive, and early subclinical changes are difficult to identify in children.

Medical Schools 52

Medical Schools Hospitals Treatment International 52

Broad Institute

JUNE 21, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

Small Molecule 52

Small Molecule Cell Based Assays Medical Schools Disease 52

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., Prior to joining EdiGene, he was Vice President of KLUS Pharma and focused on cell therapy and new technologies. as Head of Business Development.

Medical Schools 40

Medical Schools Clinical Development Therapies RNA 40

KIF1A

NOVEMBER 1, 2024

Additional KAND patients are also progressing through n-Lorem’s program for their own tailored ASO therapies, with plans for further expansion. We know that living with a rare disease can be an overwhelming and isolating journey. But there is a powerful way you can help change this— by becoming a part of our natural history study. *Add

Treatment 52

Treatment Medical Schools Research Disease 52

The Pharma Data

JANUARY 26, 2021

Dr. Short brings to VarmX extensive clinical development expertise, with a particular focus on rare diseases and gene therapy for haematological disorders. Further, he advanced gene therapy clinical programmes in haemophilia B and Fabry’s disease. Dr. Short received his Bachelor of Medicine (MBBS) degree from St.

Clinical Development 40

Clinical Development Medical Schools Therapies Pharmaceuticals 40

Broad Institute

MAY 2, 2024

If we can understand how those processes work, we can better develop drugs to target these multifaceted diseases. With rapid autopsies, you can look at a cancer’s life and see how it developed from a primary to metastatic disease. In undergrad, I thought about going to medical school, but I didn’t want to work in a hospital.

Science 124

Science Medical Schools Disease Hospitals 124