KIF1A

APRIL 6, 2024

Read the Article Rare Roundup Columbia Receives $15M for Ultra-Rare ALS Medicines Creation Gene-based therapies like Antisense Oligonucleotides have taken major strides in recent years, including Susannah Rosen’s current n-of-1 ASO trial. She graduated from UC San Diego with a B.S. degree in human biology in 2023.

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DNA Medical Schools Therapies Research 111

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. The study appears in Nature Biotechnology.

RNA 98

RNA Medical Schools Engineering Disease 98

Broad Institute

DECEMBER 6, 2023

In the future, when doctors give patients cytokine therapies targeting the immune system, they could potentially look in the Immune Dictionary for expected cell-level immune responses. There are many open questions, and having this foundational reference can significantly improve our understanding of the immune system.

Immune Response 138

Immune Response RNA Disease Medical Schools 138

Broad Institute

MARCH 8, 2024

It is currently not fully understood why a given person develops the disease or why there is a lot of variation around clinical features across people with the disease,” said senior author Miriam Udler, director of the MGH Diabetes Genetics Clinic, an assistant professor of medicine at Harvard Medical School, and an associate member at Broad.

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Medical Schools Hospitals Disease Therapies 127

Broad Institute

JUNE 1, 2023

The work was co-led by senior author Pradeep Natarajan , an associate member at the Broad, the director of preventive cardiology at Massachusetts General Hospital, and an assistant professor of medicine at Harvard Medical School. Maternal meta-analysis Scientists have long thought that HDPs are caused in part by genetics.

Doctors 133

Doctors Medical Schools Hospitals DNA 133

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. Both of the new gene therapy studies were published online Dec. SATURDAY, Dec. 5 in the New England Journal of Medicine.

Therapies 52

Therapies Disease Medical Schools Virus 52

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 29, 2024

Through further research and work as a principal investigator on clinical studies for several central nervous system indications, I laid the groundwork for a career in gene therapy drug development. AskBio) I continue to work to bring innovative gene therapies to patients in need. In my current role at Asklepios BioPharmaceutical, Inc.

Therapies 111

Therapies Disease Clinical Trials Science 111

Broad Institute

APRIL 2, 2024

He is also a professor at Harvard Medical School and Massachusetts General Hospital. But scientists haven’t been able to target those connections with therapies in part because they lack a complete understanding of metabolic pathways in the gut.

Disease 145

Disease Medical Schools Hospitals DNA 145

Broad Institute

NOVEMBER 13, 2023

Doing so would guide treatment decisions, such as whether to continue therapy. "We Such a test could help physicians detect recurrences earlier, intervene sooner, and tailor therapy to each patient. Earlier this year, the Broad entered into an agreement with Exact Sciences to further develop the MAESTRO MRD test for clinical use.

DNA 116

DNA Medical Schools Treatment Therapies 116

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.

Hospitals 121

Hospitals Treatment Medical Schools Science 121

Broad Institute

FEBRUARY 7, 2024

It examined endothelial mechanisms unrelated to lipid metabolism (a known driver of CAD risk with effective therapies, like statins) in hopes of uncovering other mechanisms driving CAD risk for which therapies may yet be developed. “Now

Disease 105

Disease Hospitals Medical Schools Therapies 105

biobide

FEBRUARY 2, 2024

Children and adolescents lack robust preclinical models to replicate the pathologies and provide precise and targeted therapies. This is why not many Pharma companies specifically specialize in discovering and developing pediatric cancer treatments; a great example of advancing new therapies for childhood cancer is the biotech Oncoheroes.

Disease 59

Disease Medical Schools Pharma Companies Clinical Trials 59

Broad Institute

JANUARY 25, 2024

“We’ve come to realize recently that there is a lot more information that we can get from our retina images than we thought was possible,” says senior author Nazlee Zebardast, director of glaucoma imaging at Mass Eye and Ear and an assistant professor of ophthalmology at Harvard Medical School.

Disease 124

Disease Research Science Medical Schools 124

Drug Target Review

JUNE 6, 2023

We are in an era of immuno-oncology (IO) revolution with many approved therapies now available to treat a broad range of cancers. For the last several years, the field has worked to unlock the potential of IO therapies for additional tumour types, and has explored options beyond checkpoint inhibitors.

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Engineering Immune Response Biochemical Pharmacology Clinical Trials 98

The Pharma Data

APRIL 22, 2023

While no cure is available for MS, existing disease-modifying therapies in the form of small molecule and protein drugs either directly target the self-reactive immune cells or broadly dampen inflammation. “Current MS therapies do not specifically target myeloid cells. who is a Core Faculty member at the Wyss Institute.

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Therapies Immune Response Small Molecule Engineering 40

Drug Target Review

JANUARY 15, 2024

About the author Daniel Kavanagh, PhD, RAC Senior Scientific Advisor, Gene Therapies, Vaccines and Biologics at WCG Dr Kavanagh engages with sponsors, CROs and research institutions on topics related to human gene transfer research. Engineering soluble T-cell receptors for therapy. Cancer Therapy With TCR‑Engineered T Cells.

Engineering 110

Engineering DNA Virus Immune Response 110

Broad Institute

JUNE 21, 2023

said Vamsi Mootha , senior author of the study, an institute member at the Broad, professor of systems biology and medicine at Harvard Medical School, and professor of molecular biology at Massachusetts General Hospital. Even with profound ataxia, these mice can still, under the correct therapeutic regimen, reverse course.”

Treatment 98

Treatment Disease Molecular Biology Medical Schools 98

Broad Institute

OCTOBER 4, 2023

The molecule’s dual mechanism of action — targeting both tumor and immune cells — is unique compared to other cancer immunotherapies including PD-1 drugs, and the researchers think it could explain why the molecule was so effective on its own in animal models and may not even need to be used in combination with other drugs such as anti-PD-1 therapy.

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Small Molecule Clinical Trials Trials Engineering 128

The Pharma Data

JANUARY 25, 2021

from Harvard Medical School in Boston, and colleagues developed evidence-based guidelines for the management of VWD and agreed on 12 recommendations. Connell, M.D.,

Disease 52

Disease Medical Schools Laboratories Therapies 52

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

Drug Target Review

MARCH 15, 2024

He has a post-doctorate in virology, viral pathogenesis, viral vector development, and vaccines evaluation from Harvard Medical School, and a PhD in virology/molecular biology. Cell Gene Therapy Insights 2017; 3(2), 131-158, 10.18609/cgti.2017.014. References Mistry RK, Kelsall E, Sou SN, et al. Biotechnol Prog. Epub 2017 Sep 7.

Engineering 105

Engineering Molecular Biology Protein Expression Medical Schools 105

Broad Institute

JANUARY 30, 2024

He saw how genomic technology had progressed during the seven years he’d spent in medical school, spawning discoveries like new mutations underlying leukemia, and he wanted to bring the same capabilities to the study of infectious disease.

Hospitals 137

Hospitals Virus Medical Schools Science 137

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. The five existing FDA-approved therapies for the disease are unable to halt or reverse this loss of function, which affects more than 700,000 people around the world. “The

Medical Schools 52

Medical Schools Clinical Trials FDA Approval Engineering 52

The Pharma Data

JUNE 25, 2021

Choueiri, director of the Lank Center for Genitourinary Oncology, Dana-Farber Cancer Institute and professor of medicine at Harvard Medical School. “It KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells.

Disease 52

Disease Therapies Treatment Medical Schools 52

Broad Institute

OCTOBER 23, 2024

The work highlights the importance of examining the often-ignored noncoding parts of the genome when diagnosing rare genetic disorders, and also points to possible future genetic therapies that fine-tune CHASERR abundance as a means to treat CHASERR - and CHD2 -related disorders.

DNA 128

DNA RNA Disease Hospitals 128

The Pharma Data

NOVEMBER 10, 2020

from Harvard Medical School in Boston, and colleagues developed machine learning models to predict antibiotic susceptibility using electronic health record data and built a decision algorithm for recommending the narrowest possible antibiotic to which a specimen is susceptible for the treatment of uncomplicated UTI. TUESDAY, Nov.

Medical Schools 52

Medical Schools Science Therapies Trials 52

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

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.

Medical Schools 52

Medical Schools Treatment Disease Therapies 52

The Pharma Data

JANUARY 17, 2021

Ophthalmologists are physicians who have completed four years of medical school and four years of residency training. Treatments they help administer include patching therapy, prisms and convergence exercises. Danielle Natale, an optometrist at the Krieger Eye Institute in Baltimore, said in an institute news release.

Medical Schools 52

Medical Schools Doctors Treatment Disease 52

Broad Institute

JUNE 30, 2023

Typically, studying drug therapies is an expensive and time-consuming undertaking. The researchers also looked at the tool’s ability to detect changes in cellular phenotypes after exposure to drugs and small molecule compounds.

Small Molecule 105

Small Molecule Cell Based Assays Disease Assay Development 105

Drug Target Review

AUGUST 16, 2024

After medical school and residency I decided to dedicate my life to women and specialised in obstetrics and gynaecology. I graduated with my BS in physiology and later attended the University of Michigan Medical School. Biotechnology revolutionises healthcare : Biotechnology is transforming medicine and healthcare.

Medical Schools 111

Medical Schools Nurses Research Production 111

Drug Target Review

MAY 16, 2024

1,2 The application of endogenous compounds is a highly pragmatic approach for the creation of disease-modifying therapies. Currently, gene therapy is being developed to correct inborn errors of metabolism, primary immune-deficiencies, and other types of diseases where the genetic lesion is defined. Front Endocrinol (Lausanne).

Disease 64

Disease Therapies Medical Schools Treatment 64

The Pharma Data

NOVEMBER 18, 2021

Choueiri, director, Lank Center for Genitourinary Oncology, Dana-Farber Cancer Institute, and professor of drug, Harvard Medical School. In KEYNOTE-564, pembrolizumab reduced the threat of complaint rush or death by 32, furnishing a promising new treatment option for certain cases at intermediate-high or high threat of rush,” saidDr.

FDA Approval 52

FDA Approval FDA Therapies Treatment 52

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. Study after study had suggested that A.

Research 52

Research Immune Response Medical Schools Disease 52

Broad Institute

MAY 2, 2024

In undergrad, I thought about going to medical school, but I didn’t want to work in a hospital. When cancer metastasizes throughout the body, different areas of the body can develop different mechanisms of resistance. If we can understand how those processes work, we can better develop drugs to target these multifaceted diseases.

Science 124

Science Medical Schools Disease Hospitals 124

Drug Target Review

JULY 1, 2024

Could you elaborate on the potential market size for SRP-001 and how it fits into the broader landscape of pain therapies? With a strong market entry strategy and continuous evidence generation, SRP-001 has the potential to transform pain therapy and meet the enormous unmet needs in this field. South Rampart Pharma, Inc.

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Treatment Clinical Trials Therapies Molecular Biology 59