Drug Target Review

SEPTEMBER 1, 2023

Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.

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Drug Discovery Today

JANUARY 13, 2021

Mission: Cure is a coalition of patients, doctors, researchers and entrepreneurs who are committed to advancing new therapies and cures for currently untreatable diseases. Their primary disease focus is on chronic pancreatitis, a rare inflammatory condition that affects over a million people across the globe.

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FDA Law Blog: Drug Discovery

MARCH 13, 2023

Knowing they could not hold PFDD meetings for every disease ( there are over 10,000 rare diseases alone ), they decided to turn PFDD meetings over to disease advocacy organizations, calling them Externally-Led Patient Focused Drug Development (EL-PFDD) meetings. There have been challenges in gene therapy development.

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Drug Target Review

JANUARY 12, 2024

How does the integration of Real-World Data (RWD) with genomic biomarker data contribute to a more comprehensive understanding of disease progression and treatment response? This one size fits all approach to drug prescribing still pervades in mostly all therapy areas except oncology. Most drugs do not work in all people.

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Broad Institute

OCTOBER 3, 2024

The findings point to factors involved in how neurons communicate and fire, suggesting potential targets for new therapies. In the future, the results could also help doctors tailor treatments to a patient’s genome. Epilepsy is one of the most common neurological disorders.

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biobide

FEBRUARY 2, 2024

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. Children and adolescents lack robust preclinical models to replicate the pathologies and provide precise and targeted therapies.

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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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PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

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Drug Target Review

OCTOBER 9, 2023

The potential of stem cell therapies is rooted in the pluripotency of these early embryonic stem cells. Dr Ceren Pajanoja, a Doctoral Researcher at the University of Helsinki, states that this precise and efficient technique enables the identification of cell profiles directly within the original tissue.

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Drug Target Review

SEPTEMBER 6, 2024

What trends are driving the increased use of in vivo delivery methods in gene therapy? This growth has been influenced by the increased use of lentiviral vectors for more prevalent diseases, earlier treatment lines and broader applications. How are new therapeutic areas emerging as potential targets for in vivo gene therapy?

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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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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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Drug Target Review

DECEMBER 13, 2023

Combinatorial analytics approaches identify combinations of features that together are associated with the disease phenotype in patient sub-groups, capturing the non-linear effects of interactions between multiple genes. The combinatorial approach is considerably more sensitive than GWAS and requires much smaller patient populations.

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Drug Target Review

JANUARY 23, 2024

In April 2023, we saw the first randomised data applying an individualised vaccine in combination with checkpoint inhibition for resectable melanoma, demonstrating benefit in terms of time to disease recurrence. We anticipate this area to grow, especially cancer vaccines, with important read-outs expected across various cancer types in 2024.

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The Pharma Data

AUGUST 22, 2021

In the early days of the COVID-19 pandemic, doctors in Wuhan noticed something surprising. “There’s often a phenomenon that doctors report anecdotally, or that’s mentioned in passing in a particular research paper, and that provides a clue – a hook,” he said. The next challenge was to figure out why.

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The Pharma Data

JANUARY 15, 2021

13 in the New England Journal of Medicine , involved patients with high blood pressure in the lungs caused by interstitial lung disease (ILD). It can have a range of causes, from smoking, to occupational exposure to toxins like asbestos, as well as autoimmune diseases like rheumatoid arthritis. The study, published online Jan.

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Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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Drug Target Review

OCTOBER 1, 2024

Since I was a child, I always wanted to be a doctor. From a human point of view, I have always considered doctors’ work as a sort of mission for the good of others. Being a doctor means giving courage and hope day in, day out to those experiencing illness. Oncology has come a long way in recent years.

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The Pharma Data

NOVEMBER 20, 2020

20, 2020 — Hormone therapy can be a lifesaver for men with prostate cancer , but it also appears to put some at increased risk of heart problems, a new study reports. At the start of the study, 4 out of 5 men had two or more risk factors for heart disease , the study says. FRIDAY, Nov.

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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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Alta Sciences

JUNE 12, 2024

I’ve always loved biological sciences, but I didn’t want to become a doctor and didn’t know what other careers in the field existed. Every study is different, so I am constantly learning about different diseases, therapeutic areas , and experimental methods while developing study protocols. What is one of your proudest achievements?

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The Pharma Data

DECEMBER 10, 2020

Dr. Pirozzi received a Doctorate of Medicine from Università Campus Bio-Medico di Roma in Italy, a Doctorate of Philosophy in Immunology from Sapienza Università di Roma in Italy, a Post-Doctorate degree in Immunology from the Institut Pasteur in Paris, France, and business training at the London Business School, UK.

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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.

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Drug Target Review

DECEMBER 12, 2023

Our linker technology platform has the potential to develop ADCs that target a wide range of different types of diseases, beyond cancer. He positioned Araris to shift the paradigm in how to think about developing targeted cancer therapies and since October 2023, he transitioned to become its CSO.

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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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PLOS: DNA Science

OCTOBER 17, 2024

FDA approved a treatment for type 3 of the ultra-rare genetic disease September 20. The quest has been ongoing for three decades, said Laurie Turner, Family Services Manager of the National Niemann-Pick Disease Foundation. The original approved was in 2003 to treat a type of Gaucher disease. Average length of life is 13 years.

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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.

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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.

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The Pharma Data

NOVEMBER 17, 2020

The researchers identified associations between early antibiotic exposure and an increased risk for childhood-onset asthma, allergic rhinitis, atopic dermatitis, celiac disease, overweight, obesity, and attention-deficit/hyperactivity disorder (hazard ratios ranging from 1.20 Abstract/Full Text. © 2020 HealthDay. All rights reserved.

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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. Mice are often expensive and time-consuming to maintain and breed. Cell Stem Cell [Internet].

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The Pharma Data

JULY 22, 2021

NOTE: ADUHELM TM (aducanumab-avwa)injection 100 mg/mL solutionis indicated for the treatment of Alzheimer’s disease. Treatment with ADUHELM should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials.

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The Pharma Data

SEPTEMBER 23, 2020

Even with the family hardships, seeing her sister battle her illness gave Lisa the strength to face this new challenge head-on and take a leading role in her disease. I challenged my doctor quite often, always asking questions, ‘What’s next? I had to rely on the doctor for all my answers.”. What can you do for me?

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PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. The care partners are the decision-makers for their loved ones with neurodegenerative disorders, handling everything from daily routines and medication management to doctor visits.

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Drug Target Review

JUNE 29, 2023

I think the cell-therapy field has evolved a lot – and is continuing to do so – in the time I’ve been working with this. Regarding the NY-ESO programme that we worked on, I started work on that in 2002 before we even knew that we were going to do cell therapy. I mean, I think cell-therapy is an great place to be.

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Codon

JULY 14, 2024

While this departs from the reductionist, single-component therapies that have dominated transfusion medicine since World War II, emerging data underscores that whole blood transfusions—blood with all its parts—yield better outcomes following severe blood loss than transfusions involving discrete blood components. Always free.

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The Pharma Data

AUGUST 31, 2020

Understanding CAR-T cell therapy in advanced B-cell blood cancers. I pressed my doctor to give me some other choice. He told me about CAR-T cell therapy. Steve , who received CAR-T cell therapy for his advanced B-cell blood cancer. Steve , who received CAR-T cell therapy for his advanced B-cell blood cancer.

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The Pharma Data

JANUARY 21, 2021

That included 71% of patients with relapsing-remitting MS, the most common form of the disease. Most people, though, eventually transition to a progressive form of the disease, and their disability worsens over time. About 85% of people with MS initially have the relapsing-remitting form, according to the MS society.

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