Science and Therapies - Drug Discovery Digest

New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If

Therapies

Therapies FDA Approval Engineering Disease

A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.

Disease

Disease Therapies Engineering Clinical Trials

Scaling Phage Therapy

Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

Therapies

Therapies Virus DNA Clinical Trials

OBN Invites Life Science Companies, Industry Leaders and Investors to BioTrinity 2022

Drug Discovery Today

FEBRUARY 4, 2022

• Face-to-face event, taking place in London, UK, 26-27th April, will feature two dedicated tracks: ‘Scientific’, focused on seven core therapy areas and ‘Life Sciences Business’ Programme features keynote from George Freeman MP, with an update on the UK Government Life Sciences VisionRegistration for BioTrinity 2022 now available online

Science

Science Government Therapies

100x improvement in sight seen after gene therapy trial

Science Daily: Pharmacology News

SEPTEMBER 6, 2024

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

Therapies

Therapies Trials Research

Macrophage cell therapy: a new hope for chronic liver disease patients

Drug Target Review

AUGUST 28, 2024

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

Therapies

Therapies Disease Engineering Medical Schools

The science of ageing and restoring healthspan

Drug Target Review

JANUARY 8, 2024

Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.

Science

Science Disease FDA Approval Clinical Trials

50 years after founding, NIDA urges following science to move beyond stigma

National Institute on Drug Abuse: Nora's Blog

FEBRUARY 1, 2024

50 years after founding, NIDA urges following science to move beyond stigma area Thu, 02/01/2024 - 11:20 Nora's Blog February 1, 2024 Image NIDA Image In 2024, NIDA celebrates its 50th anniversary. The knowledge generated by NIDA research has also led to a robust and rigorous science of drug use prevention.

Science

Science FDA Approval Treatment Research

New gene therapy for muscular dystrophy offers hope

Science Daily: Pharmacology News

JULY 17, 2024

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

Therapies

Therapies Treatment Disease Research

CAR-NK cells: promising for cancer therapy

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. NK cells can be readily extracted from umbilical cord blood or peripheral blood of adult donors.

Therapies

Therapies Cell Biology Clinical Trials Trials

Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. She passed away in 2013.

Therapies

Therapies Clinical Trials DNA Trials

Navigating the challenges of cell therapies

Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

Therapies

Therapies Engineering Treatment Molecular Biology

bit.bio joins forces with maths institute to find the ‘operating system of life’

Drug Discovery Today

OCTOBER 22, 2020

Cell coding startup and science institute partner on a moonshot mission to create every human cell type for biomedical research and therapy

Science

Science Therapies Research

Gene therapy gets a turbo boost

Science Daily: Pharmacology News

AUGUST 29, 2024

Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. But a major hurdle has been finding a safe and efficient way to deliver those genes.

Therapies

Therapies Disease Research

Six key considerations for cell and gene therapy patient support programs

Fierce BioTech

JANUARY 19, 2024

While much attention has been given to the novel science behind cell and gene therapies (CGTs), the success of every precision medicine starts and ends with the patient. Cell and gene therapy patients often require support throughout their treatment journey. Learn key considerations for developing patient support programs.

Therapies

Therapies Science Treatment

New gene delivery vehicle shows promise for human brain gene therapy

Science Daily: Pharmacology News

MAY 16, 2024

In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.

Therapies

Therapies Engineering Disease Research

FDA Returns Disappointing News for ALS Stem Cell Therapy

PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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Therapies FDA Clinical Trials Disease

Using stem cell-derived heart muscle cells to advance heart regenerative therapy

Science Daily: Pharmacology News

APRIL 26, 2024

Regenerative heart therapies involve transplanting cardiac muscle cells into damaged areas of the heart to recover lost function. However, the risk of arrhythmias following this procedure is reportedly high.

Therapies

Therapies Research

Gamma delta T cells: a rising star in cancer therapy

Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

Therapies

Therapies Engineering Molecular Biology Science

Stem cell therapy: Alzheimer’s disease symptoms

Drug Target Review

AUGUST 31, 2023

In the pursuit of a remedy for Alzheimer’s disease, a frontier in medical science is illuminating a glimmer of hope. Stem cell therapies have already demonstrated their prowess in treating diverse cancers and ailments linked to the blood and immune system.

Therapies

Therapies Disease Treatment Research

Single-dose gene therapy is potentially life-changing for adults with hemophilia B

Science Daily: Pharmacology News

SEPTEMBER 25, 2024

Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an international Phase III clinical trial.

Therapies

Therapies Clinical Trials International Trials

New Gene therapy trial shows restored hearing and speech in children born deaf, treated in both ears

Science Daily: Pharmacology News

JUNE 5, 2024

A new clinical trial of five children with inherited deafness found administering gene therapy in both ears led to restored hearing and speech, and additional gains including sound source localization, ability to hear in noisy environments, and for two children, abillity to appreciate music.

Therapies

Therapies Trials Clinical Trials International

An Alzheimer’s drugmaker is accused of data manipulation. Should its trials be stopped?

BioPharma Drive: Drug Pricing

OCTOBER 4, 2024

An experimental Alzheimer’s therapy from Cassava Sciences is still being tested in two Phase 3 studies, even as the company has come under regulatory scrutiny.

Trials

Trials Science Therapies

Advancing neurological therapies with Dr Bruce Leuchter

Drug Target Review

FEBRUARY 1, 2024

Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? Investors have historically taken a ‘guilty until proven innocent’ approach to investing in neuroscience therapies. All stakeholders have benefited from these innovations.

Therapies

Therapies Drug Development Disease Clinical Development

Risk of secondary cancers after CAR-T cell therapy low, according to large study

Science Daily: Pharmacology News

JUNE 12, 2024

In April, the FDA warned of risk of secondary cancers in people receiving CAR-T cell therapy. A large study now finds the risk is low and not related to the CAR-T cells.

Therapies

Therapies FDA

Nucleic acid therapy accelerator

Drug Target Review

NOVEMBER 23, 2023

elegans be applied to the development of targeted therapies for neuropsychiatric conditions like eating disorders, OCD, and PTSD? He is also a Professor of Biology at the University of Leuven, a member of EMBO and a Fellow of the Academy of Medical Sciences. How might the understanding of neuropeptide networks in C.

Therapies

Therapies Molecular Biology Laboratories Government

Triple combination therapy brings lasting improvement in cystic fibrosis

Science Daily: Pharmacology News

JULY 7, 2023

The mucus in the airways is not as sticky, inflammation in the lungs significantly reduced: Triple combination therapy can achieve these positive, lasting effects in patients with cystic fibrosis (CF). According to new research, this form of medication improves the symptoms of CF in many patients.

Therapies

Therapies Research

Gene therapy restores hearing in children with hereditary deafness

Science Daily: Pharmacology News

JANUARY 24, 2024

A novel gene therapy for hearing loss was administered to six children in China in a clinical trial. Each child had an inherited deafness caused by mutations in the OTOF gene, called DFNB9.

Therapies

Therapies Clinical Trials Trials Research

The evolution of cell therapy to address unmet medical needs

Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. In some cases, cell therapy can be curative for the patient.

Therapies

Therapies Small Molecule Pharmaceutical Companies Molecular Biology

New target for potential leukemia therapy

Science Daily: Pharmacology News

MAY 8, 2024

Investigators discovered that a subset of myeloid and lymphoid leukemias depend on a molecular complex called PI3Kgamma for survival.

Therapies

Therapies Clinical Trials Trials

All stem cell therapies are not created equal

Science Daily: Pharmacology News

JULY 12, 2024

Researchers have found that two of the most frequently administered stem cell therapies, which are often used interchangeably, actually contain completely different types of cells.

Therapies

Therapies Research

Promising evidence for sickle cell gene therapy

Science Daily: Pharmacology News

AUGUST 30, 2023

A clinical trial has tested a potentially curative stem cell gene therapy for sickle cell disease. The results were promising.

Therapies

Therapies Clinical Trials Trials Disease

Insights into cellular therapies for cancer treatment

Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

Therapies

Therapies Treatment Vaccine FDA Approval

Navigating Market Access Challenges in Life Sciences Commercialization

Drug Channels

MARCH 28, 2024

Bill discusses specialty therapy access barriers, including payer utilization management techniques and “no coverage” policies. Today’s guest post comes from Bill Dupere, SVP of Product Development & Partnerships at Mercalis. Contact Mercalis to learn about their Patient Support Services. Read on for Bill’s insights.

Marketing

Marketing Science Therapies Production

Expanding the impact of CAR T cell therapy: An immunotherapy strategy against all blood cancers

Science Daily: Pharmacology News

AUGUST 31, 2023

A broad new strategy could hold hope for treating virtually all blood cancers with CAR T cell therapy, which is currently approved for five subtypes of blood cancer. Scientists have demonstrated the potential efficacy of this approach a preclinical study.

Therapies

Treating newly-diagnosed Crohn's patients with advanced therapy leads to dramatic improvements in outcomes

Science Daily: Pharmacology News

FEBRUARY 22, 2024

A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.

Therapies

Therapies Clinical Trials Treatment Disease

Thinking inside the box: How SmartLabs is supporting the science of tomorrow

BioPharma Drive: Drug Pricing

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

Science

Science RNA Therapies Disease

Statin therapy may prevent cancer by blocking inflammatory protein

Science Daily: Pharmacology News

MAY 30, 2024

Scientists have found that statins -- commonly used cholesterol-lowering drugs -- may block a pathway that leads to the development of cancer in the context of chronic inflammation.

Therapies

Therapies Drugs

Researchers design gene therapy that can effectively target glioblastoma

Science Daily: Pharmacology News

OCTOBER 18, 2023

Results demonstrated the safety and preliminary efficacy of the novel gene therapy approach in high-grade glioma patients, with prolonged survival in a subgroup of recurrent GBM patients immunologically 'familiar' with the virus.

Therapies

Therapies Immune Response Research Virus

Research points to potential new medical therapy for Lyme disease

SCIENMAG: Medicine & Health

JULY 28, 2023

Lynch, lead author, is now a research fellow in the Department of Veterinary and Animal Sciences. Pearson, co-author of the paper, is now a NEWVEC post-doctoral researcher at UMass Amherst. Credit: UMass Amherst […]

Research

Research Therapies Disease Doctors

Key improvements to Parkinson's disease cell therapies

Science Daily: Pharmacology News

JULY 12, 2023

They also found that co-transplantation of neuronal cell therapy with host regulatory T cells resulted in effective suppression of needle trauma and significant improvement in the survival and recovery of grafts. These findings suggest a path for the 'realistic' use of cell therapy to treat neurodegenerative disorders.

Therapies

Therapies Immune Response Disease Research

Lessons From A Private Funding Round: Science, Relationships, And Experience

LifeSciVC

APRIL 2, 2024

The quality of science – and the ability to distinguish it from other approaches – was the entry ticket to serious engagement. The science presents an opportunity and a challenge for us at Sionna. The science presents an opportunity and a challenge for us at Sionna. That science resonates with investors.

Science

Science Small Molecule International Clinical Development

Discoveries on memory mechanisms could unlock new therapies for Alzheimer's and other brain diseases

Science Daily: Pharmacology News

AUGUST 30, 2023

Scientists have made a `paradigm shifting' discovery on the mechanisms required for learning and memory that could lead to new therapies for Alzheimer's disease and potentially Down syndrome.

Therapies

Therapies Disease

NUS researchers develop novel approach for predicting resistance against cancer therapy

SCIENMAG: Medicine & Health

JULY 18, 2023

A team of researchers from the Cancer Science Institute of Singapore (CSI Singapore) at the National University of Singapore (NUS), led by Assistant Professor Anand Jeyasekharan, has discovered a unique combination of oncogenes that could predict treatment resistance, and hence unfavourable outcomes, of patients with Diffuse large B cell lymphoma (DLBCL), (..)

Research

Research Therapies Science Treatment

New gene delivery vehicle shows promise for human brain gene therapy

A therapy candidate for fatal prion diseases turns off disease-causing gene

Trending Sources

Scaling Phage Therapy

OBN Invites Life Science Companies, Industry Leaders and Investors to BioTrinity 2022

100x improvement in sight seen after gene therapy trial

Macrophage cell therapy: a new hope for chronic liver disease patients

The science of ageing and restoring healthspan

50 years after founding, NIDA urges following science to move beyond stigma

New gene therapy for muscular dystrophy offers hope

CAR-NK cells: promising for cancer therapy

Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

Navigating the challenges of cell therapies

bit.bio joins forces with maths institute to find the ‘operating system of life’

Gene therapy gets a turbo boost

Six key considerations for cell and gene therapy patient support programs

New gene delivery vehicle shows promise for human brain gene therapy

FDA Returns Disappointing News for ALS Stem Cell Therapy

Using stem cell-derived heart muscle cells to advance heart regenerative therapy

Gamma delta T cells: a rising star in cancer therapy

Stem cell therapy: Alzheimer’s disease symptoms

Single-dose gene therapy is potentially life-changing for adults with hemophilia B

New Gene therapy trial shows restored hearing and speech in children born deaf, treated in both ears

An Alzheimer’s drugmaker is accused of data manipulation. Should its trials be stopped?

Advancing neurological therapies with Dr Bruce Leuchter

Risk of secondary cancers after CAR-T cell therapy low, according to large study

Nucleic acid therapy accelerator

Triple combination therapy brings lasting improvement in cystic fibrosis

Gene therapy restores hearing in children with hereditary deafness

The evolution of cell therapy to address unmet medical needs

New target for potential leukemia therapy

All stem cell therapies are not created equal

Promising evidence for sickle cell gene therapy

Insights into cellular therapies for cancer treatment

Navigating Market Access Challenges in Life Sciences Commercialization

Expanding the impact of CAR T cell therapy: An immunotherapy strategy against all blood cancers

Treating newly-diagnosed Crohn's patients with advanced therapy leads to dramatic improvements in outcomes

Thinking inside the box: How SmartLabs is supporting the science of tomorrow

Statin therapy may prevent cancer by blocking inflammatory protein

Researchers design gene therapy that can effectively target glioblastoma

Research points to potential new medical therapy for Lyme disease

Key improvements to Parkinson's disease cell therapies

Lessons From A Private Funding Round: Science, Relationships, And Experience

Discoveries on memory mechanisms could unlock new therapies for Alzheimer's and other brain diseases

NUS researchers develop novel approach for predicting resistance against cancer therapy

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