This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Strictly Necessary: Used for the proper function of the website
Performance/Analytics: Used for monitoring website traffic and interactions
A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.
Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.
In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. in 2013 — the field has taken off since the success of Novartis’ prostate cancer treatment Pluvicto.
The deal gives Lilly a potential new treatment for heart disease, The Wall Street Journal reported. WEDNESDAY, June 18, 2025 — Drugmaker Eli Lilly plans to buy Verve Therapeutics, a gene-editing startup, for about $1 billion upfront.
“This experience underscored the gaps in rare disease innovation, motivating me to lead Alltrna, a company pioneering engineered tRNA therapies to address a broad range of genetic diseases in a profound way unlike any other genetic modality.”
CRISPR-CAS9 is an innovative gene editing technology to precisely remove or incorporate into the gene of interest; CRISPR-CAS9 shows promising results in HIV treatment. This article outlines the current antiretroviral therapy and its adverse effects but also CRISPR/Cas technology.
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. The Natural History Studies Grants Program funds natural history studies that collect gather data on how rare diseases progress over time without treatment.
The potential therapeutic efficacy of luteolin in the treatment of cervical cancer has been identified. Therefore, we aim to elucidate the mechanism of action of luteolin in the treatment of cervical cancer through a comprehensive approach that integrates metabolomics with bioinformatics.
Oncology drug approvals in H1 2025 In the first half of 2025, the FDA’s Center for Drug Evaluation and Research (CDER) approved a total of 16 novel drugs , with half of these drugs related to the treatment of cancer.
The acquisition will expand AbbVie's exploration of in vivo CAR-T therapy, an ambitious approach that could sidestep some of the difficulties presented by current cell treatments.
Disorders such as Alzheimer’s disease , primary glioblastoma and amyotrophic lateral sclerosis (ALS) all affect the CNS and are considered fatal, while more common conditions, including depression , strokes and epilepsy , require long-term treatments. This makes it difficult to identify relevant and effective treatments.
Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).
While these therapies hold great promise for improving cancer treatment outcomes, their development presents significant challenges, especially in achieving the optimal balance between efficacy and safety. Properly managing these toxicities is crucial to enhancing the safety and therapeutic effectiveness of ADC treatments.
FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.
At the forefront of this transformation is Dr Ebrahim Delpassand, a nuclear medicine physician and the driving force behind RadioMedix (RMX), a radiopharmaceutical company focused on developing targeted diagnostics and therapies. The founding of RadioMedix was born from a recognition of unmet needs in cancer treatment.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.
In the rapidly evolving field of cell and gene therapy (CGT), the ability to manage complex biological data, optimise manufacturing processes, and accelerate drug discovery is crucial. How AI is solving CGTs biggest challenges Cell and gene therapy is inherently complex. Kosten Digital was established to bridge this gap.
Currently, treatment options for patients with relapsed or refractory disease remain limited, and prognosis is poor. Standard single-target CD19-directed CAR T-cell therapies typically enable long-term remissions in roughly 40% of patients, emphasizing a significant unmet need for new and more effective treatment strategies.
Targeting GPCRs to fight inflammatory diseases GPCR-targeting drugs are well known therapies for a range of disease types, including cardio-metabolic, central nervous system and endocrinological disorders. GPCR-targeting therapies such as DT-9046 represent a compelling opportunity in that respect, with major clinical and commercial potential.
These twins simulate how a patients condition might evolve without treatment, enabling researchers to compare the real-world effects of an experimental therapy against predicted outcomes. In 2025, I think well see breakthroughs that allow us to make significant advances in rare diseases, where the data is small.
The deal centers around a medicine that could “shift the treatment paradigm for cardiovascular disease,” Lilly said, but also follows a yearslong in decline in Verve’s share price amid skepticism over the need for such a therapy.
Broad Institute and Bayer extend their cardiovascular alliance By Leah Eisenstadt June 11, 2025 Breadcrumb Home Broad Institute and Bayer extend their cardiovascular alliance Partnership will continue with renewed focus on developing new cardiovascular therapies.
This alliance aims to address mounting challenges in the development and commercialization of mAb therapies and gene therapies, particularly those involving AAV vectors. Biologic drugs, including monoclonal antibodies, have become essential in the treatment of a wide array of conditions, from autoimmune diseases to various cancers.
Obesity treatment is undergoing a major shift, much like the advances seen in cancer care. Phenomix Sciences , led by CEO Mark Bagnall, is using precision medicine to make treatments more targeted and effective. Phenomix Sciences, built on over a decade of clinical research at the Mayo Clinic, is disrupting this outdated approach.
Not only are they chasing new targets, they are also working to understand the biology behind resistance, discover biomarkers that matter and build smarter therapies from the ground up. mCRPC is a particularly aggressive form of the disease, and patients currently have limited treatment options,” said Eleni. “As
8 With the widespread use of induced pluripotent stem cells (iPSCs), organoids can now be generated from the skin or blood cells of both healthy volunteers and disease patients, opening the door to studying various diseases and creating individually customised potential treatments. Organoids in cancer research. Nat Rev Cancer.
This means it can block estrogen receptor activity and also degrade the receptor itself, potentially offering a more effective treatment approach. Clinical Trials: Palazestrant is currently in clinical trials, including Phase 1/2 and Phase 3 studies, for the treatment of ER+, HER2- metastatic breast cancer. ribociclib).
Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. Adapted from an Allen Institute press release.
Drug resistance remains one of the most pressing challenges in oncology drug development, as it is the leading cause of treatment failure. This article explores the role in vitro models are playing in the fight against drug resistance and explains how they can accelerate the development of successful novel therapies.
Antibody therapies have transformed cancer treatment, yet their limits are becoming increasingly clear – particularly when tumours evolve, evade immune detection or develop resistance to existing drugs. The presence of p95HER2 – a shortened version of the receptor that still drives tumour growth – makes treatment even more difficult.
Although it is currently only used in veterinary therapy, carprofen proves to be a molecule with a versatile therapeutic potential for human pharmacotherapy. With ongoing research pushing the boundaries of its potential, carprofen remains a promising candidate for innovation in drug development and treatment strategies.
Flavonoids exhibit significant potential in cancer therapy by inhibiting MMP-2 and MMP-9 activity and expression, primarily through interactions with their active sites and modulation of the MAPK signaling pathway. ABSTRACT Last few decades, extensive research efforts have been dedicated to uncovering novel cancer treatments.
These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. According to Merck, the addition of WINREVAIR to background therapy resulted in a meaningful improvement in patient outcomes, particularly in delaying disease progression and reducing the risk of severe clinical events.
Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.
Teva and Fosun Pharma Forge Strategic Partnership to Develop and Commercialize Innovative Anti-PD1-IL2 Therapy (TEV-56278) in Immuno-Oncology Teva Pharmaceutical Industries Ltd. TEV-56278 is a first-in-class anti-PD1-IL2 ATTENUKINE therapy designed by Teva’s internal team of innovative scientists.
FDA Approves Label Update for Lilly’s Amyvid, Expanding Its Role in Alzheimer’s Disease Diagnosis and Therapy Guidance In a major development for Alzheimer’s diagnostics, Eli Lilly and Company (NYSE: LLY) announced that the U.S.
Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.
The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,
In cases where control or treatment groups show large variability, this could reflect true biological responses to a treatment, and removing such data could reduce the accuracy of the analysis. It is crucial during this phase to strike a balance between removing erroneous data and preserving meaningful biological variability.
Drugging such undruggable targets is essential to develop new therapies for diseases where current treatment options are limited or nonexistent. Tomonori Tamura, Masaharu Kawano, and Itaru Hamachi Chemical Reviews 2024 DOI: 10.1021/acs.chemrev.4c00745
They have interesting patterns of expression in different cancer indications; thus, diverse therapies for attacking these targets have been developed. Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis.
Antibody-drug conjugates (ADCs) are among the most exciting advances in target cancer therapy. They combine the specificity of monoclonal antibodies with the cytotoxic potency of small molecule drugs to focus treatment on malignant cells while reducing damage to healthy cells.
The investigational ADC is built using Daiichi Sankyo’s proprietary DXd technology and represents one of the newest generation of targeted cancer therapies. Current treatment strategies for mCRPC typically begin with ARPIs such as enzalutamide or abiraterone, followed by taxane-based chemotherapy , most commonly docetaxel.
We organize all of the trending information in your field so you don't have to. Join 15,000+ users and stay up to date on the latest articles your peers are reading.
You know about us, now we want to get to know you!
Let's personalize your content
Let's get even more personalized
We recognize your account from another site in our network, please click 'Send Email' below to continue with verifying your account and setting a password.
Let's personalize your content