SpliceBio gets FDA green light to start Phase 1/2 trial for inherited eye disease gene therapy
The company will start enrolling patients with Stargardt disease in the first half of 2025.
Clinical development
Biohaven’s myostatin inhibitor fails in phase 3 spinal muscular atrophy trial
Despite disappointing results in the genetic neurodegenerative disorder, the drug shows potential for treating obesity.
Tran Pham: Breaking barriers and building biopharma solutions
From discovering her knack for chemistry in high school to proving her expertise in male-dominated spaces, Tran Pham has built a dynamic career in biopharma.
Innovative COA tools to reduce steroid toxicity in autoimmune care
Collaboration to leverage Steritas’ STOX Suite in developing innovative treatments for autoimmune diseases.
FDA removes clinical hold on Novavax’s COVID-19-influenza combo vaccine
The FDA had put a clinical hold on Novavax’s COVID-19-influenza combo vaccine after an adverse event in a patient.
Top 10 rare disease pharma sees 10.4% rise in therapy start
A new case study released by AllazoHealth reveals that a leading rare disease pharmaceutical company achieved a 10.4% increase in therapy initiation, driven by AI-enabled patient outreach and personalized communications.
CRO NovAliX teams with Bruker to advance drug development
In a move set to transform drug discovery, CRO NovAliX has partnered with Bruker, a leader in scientific instruments, through a strategic investment aimed at enhancing drug development success rates.
EC approves Janssen’s Darzalex-SC combo for multiple myeloma
New Darzelex SC quadruplet regimen approved by European Commission for newly diagnosed multiple myeloma patients.
Alto Neuroscience’s depression drug trial fails at phase 2
The negative trial results have tanked the California-based biotech’s market value, with a 70% reduction in share price over the last week.
The FDA has approved Pfizer’s RSV vaccine Abrysvo for adults aged 18 to 59
The US pharma giant Pfizer recently announced that its respiratory syncytial virus (RSV) vaccine Abrysvo has received FDA approval in adults aged 18 to 59 who are at an increased risk of developing lower respiratory tract disease caused by RSV (RSV-LRTD).
Adcentrx gets FDA clearance for phase 1 trial of ADC for tumors
The FDA has given clearance to Adcentrx Therapeutics’ IND application for an antibody-drug conjugate (ADC) targeting solid tumors.
Innovations in mass spectrometry accelerate the shift toward precision medicine
Thermo Fisher’s John Lesica discusses how mass spectrometry technology enables scientific breakthroughs in genomics and proteomics to provide valuable insights for drug discovery and development.
FDA approves targeted therapy for gastric cancer and its companion diagnostic
The FDA has simultaneously granted approval to Astellas’ cancer drug Vyloy and to a companion diagnostic test developed by Roche.
Investors and big pharma no longer avoiding techbio companies
Investors and big pharma companies are waking up to the potential of techbio companies after years of avoidance, say experts at TechBio UK 2024, an event run by the BioIndustry Association (BIA).
Preparing for the US Biosecure Act - what does it mean for the pharma industry?
The US Biosecure Act was passed by the House of Representatives in September and seems likely to become law.
Women in Science - SCIEX Rebekah Sayers' on encouraging curiosity
Rebekah's father passed on his passion for science and problem solving to her and much of her childhood was spent doing fun hands-on activities from taking apart household mechanics to building rockets.
Resolution Therapeutics scores £63.5 million Series B
The UK cell therapy biotech will now advance its first candidate cell therapy to clinical trials for treatment of liver disease.
CPHI Milan 2024: Enzene targets cost savings and efficiency
Enzene's monoclonal antibody production costs to dip below $40 per gram by 2025.
AI study shows GLP-1s could help prevent heart disease in millions
Dandelion Health used its new clinical AI marketplace to analyze real-world data, showing that GLP-1 drugs may help prevent cardiovascular disease (CVD) for a seven times larger population than previously researched.
Women in science - Alison O'Neill at YPrime on thinking out loud and collaborating
Alison has more than 35 years of broad experience delivering clinical trials to sponsors with a patient centric mindset.
MinervaX and Wacker Biotech partner to scale up GBS vaccine production
MinervaX and Wacker Biotech announced yesterday that they have entered into a manufacturing agreement for MinervaX’s prophylactic vaccine against Group B Streptococcus infections.
Vaxess' Rachel Sha - 'own the seat at the table that you have and have confidence'
Rachel Sha has had a colorful career not without its challenges, but she urges women in the science industry to have confidence and answer hard questions.
Eli Lilly reports positive phase 3 results for a weekly insulin formulation
New data from the QWINT clinical trial may get Eli Lilly closer to bringing a once-weekly insulin injection to the US, following the FDA’s rejection of Novo Nordisk’s own weekly insulin formulation earlier this year.
How modern trial designs propel cancer therapies to the market - interview
Clinical trials are becoming increasingly complex as emerging technologies such as immunotherapy enter the mainstream.
FDA lifts partial clinical hold on phase 3 low blood sugar treatment
Rare disease biotech Rezolute recently announced that the FDA has lifted the partial clinical hold on RZ358 (ersodetug), a novel therapy in development for the treatment of low blood sugar (hypoglycemia) resulting from congenital hyperinsulinism.
Neuromuscular diseases in crosshairs in Forge Biologics gene therapy collaboration
Forge Biologics will help with the manufacturing of adeno-associated viruses (AAVs) in a collaboration with the non-profit Muscular Dystrophy Association (MDA) to develop gene therapies for neuromuscular diseases.
Therapy for aggressive brain cancer receives FDA clearance to start phase 1
Adaptin Bio has announced that the FDA has given the green light to start phase 1 clinical trials for its program APTN-101 targeting glioblastoma, the most common and aggressive form of brain cancer.
Candid Therapeutics launches with $370m to tackle autoimmunity with T-cell engagers
The San Diego-based biotech company Candid Therapeutics has launched with a huge $370 million with the aim to develop T-cell engager therapies for autoimmune diseases.
Neurocrine’s schizophrenia drug succeeds at phase 2, but dosing questions remain
Neurocrine Biosciences has achieved late-stage success with muscarinic M4 selective agonist in schizophrenia.
New oral cholera vaccine brings hope for global public health
An international collaboration led by Hilleman Laboratories has confirmed the launch of a new oral cholera vaccine called HILLCHOL, hoping to address significant global shortages.
First bispecific CAR-T therapy for MS receives green light from FDA to start phase 1
ImmPACT Bio has received FDA clearance to start clinical trials for its CD19/CD20 bispecific CAR-T cell therapy in multiple sclerosis (MS) patients.
Breaking barriers in science: Thermo Fisher's Melanie Adams-Cioaba on innovation and inclusion
In the rapidly evolving field of drug discovery, Melanie Adams-Cioaba stands at the forefront, leading transformative advancements in cryo-electron microscopy (cryo-EM) at Thermo Fisher Scientific.
Breakthrough delivery system could transform Alzheimer’s treatment
In a groundbreaking development, a team of biochemical engineers from South Korea has unveiled a new exosome-based delivery system, termed MAPLEX, that holds the potential to transform treatments for various diseases, including Alzheimer’s.
Injectable hydrogels revolutionize cell and drug delivery for tissue repair
Recent advancements in injectable hydrogels are setting the stage for a new era in tissue engineering and regenerative medicine.
Bavarian Nordic readies 2 million vaccine doses to fight mpox outbreak
The Danish company Bavarian Nordic plans to supply up to 2 million vaccine doses this year in response to the World Health Organization’s declaration of mpox as a Public Health Emergency of International Concern (PHEIC) earlier this month.
FDA lifts hold on BioNTech and MediLink's ADC trial After safety revisions
Two months after placing a partial hold on a phase 1 clinical trial evaluating an antibody-drug conjugate (ADC) in cancer patients, the FDA is allowing the study to proceed with the lower doses of the treatment.
Advice for young women in science - stay curious and inquisitive the rest will follow
Katarina Wikstrom has worked for over 20 years in the field of translational molecular oncology and holds the position of VP global product development at Almac Diagnostic Services.
Halda Therapeutics raises $126M to take 'hold and kill' precision cancer drugs to the clinic
The investment round will fund the first clinical trials for an innovative therapeutic modality designed to overcome drug resistance in patients with solid tumors.
Citius Pharma receives FDA approval for immunotherapy targeting rare blood cancer
The FDA has given the green light to Lymphir, the first immunotherapy for the treatment of cutaneous T-cell lymphoma (CTCL) to get approval in over five years.
How personal loss and hope drove Elaine Maynard’s work in clinical trials and patient-centric technologies
Driven by personal loss and inspired by the progress in disease management, Elaine has dedicated more than 20 years to the clinical trial technology industry.
Sangamo signs licensing agreement with Genentech worth up to $1.9 billion
The agreement will give Genentech access to Sangamo’s capsid delivery platform and epigenetic regulation technology with applications in neurodegenerative disease.
FDA grants breakthrough designation to Amgen and AstraZeneca’s Tezspire
With the FDA’s endorsement, Amgen and AstraZeneca are getting closer to adding chronic obstructive pulmonary disease (COPD) as a new indication to Tezspire.
Terran Biosciences - the difficulties of getting new schizophrenia treatments to market
Experience with severe mental health disorders in close friends and family gave Sam Clark the drive to found Terran Biosciences to explore new approaches to therapeutics in neuropsychiatry.
Adaptimmune’s Tecelra becomes the first FDA-approved cell therapy for a solid tumor
The FDA has granted accelerated approval to Adaptimmune’s Tecelra, a T cell therapy for the treatment of synovial sarcoma when other lines of treatment do not work.
Exploring sleep apnea solutions: Skye and Beacon's innovative obesity trial collaboration
In this Q&A, we delve into the collaboration between Skye Bioscience and Beacon Biosignals to enhance the evaluation of nimacimab, a promising treatment for obesity and its comorbid conditions, including obstructive sleep apnea (OSA).
AIRNA raises $90 million Series A to bring RNA editing therapies to the clinic
AIRNA plans to bring RNA editing therapies to the clinic starting with a treatment for the genetic disease alpha-1 antitrypsin deficiency.
Ann Graham from her shocking cancer diagnosis to supporting youngsters battling the disease
Ann Graham changed the direction of her career after being treated for osteocarcoma - a type of cancer that usually affects children and young people.
Novartis enlists Dren Bio to co-develop bispecific antibodies for $3 billion
Eager to tap into a high-growth market, Novartis has sealed a pact worth up to $3 billion with Dren Bio to co-develop antibody treatments for cancer.
Explore how Lonza's experts tackle parenteral drug and bioconjugate development challenges
In the rapidly evolving field of pharmaceuticals, the development of parenteral drugs and bioconjugates presents unique challenges and opportunities.