FDA approves first mesenchymal stromal cell therapy
Mesoblast has received FDA approval for Ryoncil, becoming the first and only cell therapy of its kind to be available in the US.
Markets & regulation
Cell and gene therapy access model launches in US
The sickle cell gene therapies developed by Bluebird Bio and Vertex will be the first to be offered under the new scheme.
BridgeBio wins FDA approval for inherited cardiomyopathy treatment
The approval should provide competition for Pfizer, who has a very similar treatment on the market.
Helen Harrington’s journey: Patience, passion, and problem-solving
From developing treatments for Ebola to mastering karate with her daughters, Dr. Helen Harrington’s journey is one of resilience and curiosity.
FDA approves first-ever gene therapy to be directly administered to the brain
PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.
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.
AstraZeneca’s rare disease therapy Fasenra receives EU approval
The new treatment will be a competitor of GSK’s Nucala (mepolizumab), which was approved in 2018 as the first targeted treatment for the same rare immune disease.
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.
Aldevron’s Anu Codaty on passion, leadership, and innovation
Anu Codaty, VP of global marketing and strategy at Aldevron, is blazing trails in biotech, inspired by a family legacy in medicine and driven to create life-changing therapies.
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).
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.
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).
FDA places hold on Novavax’s COVID-influenza combo vaccine
A report of a case of motor neuropathy in a patient who received the vaccine last year has led the FDA to halt its development while more information is gathered.
Evaxion’s AI-immunology platform shines in multiple clinical trials
Evaxion’s proprietary AI-Immunology platform has shown in clinical trials that it can select clinically relevant cancer vaccine targets.
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.
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.
Symbiosis expands sterile manufacturing with new facility and innovations
As CPHI Milan 2024 approaches, Symbiosis Pharmaceutical Services is gearing up to showcase major expansions and innovations in sterile biopharmaceutical manufacturing.
Quotient Sciences expands with mRNA Focus at CPHI 2024
During the show, Quotient Sciences CEO Thierry Van Nieuwenhove will share insights on how the company is adapting to new FDA regulations.
SGD Pharma to showcase new glass packaging solutions at CPHI 2024
The company will introduce its innovative siliconized moulded glass vials, positioning itself as the only global supplier offering this technology to pharmaceutical standards.
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.
Novartis’ Kisqali receives FDA approval to reduce the risk of breast cancer recurrence
Novartis announced this week that the FDA has approved ribociclib (marketed as Kisqali) for the treatment of people with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR-positive, HER2-negative) stage two or three early...
Cellevate bags €3.2 million to smash viral vector manufacturing bottleneck
The Swedish biotech company Cellevate has raised €3.2 million in a seed round to fuel the commercialization of its nanofiber-based viral vector manufacturing technology in November.
Aurora Biosynthetics launches to boost RNA therapeutics manufacturing in Asia-Pacific
The new company focuses on producing components needed to manufacture RNA-based therapies for which there is increasing demand.
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.
Eli Lilly's eczema drug receives FDA approval one year after its initial rejection
The original concerns of the US regulatory agency over the manufacturing of Eli Lilly’s Ebglyss seem to have been resolved, as the FDA greenlit the antibody drug on Friday.
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.
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.
Creyon Bio secures second patent for AI-powered oligonucleotide engineering
Creyon Bio Inc., a clinical-stage biotechnology company specializing in AI and machine learning-enabled oligonucleotide-based medicines (OBMs), recently announced the issuance of its second US patent.
European Commission grants orphan drug designation for rare disease gene therapy
The European Commission has granted Orphan Drug Designation for Vivet Therapeutics’ gene therapy product VTX-806 for the treatment of Cerebrotendinous Xanthomatosis (CTX).
FDA grants expanded approval for Emergent Bio’s mpox vaccine
The U.S. company Emergent BioSolutions recently announced the FDA approval of its application to expand the indication for ACAM2000 – previously approved for smallpox – to include the prevention of mpox in high-risk individuals.
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.
EU approves Regeneron’s bispecific antibody following US rejection
Just months after facing a rejection in the US, the bispecific antibody odronextamab (Ordspono) has been approved by the European Commission for the treatment of two types of blood cancer in patients that have failed to respond to previous treatments.
EU and UK approve Microbiome Medicines for advanced melanoma and ulcerative colitis
Microbiotica has received the green light from EU and UK regulatory authorities to start phase 1b studies for its microbiome medicines in advanced melanoma and ulcerative colitis.
FDA approves updated mRNA vaccines for COVID-19
The US FDA has granted emergency use authorization to several updated COVID-19 vaccines against a new viral strain that is currently circulating.
FDA approval of Regeneron’s myeloma drug delayed due to manufacturing issues
The US regulator has confirmed a delay to the approval of linvoseltamab while a manufacturing issue is resolved with a third-party provider.
Breaking News: Lecanemab approved in UK but unavailable on NHS
Eisai and Biogen’s new Alzheimer’s treatment, lecanemab, received a green light from UK regulators, marking a significant advance in treating early-stage Alzheimer’s disease.
Adcendo and Multitude target cancer resistance to ADCs in $1BN deal
As antibody-drug conjugates (ADCs) continue to heat up in the industry, Adcendo of Denmark has gained the global rights from Multitude Therapeutics to develop the Chinese company’s ADC for the treatment of cancer outside of the Greater China region.
Researchers develop temperature-sensitive amyloid fibrils for therapy
In a promising advancement, scientists from the University of North Carolina at Chapel Hill and Emory University have developed a novel drug delivery system using synthetic amyloid beta (Aβ) peptides.
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.
Scientists engineer brain parasite as novel drug delivery system
A team of scientists from the universities of Glasgow and Tel Aviv has developed a groundbreaking method to transform the brain parasite Toxoplasma gondii into a potential vehicle for delivering therapeutic treatments directly to brain cells.
Innovative nanoparticle method could completely change therapeutics
In a significant leap forward for therapeutic science, researchers at Cornell University have developed a pioneering method to 'cloak' proteins, enabling their efficient delivery into living cells.
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.
FDA approves Galderma’s Nemluvio for chronic skin condition
The US approval of Nemluvio marks a major milestone for Galderma, a Swiss company developing treatments for a wide range of conditions affecting the skin.
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.