Know What's Happening in Pharma
Know What's Happening in Pharma
FDA approves KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA has granted approval to KRESLADI, a gene‑editing therapy from Rocket Pharmaceuticals that treats severe leukocyte adhesion deficiency‑I in children without a matching bone‑marrow donor. This marks the first FDA‑approved product directly funded by California’s Institute for Regenerative Medicine (CIRM), highlighting the agency’s impact on translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma from GHO Capital
BioWorld’s March 12, 2026 clinic roundup aggregates a suite of data snapshots and special reports spanning biopharma, medical technology, and emerging therapeutic areas. The collection links to analyses on mRNA vaccine research, China’s GLP‑1 market, CAR‑T developments, and the med‑tech outlook for 2026. It serves as a centralized gateway for industry professionals seeking timely insights across multiple sectors. The page also highlights index insights, infographics, and review archives from the past year.
Scientists at the University of Padova synthesized fluorinated psilocin derivatives, identifying compound 4e as a lead that retains serotonergic activity while markedly reducing hallucinogenic effects in mice. In vitro assays showed 4e is a selective partial agonist at 5‑HT2A and...
Can someone explain to me why many of the same folks who love experimenting with peptides (i.e. little to no rigorous safety data) are terrified of vaccines and other drugs that have testing... How does someone square that circle?
Lawrence Livermore National Laboratory and partners applied causal‑inference machine learning to electronic health records of more than 11,000 U.S. veterans with amyotrophic lateral sclerosis. The analysis, published in The Lancet Digital Health, identified 27 existing medications that correlate with longer...
Researchers have identified a streamlined DNA construct that could replace viral carriers in gene‑therapy applications. The study demonstrates that this minimalist DNA format delivers therapeutic genes with efficiency comparable to adeno‑associated viruses while eliciting a weaker immune response. Production of...
The FDA emphasizes that generic medications are as safe and effective as brand‑name drugs while offering substantial cost savings. Over 90% of prescriptions in the United States are filled with generics, reflecting broad market acceptance. To address consumer questions, the...
The FDA launched the Adverse Event Monitoring System (AEMS), a unified dashboard that merges seven previously fragmented safety databases into a single platform. Four databases are already live, with the remaining three slated for integration by May, covering drugs, vaccines,...
The FDA has launched the Adverse Event Monitoring System (AEMS), a unified platform that merges seven previously fragmented safety‑surveillance databases. Four of those databases are live today, with the remaining three slated for integration by May 2026. The agency processes...
Industry experts highlight three intersecting forces reshaping emerging biotech: the persistent funding and regulatory hurdles faced by early‑stage startups, the rapid migration of AI from a supportive tool to an operational backbone, and breakthrough computational methods—including quantum chemistry—that are redefining...
The FDA has compiled reviews of pediatric studies conducted under the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) from 2012 to present. A total of 88 products were studied under BPCA, 544 under PREA,...
The World Health Organization issued a formal warning on 13 February 2026 about a proposed randomized trial in Guinea‑Bissau that would withhold the hepatitis B birth‑dose vaccine from roughly half of 14,000 newborns. The study seeks to measure non‑specific effects...
RFK Jr is trying to get 14 peptides, without data on safety or efficacy, licensed and approved by FDA. His favorite is BPC-157. "Only three small human studies of BPC-157 exist, for instance, the largest of which is a telephone...
Lilly warns GLP-1 knockoffs may be ‘dangerous,’ escalating war with compounders https://t.co/XOKLqIzAES @ByJonGardner $LLY $NVO #obesity
Baseimmune announced a new fibrosis‑focused pipeline leveraging its computational protein design platform to create multi‑pathway immunotherapies, starting with idiopathic pulmonary fibrosis (IPF). The company aims to deliver proof‑of‑concept efficacy data for its lead IPF program in 2026‑2027, addressing the limitations...
A meta‑analysis of six randomized trials involving 5,924 stable CAD patients with atrial fibrillation found that oral anticoagulant (OAC) monotherapy reduced cardiovascular mortality by 31% and major bleeding by 54% compared with OAC plus a single antiplatelet. The benefit persisted...
AI is reshaping clinical trial oversight by moving from periodic checks to continuous, real‑time intelligence. Advanced analytics and machine learning now power predictive, risk‑based monitoring that aggregates data from decentralized sites, wearables, ePROs and multiple clinical systems. This integration enables...
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Snakebite envenoming kills over 125,000 people each year and leaves three times as many disabled, while current horse‑derived antivenoms trigger severe allergic reactions in nearly half of patients. The high cost—up to $100,000 per course—and limited hospital access leave rural...
BridgeBio Pharma reported that its small‑molecule candidate BBP‑418 produced a 1.8‑fold increase in the α‑dystroglycan biomarker in a Phase 3 FORTIFY trial for limb‑girdle muscular dystrophy type 2I/R9, with effects sustained through 12 months. The trial enrolled 81 patients and also showed...
Aplagon Therapeutics has administered the first dose of its intravenous APAC candidate to a patient in a Phase 2a (HEALING) trial for peripheral arterial occlusive disease leading to chronic limb‑threatening ischemia in Finland. The study will enroll approximately 42 CLTI...
Pierre Fabre and $ATRA said FDA meeting to discuss the Ebvallo CRL has been scheduled. (Likely occuring within the next month.)
Jacobs and colleagues present a state‑of‑the‑art review of psilocybin and MDMA‑assisted therapies, highlighting their potential for treatment‑resistant depression and PTSD. The authors emphasize the distinct, session‑based paradigm that leverages acute neurobiological changes to produce lasting clinical benefits. However, they also...
The FDA issued draft guidance that could lower biosimilar testing requirements by permitting foreign comparator data, potentially cutting development costs by about half. Over 80 biosimilars have been approved in the U.S. since the first approval 11 years ago, yet...
The FDA is targeting telehealth marketing of GLP-1 drugs. Who’s prescribing them? Medical groups face ‘tricky’ questions when partners make misleading claims via @KatieMPalmer https://t.co/pf4Fwa7W7r
Researchers at Michigan State University used mouse models and CRISPR technology to map how cocaine rewires the ventral hippocampus‑nucleus accumbens (vHPC‑NAc) circuit. They discovered that the transcription factor ΔFosB acts as a molecular switch, accumulating with repeated cocaine exposure and...
Insulet presented data from its EVOLUTION 2C feasibility study of a fully closed‑loop (FCL) automated insulin delivery system for adults with type 2 diabetes at ATTD'26. In the 24‑patient trial, the final algorithm raised time‑in‑range (TIR) to an average 68%,...
Bavarian Nordic has signed a manufacturing agreement with the Serum Institute of India to transfer full technology for its chikungunya vaccine, CHIKV VLP (branded Vimkunya). The deal replaces an earlier partnership with Biological E and builds on the companies' existing mpox‑vaccine collaboration....
GLP‑1 drugs such as semaglutide have surged from diabetes treatment to a mass‑market weight‑loss solution, with roughly 12.4% of Americans now using them. Global sales are projected to climb from $50‑60 billion today to over $135 billion within a decade, driven largely...
The FDA unveiled the Adverse Event Monitoring System (AEMS), a unified platform that shifts drug safety surveillance from quarterly updates to real‑time reporting and is expected to save roughly $120 million over five years. Simultaneously, a direct‑to‑employer drug‑purchasing model is gaining...
Eli Lilly warned that compounded versions of its GLP‑1 drugs Mounjaro and Zepbound, when mixed with vitamin B12, may contain unsafe impurities. The alert follows growing off‑label compounding as demand outpaces supply. Lilly’s notice urges clinicians and patients to verify product sources...
The FDA announced that a single pivotal trial can now satisfy efficacy requirements for new drug applications, replacing the previous two‑study mandate. This change raises expectations for data depth, quality, and risk‑based management throughout the trial lifecycle. Guests Oxana Iliach...
Dr. Steven Quay, CEO of Atossa Therapeutics, highlighted a new focus on tolerability and prevention in estrogen‑receptor‑positive breast cancer, where five‑year survival now exceeds 90%. Atossa is developing a next‑generation SERM that aims to reduce side‑effects while maintaining efficacy and...
Researchers have introduced INSITE, an injectable platform that combines primary human hepatocytes with hydrogel microspheres to form self‑assembling, vascularizable tissue ensembles in situ. Using ultrasound guidance, the scaffold is delivered to an ectopic site where it integrates with host vasculature...
Gesynta Pharma has begun dosing the first patient in its Phase II NOVA trial, evaluating oral vipoglanstat for endometriosis. The double‑blind, placebo‑controlled study will enroll about 190 women across Europe and compare two dose levels against placebo over four menstrual cycles....
GSK has pledged £11 million to launch the Modelling‑Informed Medicine Centre (MiMeC), a joint venture with Imperial College London and the University of Oxford. The centre will develop digital twins—computer‑based replicas of lungs, liver and kidneys—to run in‑silico experiments and speed...
Researchers are expanding CAR‑T cell therapy beyond cancer to treat autoimmune diseases such as lupus and rheumatoid arthritis. Sail Biomedicine’s chief products and operations officer highlighted on the Pharmaceutical Executive podcast that the company has pivoted to RNA‑based CAR‑T platforms,...
Eli Lilly has issued a public warning after testing revealed a significant impurity formed when tirzepatide is compounded with vitamin B12. The impurity’s health effects are unknown, and the company has alerted the FDA and urged patients to consult physicians....
George Medicines has signed an exclusive licensing and supply agreement with South Korea’s Ahngook Pharmaceutical to bring its triple‑combination antihypertensive pill, GMRx2, to the Korean market. The single‑pill formulation blends telmisartan, amlodipine and indapamide in three dose strengths, aiming for...
C2N Diagnostics has signed a partnership with BeauBrain Healthcare to introduce its PrecivityAD2 blood test for Alzheimer’s disease in South Korea, targeting patients aged 50 and older with mild cognitive impairment or dementia. Clinical studies published in JAMA and npj...
A retrospective cohort of 169 critically ill patients receiving tigecycline showed significant reductions in blood glucose at three daily time points. Hypoglycemia occurred in 11.2% of patients and was linked to a nearly four‑fold increase in 28‑day mortality (OR 3.83). Larger...
If you are curious about seeing what may be the last competent FDA advisory committee left standing in action as they contribute to determining access to flu shots next fall, join me, @jessicamalaty, & @SaveAmericaMvm at 12:30 pm ET on...
The UK government has negotiated a UK‑US pharmaceutical trade deal that raises NICE’s cost‑effectiveness threshold from £20‑30k to £25‑35k per QALY. The higher threshold will deem more expensive drugs cost‑effective, increasing NHS drug spend while reducing rebate rates from 22.9%...
Mushroom mycologist Paul Stamets presented data indicating that Agarikon (Fomitopsis officinalis) mycelium possesses broad antiviral properties. In two placebo‑controlled trials, a combined Agarikon‑turkey‑tail extract reduced COVID‑19 vaccine side effects, sustained antibody titers, and accelerated recovery in hospitalized patients. The research,...
The FDA has opened a public docket to solicit comments on its long‑standing Scale‑Up and Post‑Approval Changes (SUPAC) guidances for immediate‑release solid oral, non‑sterile semisolid, modified‑release solid oral dosage forms and the manufacturing equipment addendum. The agency seeks feedback on...
New tissue models could help researchers develop drugs for liver disease by Anne Trafton @MIT Learn more: https://t.co/hkeRZ0W47B #HealthTech #EmergingTech #TechForGood #Innovation https://t.co/LNvft8iX9M
Capricor Therapeutics reported fourth‑quarter 2025 revenue of $16 million, a modest 4% year‑over‑year increase, while gross margin rose to 86% driven by higher selling prices and manufacturing efficiencies. Net loss widened to $11.9 million as operating expenses outpaced revenue growth, but the...
Vertex Pharmaceuticals reported Q4 2025 revenue of $3.2 billion, a 10% quarterly increase, and $12 billion for the full year, up 9% year‑over‑year. Gross margin reached 85.7% and non‑GAAP net income rose 24% to $1.3 billion, underscoring strong profitability. New product...
Nektar Therapeutics reported strong Q4 2020 results, highlighting robust progress on its cytokine platform. The IL‑2 agonist BEMPEG demonstrated a median progression‑free survival of 30.9 months in melanoma and is now enrolled in six registrational trials across melanoma, renal, bladder,...
Researchers at Harvard’s Wyss Institute and Dana‑Farber unveiled DoriVac, a DNA origami‑based vaccine platform that delivers antigens and adjuvants on a self‑folding nanostructure. In pre‑clinical mouse studies and a human lymph‑node‑on‑a‑chip model, DoriVac generated antibody and T‑cell responses comparable to...
Vima Therapeutics announced an additional $40 million raise, taking its Series A funding to $100 million. The capital will support parallel Phase 2 trials of its lead candidate VIM0423 in isolated dystonia and Parkinson’s disease, expanding the startup’s focus beyond the rare movement disorder....
The FDA placed a clinical hold on Regenxbio’s RGX‑111 and RGX‑121 gene‑therapy trials after a pediatric MPS I patient developed a tumor four years post‑treatment. The case marks the first documented long‑latency cancer linked to an adeno‑associated virus (AAV) vector in...
