Know What's Happening in Pharma
Know What's Happening in Pharma
FDA greenlights KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA approved KRESLADI, a gene‑editing treatment from Rocket Pharmaceuticals for severe leukocyte adhesion deficiency‑I in children lacking a bone‑marrow donor. It is the first product directly funded by California’s Institute for Regenerative Medicine, highlighting CIRM’s role in translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma
A mother’s plea after the FDA halted the Elevidys gene‑therapy trial for her son with Duchenne muscular dystrophy has reignited criticism of the 2018 Right‑to‑Try Act. The agency’s decision, triggered by two deaths in a broader trial, cuts off a potential lifeline for patients and underscores the clash between safety oversight and rapid access.
The view from my hotel room in Chamonix is quite spectacular. I spoke yesterday at a global event organized by a major pharma company. They organized it near Mt. Blanc to make the country managers literally aim higher and higher. I...
Japan’s Ministry of Health, Labour and Welfare approved Dupixent (dupilumab) for adults with moderate-to-severe bullous pemphigoid, marking the first targeted therapy for the disease in the country. The approval is based on the phase II/III ADEPT trial, which enrolled 106...
Maze Therapeutics reported that its Phase 2 trial of the genetic kidney disease candidate MZ‑001 achieved its primary efficacy and safety goals, showing a roughly 30% slowdown in eGFR decline versus placebo. The double‑blind study enrolled 150 patients with autosomal dominant...
WuXi Biologics reported record 2025 results, with revenue up 16.7% and IFRS gross profit climbing 30.9%, lifting its gross margin to 46%. The growth was driven by expanding research, development and manufacturing contracts and tighter cost control across its global...
Researchers at Baylor College of Medicine have identified a brain‑based pathway that underlies metformin’s glucose‑lowering effect. The study shows that metformin suppresses the Rap1 protein in the ventromedial hypothalamus, a region critical for whole‑body glucose regulation. Mice lacking hypothalamic Rap1...
Researchers at the Salk Institute identified the protein Med14 as the molecular bridge that links GLP‑1 agonist drugs to broad genomic responses that enhance pancreatic beta‑cell health. The team showed that phosphorylation of Med14 is essential for activating gene programs...
Novo Nordisk and United Laboratories reported Phase‑II data for the GLP‑1 agonist UBT251 in 211 Chinese patients with type‑2 diabetes. Over 24 weeks, UBT251 achieved a 2.16 % HbA1c reduction, outperforming semaglutide’s 1.77 % and placebo’s 0.66 % from a baseline of 8.12 %....
Gilead announced a $2.1 billion acquisition of Ouro Medicines and its T‑cell engager OM336, planning to split the deal with long‑time partner Galapagos. Johnson & Johnson and Protagonist Therapeutics secured FDA approval for Icotyde, an IL‑23 receptor blocker that becomes Protagonist’s...
Basecamp Research has launched the Trillion Gene Atlas, a platform that will collect and model genetic information from more than 100 million species, expanding known evolutionary diversity by roughly 100‑fold. The initiative aims to give AI models a vastly broader biological...
Merck & Co. is in advanced talks to acquire Terns Pharmaceuticals in an all‑cash transaction valued at about $6 billion. The deal would add a promising rare‑cancer therapy to Merck’s portfolio and could be announced as early as Wednesday.
A multinational research team has created magnetic silk‑iron nanocomposite particles that can be steered with external magnetic fields to deliver therapeutics directly to otherwise inaccessible tissues. The breakthrough promises higher efficacy and lower side‑effects for treatments ranging from cancer to...
Pfizer and French biotech Valneva announced that their experimental Lyme disease vaccine, PF-07307405, achieved 73.2% efficacy in a Phase III trial, marking the most advanced effort to bring a U.S. Lyme vaccine to market despite missing its primary statistical benchmark. The...
A genomic survey of over 18,000 *Escherichia coli* genomes has mapped 90 capsular K‑loci, revealing that five capsule types (K1, K5, K52, K2, K14) cause more than half of bloodstream and urinary‑tract infections in Europe. The study links these high‑risk...
Researchers at Australia’s Peter Doherty Institute have created a portable, CRISPR‑based diagnostic that simultaneously detects syphilis, HSV, chlamydia and gonorrhea in under an hour. The assay also identifies a key antibiotic‑resistance gene in gonorrhea, delivering 97‑100% accuracy compared with laboratory...
Merck (MSD) has entered a multi‑year research partnership with Quotient Therapeutics to uncover new drug targets for inflammatory bowel disease using Quotient’s somatic genomics platform. The deal provides Quotient with $20 million upfront and up to $2.2 billion in regulatory, development and...
Jacob Van Naarden, formerly COO of Loxo Oncology, joined Eli Lilly after its $8 billion cash acquisition of Loxo in 2018. He now serves as president of Lilly Oncology and head of business development, overseeing the company’s dealmaking and pipeline expansion. The purchase...
Ionis Pharmaceuticals received FDA acceptance of its new drug application for zilganersen and a priority‑review designation for treating Alexander disease, with a PDUFA target action date of September 22, 2026. The Phase III trial enrolled 54 patients aged 1.5 to 53...
In this episode, attorney Harrison James explains how the Racketeer Influenced and Corrupt Organizations Act (RICO), originally aimed at organized crime, is being used in a landmark civil class action against Takeda Pharmaceuticals and Eli Lilly over the diabetes drug Actos....
French imaging contrast agent maker Guerbet is confronting a severe FDA warning after inspectors found significant good manufacturing practice violations at its Raleigh, North Carolina plant. The citation has already depressed Americas revenue by 4% year‑over‑year and reduced MRI‑related sales,...
Cybin Inc. announced topline results from a Phase 2 trial of its anxiety drug candidate HLP004, showing a 10‑point reduction on a standard anxiety rating scale. The study involved 36 patients already on antidepressants, and the benefit persisted for at least...
Trace Neuroscience, launched Nov 2024, is developing an antisense drug, TRCN‑1023, to restore UNC13A protein function in ALS patients. The target emerged from parallel discoveries at UCL and Stanford, and the company plans intrathecal delivery and biomarker‑guided trials. Leveraging lessons...
Researchers at Trinity College Dublin have demonstrated that a single dose of interferon‑gamma can “train” human macrophages to more effectively kill drug‑resistant bacteria such as MRSA and Mycobacterium tuberculosis. The IFN‑γ‑trained cells undergo epigenetic reprogramming, rely on glutamine metabolism, and...
The recent study showing D+Q causes demyelination in brain cells leads to more questions than it answers: (I only read the abstract cause paper new & paywalled.) D+Q has been used a lot in mice & humans. Why hasn't this been noticed...
Based on previous form $MRK will probably now rerun the $TERN study, just to prove to itself that the data were real.
Researchers at the University of Illinois Chicago have repurposed the FDA‑approved cancer drug doxorubicin to combat drug‑resistant herpes simplex virus type 1 (HSV‑1). Using their AI‑driven platform HerpDock, they identified doxorubicin’s ability to block the PI3K‑AKT‑mTOR pathway that the virus exploits,...
For those wondering if $MRK might be outbid, here are the deal termination terms from $TERN https://t.co/TbM3rWyEoG
It's amazing that 25 years after Gleevec we are still watching drug companies pay billions for new drugs for CML. And that, in Adam's previous story, a once deadly cancer was referred to as a chronic disease. Anyway, $MRK buys $TERN...
The FBI announced indictments against Shandong Believe Chemical Company, Shandong Ranhang Biotechnology and six Chinese citizens for exporting fentanyl precursors to the Gulf Cartel. The charges, part of Operation Box Cutter, highlight a new level of cooperation between U.S. and Chinese...
$AVXL pulled its blarcamesine Alzheimer's application from the EMA after regulators there said there was no fking way it was going to approve a drug that does not work.
$NVO triple G drug at 2.16 A1c reduction at only week 24 vs a lower 1.9 A1c reduction at a longer 40 weeks for the max dose of the $LLY triple G. But Novo has no pipeline…? 🤷🏻♂️ https://t.co/CFcnGZGMqt
Lenz Therapeutics reported a Q4 loss of $1.16 per share on $1.6 million in revenue, far below analysts' expectations of a $0.90 loss on $3.1 million. The miss sent the biotech’s shares down 11.2% as investors reacted to higher SG&A costs and...
PandaOmics and Lobster hybrid for novel target discovery and disease hypothesis research. @steipete will have serious impact on human life
The article outlines BioCentury’s cookie policy, detailing the categories of cookies used on its website—strictly necessary, functional, marketing, advertising, and analytics. Each type is described in terms of purpose, activation status, and impact on user experience. The policy emphasizes that...
University of Pennsylvania bioengineers have created a redesigned lipid nanoparticle, aroLNP, that delivers at least ten‑fold less mRNA to the liver while preserving lymph‑node uptake. The breakthrough could lower the dose needed for effective vaccination and expand mRNA therapeutics.
Intelligent and miniaturized drug delivery devices (IMDDDs) combine biotechnology, AI, electronics, and novel materials to provide precise, programmable drug release inside the body. These platforms integrate real-time sensing with adaptive control, enabling dose adjustments based on biomarkers such as glucose...
The U.S. FDA has approved a label update for Ferring Pharmaceuticals' ADSTILADRIN® (nadofaragene firadenovec‑vncg) that permits an accelerated water‑bath thawing process completed in roughly 25 minutes. The therapy, the only FDA‑approved non‑replicating intravesical gene‑therapy for high‑risk BCG‑unresponsive non‑muscle invasive bladder...
The Federal Trade Commission and CVS Caremark have reached a settlement that resolves all claims alleging the pharmacy‑benefit manager inflated insulin prices through anticompetitive rebate practices. Court filings on March 23 indicate the agreement ends the FTC’s lawsuit in its...
Sen. Josh Hawley (R-MO) has demanded that Danco Labs, the maker of mifepristone and misoprostol, turn over any internal communications it has had with the FDA, telehealth providers, pharmacists, patients, state medical boards, and attorneys general concerning the safety of...
Doctors for America met with bipartisan lawmakers to flag several regulatory concerns at the FDA. The group warned that “America First” provisions in the upcoming PDUFA negotiations could increase user fees and hinder drug access, while the agency’s reliance on...
Absci Corporation announced that its AI‑designed antibody ABS‑201 has been dosed in the first three single‑ascending‑dose cohorts of the Phase 1/2a HEADLINE trial, showing favorable safety and early hair‑growth signals in human ex vivo studies. The company also unveiled Origin‑1, a generative‑AI...
Biomea Fusion announced its FY 2025 results, reporting $56.2 million in cash and a net loss of $61.8 million, roughly half the loss recorded a year earlier. The company highlighted durable 52‑week HbA1c reductions of 1.2 % from icovamenib in type 2 diabetes and launched...
Bristol Myers Squibb received FDA approval expanding Sotyktu (deucravacitinib) to treat adults with active psoriatic arthritis, making it the first oral selective TYK2 inhibitor for this indication. The label extension is backed by the POETYK PsA-1 and PsA-2 trials, where...
Karyopharm Therapeutics announced top‑line data from its Phase III SENTRY trial, which evaluated selinexor combined with ruxolitinib in patients with myelofibrosis. The company reported that the regimen met its primary efficacy endpoints and demonstrated a manageable safety profile. Alongside the clinical...
A JAMA Network Open study examined 64 pharmaceutical kickback settlements from 2000 to 2025, uncovering $10.25 billion in penalties. The drugs at the center of those cases generated $458.6 billion in U.S. sales, meaning fines represented just 2.2 percent of related revenue. The...
BioAge Labs reported full‑year 2025 results, highlighting positive Phase 1 data for its oral NLRP3 inhibitor BGE‑102, which achieved up to 86% reduction in hsCRP and strong suppression of IL‑1β, IL‑6, and fibrinogen. The company announced a Phase 2a cardiovascular risk trial...
The FDA issued a final rule on March 5, 2026 establishing a uniform 12‑digit National Drug Code (NDC) that will replace the current variable 10‑digit format, with an effective date of March 7, 2033 and a seven‑year preparation window followed by a three‑year transition...
CVS Health announced a proposed settlement with the Federal Trade Commission concerning its insulin pricing practices. The agreement, negotiated by CVS’s pharmacy benefit manager Caremark, is modeled after the FTC’s recent deal with Express Scripts, a Cigna subsidiary. While the...
Researchers analyzed real‑world claims data from 12,245 Crohn’s disease patients treated between 2016 and 2022 to compare safety outcomes across five advanced therapies, including TNF antagonists, vedolizumab, ustekinumab, risankizumab, and upadacitinib. Incidence rates for serious infections, major adverse cardiovascular events,...
Aurinia Pharmaceuticals' shares climbed about 6% to $14.88 after board chair Kevin Tang was appointed chief executive officer, accompanied by a slate of new senior executives. The leadership overhaul is expected to reshape the company's financial strategy and investor outlook.
