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
The WHO’s INN proposed list 134, released in early 2026, reveals several late‑stage drug candidates that were previously hidden from public view. Notably, two NLRP3 inhibitors—abdenoflast and parunoflast—appear to map to Eli Lilly’s newly acquired Ventyx assets VTX2735 and VTX3232, both showing promising Phase 2 data. The list also discloses LRRK2 inhibitors liradasertib and pelerasertib, which likely correspond to Denali Therapeutics’ DNL151 (BIIB122) and Halia Therapeutics’ HT‑4253, indicating continued investment in neuro‑degenerative targets. These disclosures provide a rare glimpse into the strategic pipelines of major biotech players.
Novo Nordisk announced a €432 million ($506 million) investment to expand its Athlone, Ireland facility, increasing capacity for oral products such as the newly launched Wegovy pill. The expansion is intended to secure supply outside the United States and help the company...
Note for $TGTX, this year has $100m in expenses building inventory for Subq - from a theoretical accounting perspective should really be capitalized (and then written-off if failed or amortized in year of sale), but in actuality gets deducted from...
CPHI Middle East will return to Riyadh in May 2026, building on the 2024 edition that attracted over 30,000 visitors from more than 100 countries. The event promises expanded networking opportunities, direct market access, and the latest regulatory and innovation...
I used to teach that osteoarthritis was "wear and tear" — lose weight, take painkillers, wait for a knee replacement. A study just published in Cell Metabolism proved that wrong. Semaglutide (Ozempic) didn't just reduce joint pain in osteoarthritis patients — it...
Bioxytran announced positive phase 1b/2a data for its oral antiviral ProLectin‑M in a randomized, double‑blind, placebo‑controlled trial of 39 mild‑to‑moderate COVID‑19 patients in India. The highest dose (16,800 mg/day) achieved viral clearance in 90% of participants by day 5 versus 20% on placebo,...
The Module 2, Section 2 lecture introduces the druggable interactome, compiling key resources that map the human druggable genome, protein expression, kinase families, transcription‑factor proteomics, GPCRs, and ion‑channel complexes. It highlights quantitative estimates—over 3,000 proteins deemed druggable and hundreds of actionable kinases—while...
The large‑cap pharmaceutical sector is rebounding in 2026 after a muted 2025, buoyed by strong quarterly results, aggressive M&A, and robust pipeline activity. Zacks ranks the industry at #172, but it posted a 10.8% gain over the past year, outpacing...
The European Union has granted approval for Vueway (gadopiclenol) to be used in neonates, infants, and toddlers, marking a pivotal expansion of pediatric MRI contrast agents. Vueway delivers diagnostic‑grade imaging at half the dose of conventional macrocyclic GBCAs, thanks to...
Biotech Aardvark Therapeutics announced a voluntary pause of the Phase 3 HERO trial for its Prader‑Willi drug ARD‑101 after routine safety monitoring identified reversible cardiac observations in healthy volunteers receiving supratherapeutic doses. The pause affects both the main trial and an...
The Duchenne muscular dystrophy patient & advocacy community keeps a gene therapy and multiple drugs on the US market, stopping the FDA from taking action, despite failed confirmatory clinical trials showing no efficacy and questionable safety. The Huntington's disease patient and...
Merck and Pfizer’s Keytruda‑Padcev doublet delivered a dramatic survival advantage in the Phase 3 EV‑304 study of muscle‑invasive bladder cancer. The regimen cut the risk of disease progression, recurrence or death by 47% compared with gemcitabine‑cisplatin and lowered overall‑mortality risk by...
A recent open‑access study demonstrates that hydrogen‑deuterium exchange mass spectrometry (HDX‑MS) can reliably map binding sites of extremely weak fragment hits (up to 7 mM KD) against Cyclophilin D. By optimizing protein concentration at 10 µM and testing fragments at 2.5‑10 mM, the...
Chinese drugmakers, led by Jiangsu Hengrui Pharmaceuticals, are emerging as global biotech contenders. Hengrui topped 2024 trial sponsorship, boasts a pipeline of 100 investigational drugs and 400 trials, and secured a $500 million partnership with GSK that could yield $12 billion. Analysts...
$QURE AMT-130 update: Still blocked The FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for...
AI of whole slide images for cancer molecular markers is not ready for clinical use due to confounding and biases ("shortcut learning"), supported by multiple examples @natBME https://t.co/zG8uVIxrJf https://t.co/hQ0tjiapIJ
Real‑world data (RWD) is reshaping natural history studies by delivering longitudinal, patient‑level information that was previously only obtainable through costly prospective trials. Advances in artificial intelligence and natural language processing now allow rapid extraction of structured data from unstructured sources...
A new mechanism for improved cognitive function from exercise in the Alzheimer's disease model Skeletal muscle EC vesicles interact with the brain and rev up microglia function https://t.co/eZu14YVasY
Safety concerns spur Aardvark to halt key Prader-Willi drug trial https://t.co/PWkOuDMIvo $AARD - 52% $SLNO
The U.S. FDA granted accelerated approval to Ascendis’ Yuviwel (navepegritide) for increasing linear growth in children aged two years and older with achondroplasia and open epiphyses. Approval rests on three placebo‑controlled trials and up to three years of open‑label extension...
Stanley Plotkin, known as the “godfather of vaccines,” in an interview with @HelenBranswell: “All I can say is that I’m beginning to regret having lived so long — because we’re going downhill.” https://t.co/A2zcoP2Ghd
New @NatureMedicine A randomized trial of antibody vs activin type II receptors with or without semaglutide (Ozempic). The antibody, bimagrumab, promotes muscle growth. Marked weight loss with the combination https://t.co/XUK93GTAmA
Cell therapy manufacturing is vulnerable to contamination because living cells cannot be terminally sterilized. Regulatory bodies such as EU GMP Annex 1 and the FDA now require risk‑based contamination control strategies that prioritize closed, sterile, and automated processes. Closing open steps...
Stanley Plotkin had a major hand in the development of a number of vaccines in use today; he designed the rubella vaccine. He remembers the world before widespread use of vaccines & knows what's coming as vaccine policy is rewritten...
2March: How did @generate_biomed CEO @mike_nally describe his whirlwind of emotions as his company went public? Read what he and CFO Jason Silvers, MD, JD said about $GENB plus updates on $CASIF, $NVAX, $PMVP & $SRPT in my latest StockWatch...
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending Dupixent (dupilumab) for paediatric chronic spontaneous urticaria (CSU) in children aged 2‑11. The recommendation is based on robust Phase III data from the LIBERTY‑CUPID...
Someday we'll be able to track our immune system like we do glucose Today @NatBME https://t.co/HFcFiUeyFg Previously @ScienceMagazine https://t.co/EcgBdwGxcd https://t.co/SpoZ5bLtjx
Who's heard of Flare Therapeutics? #GU26 late-breaker shows promise in hitting "undruggable" PPAR-γ target, with a few major caveats. Via @ApexOnco -> https://t.co/1GepvywtL0
President Trump has pressed European nations to raise their drug spending, arguing that higher foreign prices would allow the United States to lower its own prescription costs. While no clear price hikes have materialized yet, the demand has sparked uncertainty...
“Nobody wants to feel cornered:" Europe braces for the fallout of Trump's drug pricing plan https://t.co/HZjZH0mdCU
$NTLA drug is off hold but now being restricted by the FDA to the least severe/sick patients, which would logically be those least likely to want/need an experimental gene editing therapy. Existing options will dominate. Target/disease selection is a...
Opus Genetics presented early Phase I/II data for its OPGx‑BEST1 gene therapy targeting best vitelliform macular dystrophy and autosomal‑recessive bestrophinopathy. In a sentinel 63‑year‑old participant, the treatment was well tolerated and delivered a 12‑letter gain in best‑corrected visual acuity after...
$MRK claims a survival benefit in ovarian cancer irrespective of PD-L1 status, but FDA & EMA say BS. Via @ApexOnco -> https://t.co/Cld1O7Uuvp #ESGO26
BioMarin’s enzyme substitution therapy Palynziq received FDA approval for use in adolescents aged 12 and older with phenylketonuria (PKU). The decision was based on Phase III PEGASUS trial results showing 44.4% of participants lowered blood phenylalanine below guideline levels, with a...
Eli Lilly announced a $3.5 billion life‑sciences manufacturing complex in Lehigh Valley, Pennsylvania, slated to open in 2031. The facility will produce the company’s next generation of weight‑loss drugs and employ roughly 850 engineers, scientists and technicians. The deal, the largest life‑sciences...
The article profiles 18 UK biotech firms spanning oncology, immunology, regenerative medicine and AI‑driven drug design, highlighting recent milestones such as Amphista’s $30 million upfront deal with BMS, Autolus’ FDA and EU approvals for obe‑cel, and Isomorphic Labs’ $600 million raise to...
X4 Pharmaceuticals’ Xolremdi (mavorixafor) received a positive opinion from the European CHMP, recommending approval under exceptional circumstances with an EC decision expected in Q2 2026. The recommendation is based on the global Phase III 4WHIM trial involving 31 patients aged 12 and...
European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending conditional approval of Ipsen’s Ojemda (tovorafenib) as monotherapy for pediatric low‑grade glioma with BRAF fusions, rearrangements or V600 mutations. The recommendation is based on...
The FDA will render decisions this March on five high‑profile drug applications, including two delayed from last year. Bristol Myers Squibb seeks a psoriatic arthritis label for Sotyktu, backed by Phase 3 data showing a 54.2% ACR20 response. Aldeyra, Rhythm, GSK...
Altor is hosting a free webinar on March 12, 2026 to teach cold‑chain professionals best practices in thermal testing. The session will cover how nuanced variables affect test outcomes and which questions to ask to validate designs. Speakers include Altor’s Director of...
Quanterix (QTRX) reported FY 2025 revenue of $16.1 million, down $11 million from the prior year, driven by steep declines in collaboration and contract‑manufacturing income. R&D spending fell modestly to $140.7 million, while SG&A rose to $65.5 million due to higher professional fees for...
Xeris Biopharma reported record Q3 2025 product revenue of $74.1 million, a 40% year‑over‑year increase driven primarily by Recorlev’s 109% revenue jump. The company posted its first quarterly net income and an adjusted EBITDA of $17.4 million, reflecting strong operating leverage and...
UniQure reported a regulatory setback as the FDA now deems the Phase 1/2 external‑control data for AMT‑130 insufficient for a Biologics License Application, creating uncertainty around the U.S. filing timeline. Despite this, the high‑dose AMT‑130 achieved a statistically significant 75% slowdown...
Nuvation Bio reported $7.7 million net product revenue for Iptrozi in Q3 2025, driven by 204 new patient starts and payer coverage expanding to over 80% of U.S. lives. Clinical data showed an 89% overall response rate and a median...
UroGen Pharma reported over $127 million in cash at the end of Q4 2025, affirming sufficient liquidity to reach profitability if current plans hold. The company highlighted steady month‑over‑month growth in Zasturi enrollment, with community treatment share now around 35‑40% and institutional...
According to ITOA, NAD declines with age, causing epigenetic changes & disease susceptibility In 2010, we showed NAD-dependent enzyme, SIRT3, protects the heart 🐁 New study says NAD precursor NMN protects the heart via SIRT3 in mice fed a Western diet https://www.sciencedirect.com/science/article/pii/S0022316626000842
OK. Here it is. Over the last year, the @OctantBio and OpenADMET team have been hard at work developing scalable, quantitative, data-rich, and low-cost methods for assessing CYP reactivity and inhibition. The interplay between building a data engine and building...
LogiPharma 2026 will debut in Vienna’s Austria Center, moving from France after four years. Under new event director Ben Sharples, the conference introduces interactive panels, Oxford‑style debates and hands‑on training through the LogiPharma Academy. The program splits into two interconnected...
Ginkgo reported full year 2025 revenue of $170M at the end of last week. When it went public at a $15B valuation in 2021, they guided to investors that they’d be adding $7.6B in new business PER YEAR in 2025...
Researchers at the Innovation Center of NanoMedicine unveiled PL‑display, a cell‑free platform that immobilizes individual peptides on magnetic beads for rapid screening. The method delivers over ten‑fold efficiency gains versus traditional cell‑based displays and can operate under high‑temperature, high‑salt, or...
