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
BioNTech’s Q4 2024 earnings call highlighted a strategic pivot toward oncology, emphasizing the advancement of its bispecific antibody BNT327 and the recent acquisition of Biotheus to secure Chinese manufacturing and development capabilities. The company reported positive Phase 2 data for its off‑the‑shelf FixVac melanoma vaccine and robust immune responses from the personalized mRNA therapy autogene cevumeran, though efficacy signals remain modest. BioNTech also confirmed it retained a leading share of the global COVID‑19 vaccine market while outlining a data‑rich 2025 pipeline focused on adjuvant settings and combination regimens.
Indiana University researchers linked clonal hematopoiesis of indeterminate potential (CHIP) to heightened severity of inflammatory bowel disease. Analysis of UK Biobank and All of Us data showed women with DNMT3A mutations and younger individuals with TET2 mutations face higher Crohn’s...
An international team genetically deleted the ENA1 gene from Saccharomyces boulardii, a common probiotic yeast. In immunosuppressed mice, the ENA1‑deficient strain showed no mortality, raising survival from 30‑40% to 100% compared with wild‑type isolates. The edit also reduced osmotic stress...
Senate Democrats have pressed major pharmaceutical companies to clarify the secretive most‑favored‑nation (MFN) pricing agreements they struck with the Trump administration in December. Lawmakers are questioning how those deals align with the Centers for Medicare & Medicaid Services’ new GENEROUS...
Roche’s oral selective estrogen receptor degrader (SERD) giredestrant failed to meet its primary endpoint in a late‑stage Phase III trial, but the data revealed meaningful activity in specific patient subgroups, particularly those with ESR1 mutations. The miss prompted Roche to...
A new AI-driven workflow predicts the outcomes of asymmetric cross-coupling reactions, enabling chemists to efficiently identify optimal molecular structures while significantly reducing laboratory time and costs. drugdiscovery
Vertex Pharmaceuticals announced that its experimental IgA nephropathy drug povetacicept met primary and key secondary endpoints in a Phase 3 trial, cutting urine protein by roughly 50% versus placebo after 36 weeks. The interim results also showed reductions in abnormal antibodies...
Researchers at Rice University and Baylor College of Medicine used a non‑editing CRISPR system to activate the PPARGC1A gene, boosting mitochondrial production in human cardiomyocytes. The technique safely increased cellular energy output, as shown by higher oxygen consumption in cell...
SAB Biotherapeutics announced full‑year 2025 results, highlighting the launch of its registrational Phase 2b SAFEGUARD trial for SAB‑142 and the completion of a $175 million oversubscribed private placement. Phase 1 data demonstrated a favorable safety profile, no serum sickness and low immunogenicity across...
Shield Therapeutics announced that the FDA has approved Accrufer, its ferric maltol oral iron formulation, for children ages 10 and older, making it the first prescription oral iron therapy for this age group. The approval expands the drug’s label beyond...
Statista projects that the U.S. pharmaceutical market will be led by ten blockbuster drugs in 2026, with Merck’s Keytruda topping the list at $12.7 billion in sales. Four oncology therapies—Keytruda, Opdivo, Imbruvica and Ibrance—are among the top ten, underscoring cancer’s continued...
What does it actually take to bring a biological product to market? For more than 20 years, Ingenza Ltd has helped teams avoid the most common failure points in synthetic biology. Freedom to operate issues. Low productivity. Fragile processes. Late CMC...
The U.S. Food and Drug Administration issued a warning‑letter close‑out to APS BioGroup, Inc. on April 5, 2018, confirming that the company’s corrective actions addressing the July 2017 warning letter were satisfactory. The agency stressed that this closure does not relieve APS BioGroup...
The FDA issued a Warning Letter to Dr. James Findling after a 2019 inspection revealed serious protocol violations in a clinical trial of an investigational drug. The investigator randomized two subjects who had exceeded permitted dose levels and failed to...
The FDA issued Warning Letter #588155 to Health Pharma USA LLC after a May‑June 2019 inspection uncovered multiple CGMP violations at its Rahway, New Jersey facility. Major deficiencies included a dysfunctional quality‑control unit that released products before review, incomplete batch...
Vinay Prasad has left the FDA’s Center for Biologics Evaluation and Research (CBER) for the second time in under a year, signaling a notable leadership turnover at a key regulatory hub for biologics, vaccines and gene therapies. In parallel, GlaxoSmithKline...
Medicines plus healthy habits create outcomes that neither could achieve alone. A new @StanfordMed study published in JAMA shows digital lifestyle nudges may catalyze a critical first step toward behavior change for patients on GLP-1 medications. “Achieving your best health involves a...
I've covered most of the big controversies the FDA has faced over the past 25 years: Vioxx, drug-coated stents, and Aduhelm all spring to mind. In all of these cases, the FDA's advisory panel system, in which the agency calls together...
Taisho Pharmaceutical’s vornorexant (TS‑142), marketed as Vorzzz®, received Japanese regulatory approval in August 2025 as a dual orexin‑1/2 receptor antagonist for insomnia. The drug distinguishes itself from existing DORAs through rapid absorption and a short elimination half‑life, aiming to minimize...
Would you like to hear an opinion on how the future of drug discovery will look like as foundation models become more capable in drug discovery tasks? Check out our dialogue and the first pilot with the foundation model genius @ramin_m_h...
Researchers led by Harvard chemist Christina Woo have mapped a previously unknown allosteric pocket on cereblon, the E3 ligase that underpins billions of dollars in cancer‑drug activity. The study shows that binding a small molecule to this hidden site can...
AbbVie announced top‑line Phase 1 multiple ascending‑dose data for its amylin analog ABBV‑295, showing 7.75‑9.79% weight loss after 12 weeks of treatment. The long‑acting compound was administered every other week then monthly, with a favorable tolerability profile and no serious adverse...
AbbVie and its partner Gubra released Phase 2 data on a long‑acting amylin analogue that produced significant weight loss in obese participants, with reductions approaching double‑digit percentages and a clean safety signal. The study highlighted dose‑responsive efficacy and tolerability, positioning the...
Re: Prasad, from this morning's @statnews Readout newsletter: FDA Commissioner Marty Makary is in Miami this week speaking to health care investors attending a slew of broker conferences, noted Mizuho health care strategist Jared Holz. That should make for some...
GlobalData Healthcare attended the World Evidence, Pricing and Access (EPA) congress in Amsterdam, where industry leaders debated mounting market instability. Sessions focused on the impact of the Most Favored Nation (MFN) policy, shifting US tariffs, rapid AI integration, and the...
Stable GLP‑1 receptor agonists such as Exendin‑4 and Ozempic improve beta‑cell viability by modulating gene expression. Researchers at the Salk Institute discovered that these drugs induce phosphorylation of Med14, a core subunit of the Mediator transcription complex. Phosphorylated Med14 enables...
Regeneron’s Chinese partner Hansoh announced that its dual GLP‑1/GIPR agonist olatorepatide achieved a 19% mean weight loss in a Phase 3 trial of 604 obese or overweight adults, meeting both co‑primary endpoints. The study reported lower gastrointestinal adverse events compared with...
Bristol Myers Squibb announced that its oral CELMoD candidate mezigdomide met primary endpoints in the Phase 3 SUCCESSOR‑2 trial for relapsed or refractory multiple myeloma. The open‑label study showed a statistically significant improvement in progression‑free survival compared with the current standard...
Medidata, a Dassault Systèmes brand, has partnered with eSource specialist CRIO to automate clinical data flow from site systems into the Medidata Platform. The plug‑and‑play integration now serves over 2,500 research sites in roughly 30 countries, delivering near‑100% data accuracy and...
The new Huberman Lab episode is out: Avoiding, Treating & Curing Cancer With the Immune System | Dr. Alex Marson 0:00 Alex Marson 2:21 Diseases & Current Biological Landscape; AI & Computational Tools 5:56 Immune System, Innate vs Adaptive Immune System 10:55 Thymus, T...
Actually he has it a bit backwards, those vaccinated with IPV can still acquire poliovirus and shed virus, but it prevents the virus from entering the bloodstream + protects almost 100% paralytic polio, just like the Covid vaccine protects 80-90%...
Bristol Myers Squibb reported positive interim Phase 3 results from the SUCCESSOR‑2 study, showing that oral mezigdomide combined with carfilzomib and dexamethasone (MeziKd) significantly extended progression‑free survival versus carfilzomib‑dexamethasone alone in relapsed or refractory multiple myeloma. The trial marks the first...
Vertex $VRTX says its drug successfully reduced marker of kidney disease in late-stage trial https://t.co/TbEkcHVYBB via @Jasonmmast
Bristol Myers claims success in study of another next-gen blood cancer drug https://t.co/fqPbZ2Szv8 by @gwendolynawu $BMY
Eli Lilly announced that the CMS CMMI BALANCE Model will cover its GLP‑1 obesity drugs—Zepbound, Mounjaro and, if approved, orforglipron—under Medicare Part D starting Jan 1 2027. After meeting the deductible, beneficiaries will pay a $50 monthly out‑of‑pocket cap, while pre‑deductible cost sharing is...
Check out this article I was apart of @MensHealthMag where I discuss currently approved FDA drugs that are potential geromedicines and our work on centenarians. https://t.co/e95XvnY9fb
New FDA draft guidance outlines important streamlining of path to getting low-cost biosimilars to the market - could reduce cost of pharmacokinetic studies and allow ex-U.S. comparitor products to be used for proving biosimilarity to U.S.-licensed drugs https://t.co/ErjNOga0qo
Shalabh Gupta, CEO of Unicycive Therapeutics, argues that biotech can benefit from tech‑driven design thinking, rapid prototyping, and disciplined capital allocation. He highlights how the company repurposed automotive battery‑shrinkage technology to create Oxylanthanum carbonate, a kidney‑disease candidate now under FDA...
UniQure leads genetic medicine biotech rally after news of Prasad’s exit https://t.co/zu1CtJfj29 by @realJacobBell $QURE $RGNX $ATRA $LXEO #GeneTherapy
Just published @NatureMedicine A daily multivitamin (MVM) slowed epigenetic aging in a randomized trial after 2 years; effect was small (~2 months) and not seen with cocoa extract supplement (vitamin was Centrum Silver) https://t.co/snOMNsTzW7 https://t.co/a6MhMuRhJb
Xenon Pharmaceuticals reported that its Phase 3 X‑TOLE2 trial of azetukalner, a novel Kv7 potassium channel opener, achieved a 53.2% reduction in focal onset seizures at the 25 mg dose, far exceeding expectations and representing the highest placebo‑adjusted efficacy recorded in a...
Not just replication, but triplication of the benefit of revving up cancer immunotherapy with FMT (Yes💩) https://t.co/rXyQdO74FJ
Chinese hamster ovary (CHO) cells remain the backbone of biopharmaceutical manufacturing, but traditional gene‑editing tools struggle with low knock‑in efficiency. Transposase‑based platforms such as Leap‑In and piggyBac now provide high‑efficiency, multi‑copy integration and can handle large DNA cargos up to...
Two terrific #FDA related pieces by @lizzylawrence.bsky.social & @matthewherper.bsky.social. Matt's is a look at lessons to be drawn from Vinay Prasad's chaotic tenure; Lizzy's delves into the FDA's turning away from adcomms. https://t.co/gC5GNTiPm1 & https://t.co/ygvylHi0uz
The Chemical Probes Portal’s >30‑fold selectivity rule, originally based on cell‑free assays, is challenged by a new open‑access J. Med. Chem. study comparing DiscoverX kinase panels with NanoBRET cellular profiling. Researchers found that most inhibitors appear less potent in living...
The Associated Press published a story alleging that FDA senior regulator Dr. Tracy Hoeg was pushing a petition from Dr. Adam Urato to add pregnancy‑risk warnings to antidepressants and that their personal relationship constituted a conflict of interest. The author,...
. @matthewherper is so damn reasonable. Smart, too. 5 lessons from Vinay Prasad’s turbulent tenure at the FDA https://t.co/R65vPj5ep5
Tacit Therapeutics, an RNA‑editing startup focused on neurological disorders, announced its launch backed by a $19 million financing round. The capital, led by Andreessen Horowitz and DCVC, will fund the development of ADAR‑based therapeutics targeting diseases such as ALS, Huntington’s, and...
The module on target validation walks through how phenotypic and target‑based strategies intersect in immune‑focused drug discovery. It highlights recent literature on TYK2 pseudokinase stabilization as a mechanism to block T‑cell signaling, and cites Icotrokinra and Deucravacitinib as successful examples....
The #VRBPAC committee is scheduled to meet Thursday to advise #FDA on #flu vaccine strain selection. VRBPAC is supposed to have at least 15 members. It currently has 7 voting members & no chair. It has met only twice since...
