Know What's Happening in Pharma
Know What's Happening in Pharma
FDA approves KRESLADI, first CIRMâfunded geneâediting therapy for LADâI
The FDA has granted approval to KRESLADI, a geneâediting therapy from Rocket Pharmaceuticals that treats severe leukocyte adhesion deficiencyâI in children without a matching boneâmarrow donor. This marks the first FDAâapproved product directly funded by Californiaâs Institute for Regenerative Medicine (CIRM), highlighting the agencyâs impact on translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma from GHO Capital
In this episode, the hosts dissect why investors misunderstand Hims & Hers' (HEMS) gross margin potential, emphasizing that as a compounder without its own API manufacturing, HEMS cannot achieve margins higher than drug manufacturers. They illustrate how HEMS' margin trajectory is decliningâfrom 73.5% to 71.9%âdue to a product mix shift toward higherâaverageâorder-value items with lower margins, such as sexualâhealth drugs like Sildenafil. The discussion highlights the importance of supplyâchain ownership in margin economics and uses concrete examples to explain the margin dynamics at play.
This weekâs coverage juxtaposes longevity hype with emerging science, highlighting David Sinclairâs bold claim that aging could be reversed within 10â20 years, a new Nature paper confirming adult hippocampal neurogenesis, and research showing men lose the Y chromosome as they...
Researchers at Adelaide University conducted the first inâvivo study of salcaprozate sodium (SNAC), the absorption enhancer used in oral semaglutide tablets such as Ozempic and Wegovy. Over a 21âday period in rats, repeated SNAC exposure reduced fibreâdegrading gut bacteria, lowered...
$MRK Welireg could be about to expand its reach at last. #GU26 data via @ApexOnco with relevance to $RCUS -> https://t.co/k59284INdK
Looks like Miami is getting ready for biotech week (the week after next). Note: BiotechTV will be broadcasting from the Barclays conference March 10 & 11. https://t.co/ToZH0EgOVz
Epic Systems announced its first pharmaceutical customer, EliâŻLilly, for the newly launched Epic Discovery platform. The deal marks the inaugural sale of a Health Grid product to a lifeâsciences firm, allowing Lilly to streamline clinical trial creation, site identification, activation,...
A phaseâ2 trial of relmacabtagene autoleucel (relmaâcel), a CD19âdirected CARâT therapy, enrolled 59 Chinese patients with relapsed/refractory mantleâcell lymphoma after BTKâinhibitor failure. The study reported a 71.2% overall response rate and a 59.3% complete response rate, with median time to...
But note IL11 knockout mice are infertile (females) and have low body weight and some osteoporosis. Still interesting angle though.
How is it that a paper that claims to show how to do target trial emulation does not address confounding by indication and its ramifications for data collection? https://t.co/dzGZZdpBoc #Statistics
A new analysis quantifies the operational and financial benefits of using Genmabâs epcoritamab, a dualâindication bispecific antibody, for both relapsed/refractory diffuse large Bâcell lymphoma (DLBCL) and follicular lymphoma (FL). In a communityâpractice model of 100 patients, the study projects 3,110...
Some very good news about engineering our cells vs cancer (accelerating the CAR). A short thread 1. For background, a new 5â review on cancer immunotherapy @Cancer_Cell https://t.co/1Qz2rCs8I5 https://t.co/sJ6BwaXyjk
A single-cell atlas characterizes dysregulation of the bone marrow immune microenvironment associated with outcomes in multiple myeloma https://t.co/5t0M2eX0fB https://t.co/p3kv8LEzrC
A phaseâ2b trial enrolling 94 adults with chronic hand eczema (CHE) without etiologic preâselection uncovered a mixed immune signature spanning typeâŻ2, typeâŻ3 and typeâŻ1 pathways. Dupilumab, an ILâ4RÎą antagonist, delivered a 59.8% mean improvement in modified Total Lesion Symptom Score...
This weekâs Endpoints Weekly highlighted a surge of GLPâ1 developments, including new trial data and expanded indications that reinforce the classâs dominance in obesity and diabetes treatment. The newsletter also released its annual TopâŻ100 venture investors list, showing a notable...
The EMAâs Committee for Medicinal Products for Human Use (CHMP) recommended six new medicines, including three orphan drugs already approved by the FDAâOjemda for pediatric lowâgrade glioma, Palsonify for acromegaly, and Xolremdi for WHIM syndrome. The agency also gave a...
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![[Review] Autosomal Dominant Polycystic Kidney Disease](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/e5c772f073be33a83118f46a1ac91f13.webp)
Autosomal dominant polycystic kidney disease (ADPKD) remains the most common hereditary cause of chronic kidney disease, imposing substantial morbidity, mortality, and healthcare costs worldwide. Recent advances in molecular genetics and highâresolution imaging have sharpened diagnostic criteria and enabled more accurate...
![[Comment] Safeguarding Genomic Integrity in Pluripotent Stem-Cell Therapies](/cdn-cgi/image/width=1200,quality=75,format=auto,fit=cover/https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/fba52b2aab9c591bf0e059f57acce9ea.webp)
Human pluripotent stem cells (hPSCs) are emerging as a transformative platform for Parkinsonâs disease, with recent phase I/II trials showing successful engraftment of hESCâ and iPSCâderived dopaminergic neurons. Yet extensive data reveal that cultured hPSCs frequently acquire recurrent genetic lesionsâmost...
Minnesotaâs Department of Health reports that hospitals and clinics in the state earned at least $1.34âŻbillion in 2024 from the 340B drug discount program. Participants received $3.045âŻbillion in discounted medicines but paid $1.53âŻbillion plus $165âŻmillion in administration fees. The largest...
Ultrasonic spray coating is emerging as a core engineering discipline for drugâeluting implants, with parameters such as drugâtoâpolymer ratio, nitrogen carrierâgas flow, nozzle height, and spray power dictating coating thickness, profile, and elution behavior. The article explains how variations in...
Researchers at Cornellâs Weill Institute introduced QUASARRâseq, a highâthroughput assay that measures promoter and enhancer activity simultaneously. The study found that most human regulatory elements can function as both promoters and enhancers, following a unified regulatory logic. A bidirectional feedback...
Kencoâs viceâpresident of life sciences, Tim McClatchy, detailed how the firm is hardening cybersecurity across its manufacturerâtoâhome delivery network while deploying AI to streamline labor planning and route optimization. He explained the specific encryption and verification steps used at each...
The iShares U.S. Pharmaceuticals ETF (IHE) is anchored by Johnson & Johnson and EliâŻLilly, whose earnings per share have nearly doubled in the past year. IHEâs aggregate oneâyear EPS growth estimate is an impressive 19.07% while trading at a modest...
President Trumpâs "mostâfavored nation" (MFN) drug pricing agreements, touted as a safeguard against excessive prescription costs, have been revealed to run for three years for several participants. SEC filings show that 16 pharmaceutical firms have entered these deals, each with...
Researchers have engineered sizeâshifting lipid nanoparticles that grow from ~100âŻnm to >300âŻnm after intraperitoneal injection, exploiting a capsuleâfilter mechanism that blocks entry into the liver and spleen while allowing passage to the pancreas. The enlarged particles deliver mRNA payloadsâincluding CRISPRâCas9...
This morning @generate_biomed celebrated its IPO on the @NasdaqExchange. We founded Generate at @FlagshipPioneer in 2018, asking: What if we could generate novel protein therapeutics using generative AI tools â without relying on traditional trial-and-error discovery? Todayâs IPO is a major...
Eli Lilly unveiled LillyPod, a DGX SuperPOD built with 1,016 NVIDIA Blackwell Ultra GPUs delivering more than 9,000 petaflops of AI performance. The system powers genomics, proteinâdiffusion, smallâmolecule graph neural networks and foundation models, allowing billions of inâsilico experiments. Constructed...
The FDA approved Hernexeos through the national priority voucher program, a mechanism that fastâtracks drugs addressing unmet medical needs. Industry leaders are evaluating directâtoâconsumer (DTC) models to broaden access to HIV prevention tools, leveraging digital and telehealth channels. Additionally, the...
An intriguing tale of three cities. How do the PD1xCTLA4 bispecific agents lorigerlimab, volrustomig, and cadonilimab stack up when it comes to design and performance? Some useful and pointed lessons here for biotech execs: https://t.co/xaSW15Fvdl https://t.co/dhXbiI3N6v
Three bispecific antibodies targeting PDâ1 and CTLAâ4âlorigerlimab, volrustomig, and cadonilimabâillustrate divergent engineering strategies. Lorig erlimab relies on a knobâintoâhole heterodimer, volrustomig adopts a common light chain format, and cadonilimab incorporates Fcâsilencing mutations. Their distinct designs have produced markedly different safety...
Bayer's HYRNUO (sevabertinib) received accelerated FDA approval on NovemberâŻ19,âŻ2025 for adult patients with HER2âmutated, nonâsquamous nonâsmall cell lung cancer who have progressed after prior therapy. The approval is based on the singleâarm SOHOâ01 trial, which enrolled 122 patients across 78...
Atrium spins out of Avidity, aiming to target rare heart diseases with RNA https://t.co/xLp2oQDHGy $RNA $NVS #startups
A roundâup of leading experts highlights how cellular reprogramming has moved from a laboratory curiosity to a nearâclinical antiâaging platform. Researchers describe partial, epigeneticâfocused approaches that can rejuvenate cells without erasing identity, and four biotech firms outline their distinct delivery...
âThe era of de novo proteins is coming a lot faster than probably people recognize,â Generate's CEO Mike Nally tells me. âI think within the next year or two youâll start seeing de novo designs enter the clinic.â https://t.co/u3WQcDD3jL
The Inflation Reduction Act reshapes Medicare PartâŻD by granting CMS authority to negotiate prices for highâcost drugs and instituting a $2,100 annual outâofâpocket cap for beneficiaries starting this year. The first ten drugs, including Ozempic and Wegovy, will have negotiated...
The Block layoff news was a big deal yesterday. Iâm sorry to say that ML driven automated labs are going to do the same to lab jobs in life sciences that AI is starting to do to other industries.
Dan Chancellor of Norstella explains that the looming patent cliff and persistent growth gaps are forcing pharma companies to rely heavily on mergers and acquisitions. 2025 saw a record $220âŻbillion in deal value, driven by several megaâtransactions and numerous boltâon...
AI is helping to make the diagnosis of rare diseases. A new multicenter study in Japan showed smartphone AI diagnosis of acromegaly via hands was better than experienced endocrinologists https://t.co/qBiazetToV https://t.co/dSZpJ2us84
Elizabeth Cairns' piece on $Novo is reminiscent of Bristol Myers' strategic fail against Merck and Keytruda. They were focused on the wrong things, made bad trial design decisions and got stuck in the mud of their own making. Everyone should...
The episode covers three main business stories: Tamarin Bioâs $13.6M Series A to build an AI modelâcoordination platform that makes tools like AlphaFold accessible to wetâlab biologists; the FDAâs reversal on Modernaâs mRNAâ1010 seasonal flu vaccine, spurring a modest stock...
AMSBIO (Oxford, UK) now provides readyâtoâuse lentiviral particles with titers exceeding 1Ă10⡠IFUâŻ/âŻmL, eliminating the need for inâhouse virus production. The thirdâgeneration selfâinactivating vectors are supplied in researchâgrade and GMPâgrade formats, requiring only BSLâ2 containment. Highâtiter, purified particles enable efficient...
âYou canât stand there after the fact and blame the open-label design,â he said. âYou designed it. You designed a noninferiority margin. You picked the patients. And then the trial doesnât work.â https://t.co/acS5i5s5NU
A year-long trastuzumab emtansine (TâDM1) regimen showed 98% threeâyear invasiveâdiseaseâfree survival in earlyâstage HER2âpositive breast cancer, comparable to standard paclitaxelâplusâtrastuzumab. The ATEMPT trial of nearly 500 patients found similar overall survival but markedly lower neuropathy and hair loss with TâDM1,...
The FDA granted accelerated approval to Boehringer Ingelheimâs oral HER2 kinase inhibitor Hernexeos six weeks after the company filed its application, marking the second approval under the new Commissionerâs National Priority Voucher (CNPV) program. The decision expands Hernexeos use to...
Todayâs brief highlights four preâclinical advances: Paratus Sciences unveiled PSâ1001, a panâinflammasome blocker targeting ILâ1β and ILâ18 for hidradenitis suppurativa; researchers identified the SCAN circuit as a core pathway disrupted in Parkinsonâs disease; RXâ10616 demonstrated enhanced radiotherapy response in headâandâneck...
Prospect Therapeutics announced the identification of a new series of smallâmolecule inhibitors targeting Janus kinase 1 (JAK1) and tyrosine kinase 2 (TYK2). The lead compounds demonstrate nanomolar potency and selectivity in biochemical assays and effectively suppress cytokine signaling in cellular...
The Consolidated Appropriations Act of 2026 bans pharmacy benefit managers (PBMs) from receiving compensation tied to drug list prices and mandates 100% rebate passâthrough to plan sponsors by August 2028. The law also allocates roughly $190âŻmillion to the Centers for...
1/@TesseraTx has announced that the U.S. FDA has granted Fast Track and Orphan Drug designations to TSRA-196 - its leading In Vivo Gene Editing program which is being jointly developed with $REGN, for the treatment of patients with alpha-1 antitrypsin deficiency...
The FDAâs Drug Trials Snapshots program publishes concise, consumerâfocused summaries of the demographic makeup and outcomes of pivotal clinical trials for newly approved drugs. Launched in 2015, the tool covers only New Molecular Entities and original biologics approved after that...
Ionis Pharmaceuticals received FDA acceptance of its supplemental NDA for olezarsen and a priorityâreview designation, with a PDUFA action date of June 30, 2026. The decision is backed by two PhaseâŻIII CORE studiesâCORE (n=617) and CORE2 (n=446)âthat evaluated onceâmonthly subcutaneous...
