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
Scientists at Baylor College of Medicine identified a brain pathway that enables metformin to lower blood sugar, a discovery that could expand the drug’s use beyond diabetes and fuel new longevity protocols among biohackers.
Cellular senescence has become a focal point for longevity medicine, prompting a surge of senolytic and senomorphic drug development. Pioneering studies showed that clearing senescent cells can extend healthspan, leading biotech firms like Rubedo, SENISCA, Deciduous Therapeutics, and Arda Therapeutics...
A research team has created magnetic silk‑iron nanoparticles that can be steered with external magnetic fields to deliver therapeutics to otherwise inaccessible disease locations. The nanoplatform merges biocompatible silk fibroin with iron oxide, enabling magnetic control while maintaining safety, a...
The World Health Organization announced new policy guidance aimed at simplifying tuberculosis detection tests, signaling a major push for more accessible diagnostics. While the guidance outlines a framework for low‑cost, point‑of‑care testing, specific technical details were not disclosed in the...
One large psilocybin dose beat nicotine patches by 6x odds for smoking cessation. 82 otherwise-healthy cigarette smokers, 42 received a single high-dose 30mg/70kg psilocybin session, and 40 initiated an 8- to 10-week course of nicotine patch treatment. At 6 months; participants were 6x...
Kodiak Sciences announced that its experimental eye drug Zenkuda (tarcocimab tedromer) achieved positive topline results in the GLow2 Phase 3 trial for diabetic retinopathy, outperforming sham treatment. The study met its primary endpoint, delivering a statistically significant gain in visual acuity...
Cryopreservation remains a linchpin for cell and gene therapy (CGT) manufacturing, yet frozen material is not inherently stable. Real‑world operations introduce transient warming events (TWEs) when products are moved, accessed, or shipped, silently degrading viability and potency. Traditional reliance on...
Dr. Emanuela Offidani, Tris Pharma’s Medical Director of Digital Health Strategy, highlighted that ADHD is increasingly recognized as a lifelong condition that often goes undiagnosed in women because symptoms are less overt. She explained that Tris Pharma’s proprietary LiquiXR delivery...
I have a story today about the quest to turn eggs into low-cost factories for medicines. Gift link: https://nyti.ms/4bzSabB
My article in today's issue of the Journal of the American Medical Association Health Forum, on FDA's new rare disease guidance, and how the agency can build on these policy steps to promote innovation for inherited disorders, authored with Maarika...
The Pistoia Alliance has published a peer‑reviewed best‑practice framework guiding ethical use of social‑media listening in drug development. The framework, co‑authored by experts from Bayer, Roche, Boehringer Ingelheim, Chiesi and Semalytix, outlines standards for data anonymization, bias mitigation, and AI‑driven...
FDA clears Denali drug in ‘clear step’ for rare disease biotechs https://t.co/x9yL4nyPaj by Kristin Jensen $DNLI #biotech
Of all the IO companies I've met and covered in the past ~15 years I'm struggling to think of one that's been more disappointing vs its initial promise than $IPHYF. Will it even last beyond Q3? Via @ByMadeleineA -> https://t.co/DepdZfDBdY
Corcept Therapeutics saw its shares climb almost 20% after the U.S. FDA approved its lead drug Lifyorli in combination with nab‑paclitaxel for platinum‑resistant ovarian, fallopian tube and primary peritoneal cancer. The approval, based on a 381‑patient trial, validates the company's...
This week's Biotech Scorecard: Allogene $ALLO: A preview of its April interim analysis. Very interesting (to me, at least) There just might be a path forward for off-the-shelf CAR-T therapy in B-cell lymphoma. Frontline consolidation. Read all about it: https://t.co/tcPFelUH3C
Precigen (PGEN) reported fiscal Q4 2025 revenue of $9.7 million, up 149% year‑over‑year, and projected first‑quarter 2026 sales to exceed $18 million. The surge stems from the rapid commercial rollout of its FDA‑approved gene‑therapy Papzimius and broader payer coverage across the United...
Biotech start‑up Neion Bio is pioneering a method to turn chicken embryos into miniature drug factories. Scientists micro‑inject genetic constructs into three‑day‑old embryos, reprogramming the developing bird to synthesize pharmaceutical compounds within the egg. The approach promises faster, cheaper production...
Novocure disclosed topline Phase‑2 PANOVA‑4 data showing that Tumor‑Treating Fields (TTFields) combined with Tecentriq and gemcitabine/nab‑paclitaxel achieved a 74.4% disease‑control rate in metastatic pancreatic ductal adenocarcinoma (mPDAC). The trial, enrolling 78 patients, outperformed the historical MPACT control arm’s 48% DCR. Secondary...
Pinnacle Medicines, a two‑year‑old biotech focused on oral peptide therapeutics, secured $89 million from a mix of U.S. and Chinese investors. The financing will fund its platform that converts injectable peptides into pill form, building on the recent success of Novo...
In this episode, Bruno Quinney highlights three breakthrough studies: Edinburgh researchers engineered E. coli to convert PET plastic waste into the Parkinson's drug L‑DOPA, offering a sustainable route to a vital medication; scientists identified the enzyme DHX8 as a key...
Otsuka Pharmaceutical presented Phase III VISIONARY trial data for Voyxact (sibeprenlimab‑szsi) in IgA nephropathy patients at risk of progression. At 48 weeks, 82.5% of patients receiving 400 mg subcutaneous Voyxact achieved negative microscopic hematuria versus 52.6% on placebo, with median time to...
Scholar Rock's anti-TGFb1 antibody clinical study with early data in oncology indications... minimal AEs and signs of durable efficacy in checkpoint-refractory patients. $SRRK https://t.co/8xsN4wtbMV
Exazym®'s BOLD amplification technology boosts the sensitivity of a human cardiac troponin I sandwich ELISA by 180‑fold, lowering the detection limit to 0.07 pg/mL. The webinar presented by Cavidi’s Peter Stenlund shows how the method integrates into standard ELISA workflows with...
Autoimmune drug development is moving upstream, targeting pathogenic immune cells rather than single cytokines. AnaptysBio’s Chief Medical Officer, Paul Lizzul, highlighted the company’s cell‑selective immunomodulation strategy, including CD122 antagonism that modulates both CD4 helper and CD8 cytotoxic T cells. Early‑phase...
The U.S. Senate has advanced legislation that would fund expanded clinical trials of psychedelic therapies for veterans suffering from PTSD, depression and traumatic brain injury. The bill, backed by a bipartisan coalition, marks the first federal effort to systematically study...
Eli Lilly announced top‑line results from the Phase III TRANSCEND‑T2D‑1 trial, showing its investigational triple‑hormone agonist retatrutide reduced HbA1c by up to 2 percentage points and produced an average 16.8 % weight loss over 40 weeks. The data position retatrutide as a potential...
Atossa Therapeutics released its fourth‑quarter 2025 earnings and announced that the FDA granted Rare Pediatric Disease and Orphan Drug designations to its lead candidate (Z)-endoxifen for Duchenne muscular dystrophy. The company said the designations accelerate its rare‑disease strategy while it...
Here's prescribing info for $CORT Lifyorli -> https://t.co/i46Whl5lX6 Why so different from the experience in Cushing's? https://t.co/EqvbCApqHn
Dyadic (DYAI) posted Q4 2025 revenue of $3.1 million, a modest rise driven by COO Joseph P. Hazelton’s operational focus. The earnings call highlighted new product launches, global distribution deals and a growing reliance on grant‑linked R&D, offsetting a drop in...
Corcept Therapeutics saw its shares climb almost 20% after the U.S. FDA gave the green light to Lifyorli in combination with nab‑paclitaxel for platinum‑resistant ovarian, fallopian tube and primary peritoneal cancers. The approval, based on a 381‑patient Rosella trial, validates...
Biogen has signed an exclusive license with South Korean biotech Alteogen for ALT‑B4, a recombinant hyaluronidase enzyme that facilitates subcutaneous administration of biologics traditionally given intravenously. The agreement provides Alteogen with $20 million upfront, a $10 million payment upon initiation of a...
Jiangsu Aidea Pharmaceutical, a Shanghai‑Star Market listed firm specializing in HIV/AIDS therapies, is preparing a Hong Kong IPO to fund its expansion into the United States and Europe. The company projects the global AIDS‑treatment market to exceed $1.45 billion by 2027...
Dr. Joseph Sansone has filed Senator Ron Johnson’s investigative subcommittee letter as supplemental authority in his appellate lawsuit against Florida Governor Ron DeSantis and Attorney General James Uthmeier, alleging that mRNA COVID‑19 injections function as biological weapons. The filing cites...
Lenz Therapeutics' shares slid 11.2% after the biotech reported a Q4 loss of $1.16 per share on $1.6 million in revenue, well below analyst expectations. The miss stemmed from weaker-than-anticipated uptake of its newly launched VIZZ eye‑drop and a surge in...
Ascentage Pharma reported unaudited 2025 results, showing product sales surged 90% to $82.1 million while total revenue fell 41.5% after a $92.9 million drop in intellectual‑property income. Olverembatinib generated $62.2 million, up 81% YoY, and the newly launched Lisaftoclax contributed $10.1 million in its...
Lawrence Tallon, the MHRA chief, is set to oversee the launch of the Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2025 on April 28, 2026. The amendment represents the most extensive overhaul of U.K. clinical‑trial rules in two decades, targeting faster approval,...
Alpha-amyrin, a molecule found in passion fruit, has demonstrated the ability to protect brain mitochondria and reduce memory loss in Alzheimer's mouse models, suggesting potential for future therapeutic development. neuroscience
Proud of the work of our Seaport team & collaborators published today in @ScienceTM. This ~13 page peer reviewed paper is the first comprehensive data disclosure of our GlyphAllo™ program from discovery through initial human proof‑of‑concept.
The FDA’s Regulatory Education for Industry (REdI) Annual Conference will take place May 19‑20, 2026, offering both virtual and in‑person sessions at the White Oak Campus in Maryland. The two‑day program features three dedicated tracks—drugs, devices, and biologics—allowing participants to...
Here it is again. Amazing how researchers could get away with this back then. Regular systematic bias of excluding TAD nonresponders. Also note another thing inconsistent with core psychedelic trial period i.e., that Ps were allowed to continue psychotherapy outside...
Denali Therapeutics received accelerated FDA approval for Avlayon (tividenofusp alfa‑eknm), the first enzyme replacement therapy designed to cross the blood‑brain barrier for Hunter syndrome. The approval is based on a phase 1/2 trial of 47 boys up to 18 years, showing...
The FDA’s Center for Drug Evaluation and Research released its FY 2025 GDUFA Science & Research Report, detailing more than 50 funded projects across eight priority scientific initiatives. The program targets bioequivalence, manufacturing standards, and advanced analytical methods to streamline abbreviated...
Under the Hatch‑Waxman law, generic manufacturers must file a Paragraph IV certification asserting that a listed patent is invalid, unenforceable, or not infringed to obtain FDA approval. The first substantially complete ANDA with such a certification secures a 180‑day exclusivity period,...
Researchers at Longeveron reported that a single infusion of laromestrocel, a mesenchymal stem‑cell therapy derived from donors aged 14‑18, significantly boosted mobility in frail seniors. In a double‑blind trial of about 150 participants aged 70‑85, the highest dose (200 million cells)...
The FDA announced new guidance that expands flexibility in chemistry, manufacturing, and controls (CMC) for cell and gene therapies (CGTs) across their development lifecycle. Sponsors can now defer full cGMP compliance until later trial phases and make iterative manufacturing changes...
The FDA granted accelerated approval to Avlayah (tividenofusp alfa‑eknm), a weekly IV infusion, for treating neurologic manifestations of Hunter syndrome in pediatric patients weighing at least 5 kg. The approval is based on a phase 1/2 trial that demonstrated a 91% average...
The Kansas House of Representatives approved a bill that would tighten regulations on pharmacy benefit managers (PBMs) to help lower prescription‑drug prices. The measure now heads to the Senate, where it could reshape how insurers negotiate drug rebates and pricing.
