
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
Two pre‑clinical studies propose active clearance of β‑amyloid as a new Alzheimer’s strategy. Researchers at Washington University engineered astrocytes with chimeric antigen receptors (CARs) that engulf plaques, while another team designed bispecific peptides that ferry amyloid into cells for lysosomal degradation. Both approaches demonstrated reduced plaque load and cognitive benefits in animal models, challenging the reliance on passive anti‑β‑amyloid antibodies.

EAA241 - Ph 2 RCT Dara-Bor-Dex vs Cy-Bor-Dex in the treatment of Newly Diagnosed Multiple Myeloma with Light Chain Cast Nephropathy (LCCN) [Study activated 8/11/25] @keruakous https://t.co/1NgvVZ3fTA #NCT07085728 #mmsm @eaonc https://t.co/IvK3UNeem9

#EAOnc EQUATE EAA181 Effective Quadruplet Utilization After Tx Evaluation: Ph3 RCT NDMM Not Intended for Early ASCT [Activated: 10/27/20] PI= @myelomaMD https://t.co/qD26HMx3J8 #NCT04566328 #mmMRD #mmsm @eaonc @mweissmdphd @VincentRK @LynneWagnerPhD Wei Snyder Kostakoglu @mtmdphd https://t.co/FLK0sJ0N1w
Senator Josh Hawley introduced a bill to immediately withdraw the FDA's safety approval for mifepristone, the primary abortion medication. The legislation follows recent Supreme Court and Trump‑era reviews of the drug and cites a controversial conservative study alleging serious adverse...

#EAonc EAA173 - Daratumumab to Enhance Therapeutic Effectiveness of Revlimid in Smoldering Myeloma (DETER-SMM) - PI: @nsc_natalie https://t.co/VtBMJUjI5X Activated: Apr 30, 2019 #mmsm @eaonc #NCT03937635 @VincentRK @mweissmdphd https://t.co/ikQKjHjscU

.@SWOG S2213 Ph3 RCT Dara-VC Induction Followed by ASCT or Dara-VCD Consolidation & Daratumumab Maintenance in Pts w/ Newly Diagnosed AL Amyloidosis [Activated: 12/1/23] https://t.co/OizUfJCc2c #mmsm #bmtsm https://t.co/FoT3i0xGcL

Three‑year follow‑up of the AGENT IDE trial shows the Agent paclitaxel‑coated balloon (DCB) outperforms uncoated balloon angioplasty in treating in‑stent restenosis (ISR), with target‑lesion failure (TLF) rates of 32.7% versus 40.9% (hazard ratio 0.72). The advantage is driven mainly by...

Genomic landscape of multiple myeloma and its precursor conditions [May 21, 2025] Jean-Baptiste Alberge et al. @IrenemGhobrial @NatureGenet https://t.co/DDWGRrpI4Y #mmsm #PrecisionMedicine #cagenome https://t.co/LY1MzFXSBN

Multimodal antigenic escape to GPRC5D-targeted T cell engagers in multiple myeloma [Jan 15, 2026] @hollyleeYJ et al. @NBahlis @NatureMedicine https://t.co/mz393mPMAq #mmsm #PrecisionMedicine #tcellrx THREAD: https://t.co/lNX9b7LsnR HT @AuclairDan https://t.co/2qih7LwP1c

Foghorn Therapeutics announced a financial update highlighting progress on its lead oncology candidate FHD-909, which remains on schedule in Phase 1 dose‑escalation for SMARCA4‑mutant NSCLC. The company also confirmed that its selective CBP and EP300 degrader programs are slated for...

Elutia Inc. reported its Q4 and full‑year 2025 results, highlighting the $88 million sale of its BioEnvelope business to Boston Scientific and a cash position of $36.4 million plus $8 million in escrow. The company submitted a 510(k) for the base biologic matrix...

Codexis reported FY 2025 revenue of $70.4 million, a 19% increase year‑over‑year, driven largely by a $37.8 million technology‑transfer agreement with Merck. Q4 revenue jumped 81% to $38.9 million, and product gross margin improved to 64% for the year. The company ended 2025 with...

The FDA announced a unified adverse‑event database that will eventually incorporate all of its existing reporting systems, including the vaccine‑side‑effect tracker jointly operated with the CDC. The new platform, called the Adverse Event Monitoring System, centralizes data from drugs, biologics,...

The FDA issued its fourth revision of draft biosimilar development guidance, allowing scientifically justified streamlining of pharmacokinetic (PK) studies and estimating up to a 50% cost reduction—about $20 million per program. The new guidance also expands the use of clinical data...
Researchers at BOKU University in Vienna have engineered a novel chromatography resin designed to capture secretory immunoglobulin A (IgA) at titers suitable for commercial manufacturing. The resin employs a reengineered bacterial surface ligand, analogous to Protein A, within a macropore...
Sen. Markey (D-Mass.) tells me outside the Capitol he doesn’t know who Vinay Prasad is and would need to be briefed by staff to respond to questions on the FDA
Researchers have demonstrated that mesenchymal stromal cells (MSCs) can actively internalize isolated, functional mitochondria through endocytic pathways. The study shows that the internalized organelles retain structural integrity and boost cellular proliferation, stress tolerance, and oxygen consumption. Chemical inhibition of endocytosis...
Consider this arc: peptides were around in niche communities, GLPs from Pharma then exploded, people realized they could take less or source them elsewhere, regulation enters & now gray & black market are going bye-bye & companies like HIMs &...
Researchers at Massachusetts General Hospital, in partnership with Full Circles Therapeutics, have introduced a circular single‑stranded DNA donor platform called INSTALL that enables kilobase‑scale gene insertion without triggering the cGAS immune sensor. The method combines a short double‑stranded DNA segment...
Researchers at Biohub identified a three‑amino‑acid cocktail—methionine, arginine and serine—that dramatically improves lipid nanoparticle (LNP) delivery of therapeutic mRNA and CRISPR components. Co‑administering the supplement boosted protein expression up to 20‑fold and raised gene‑editing rates from roughly 25% to nearly...
Johns Hopkins researchers have engineered a streamlined biodegradable polymeric nanoparticle that delivers mRNA to T cells, prompting them to generate CD19‑CAR receptors that target disease‑causing B cells. In mice, a single intravenous dose eliminated 95% of circulating B cells within...
If big pharma sold to big 3 distributors at net prices rather than list prices, patients would save on average 40pct on their specialty and brand medications. This isn't hard. @BernieSanders @HawleyMO @SenTedCruz @SenateGOP @SenWarren @AOC https://t.co/COdRaMJPgq
VIDO swiftly responded to COVID‑19 by designing a subunit vaccine candidate within days of the SARS‑CoV‑2 genome release, isolating the virus, and establishing animal models that enabled a Phase 1 human trial by early 2021, making it the first Canadian university...

Costco announced a partnership with digital health platform Sesame and fertility network IVI RMA North America to offer members dramatically reduced prices on fertility medications, with discounts of up to 80 percent. The program guides members through a digital intake,...

The FDA’s acting CDER director, Dr. Tracy Beth Hoeg, is reportedly moving to hire Dr. Adam Urato, a maternal‑fetal specialist who is petitioning the agency to add boxed warnings about alleged pregnancy risks of antidepressants. Urato’s claims rely on limited...
Veradermics closed a $294.8 million IPO, pricing 17 dollars per share and debuting on the NYSE under the ticker MANE. The capital will fund its late‑stage clinical program for VDPHL01, an extended‑release oral minoxidil aimed at androgenetic alopecia. The company has completed...
UCB’s Senior Vice President Dr. Kim Moran discusses the company’s rare disease pipeline, including therapies for mitochondrial TK2 deficiency, on Xtalks Life Science Podcast episode 246. Moran, who pioneered UCB’s Insights‑to‑Impact function, explains how patient‑derived data informs strategy, digital transformation,...

The KRAS niche has been a deluge of new selective and pan/multi inhibitors. Now there are combos to watch out for – we may be making progress. Here are insights on 7 of them: https://t.co/F9HmM8A4ud https://t.co/IqWw6n2m3k
Biogen reported Phase Ib data for its antisense oligonucleotide salanersen, showing substantial neurofilament light chain reductions and new motor milestones in children with spinal muscular atrophy (SMA) who had previously received onasemnogene abeparvovec. The study of 24 participants demonstrated a roughly...
Lonza has broadened its agreement with Genetix Biotherapeutics to increase manufacturing capacity for ZYNTEGLO, the sole FDA‑approved gene therapy for transfusion‑dependent beta‑thalassemia. Production will continue at Lonza’s Houston, Texas, dedicated cell‑and‑gene therapy site, with provisions to scale up for future...

Another potential GLP-1 drug benefit seen in people with diabetes and brain metastases for improved survival; a retrospective study that needs further assessment but intriguing https://t.co/nZm3xEYQMH https://t.co/V76S7o1MJb
The article surveys seven early‑stage programmes tackling KRAS, each proposing a distinct therapeutic angle. While many firms still chase the classic model of deeper, longer, or more selective pathway blockade, these initiatives span elegant biochemical tricks to counterintuitive concepts that...
The U.S. Food and Drug Administration approved leucovorin, a synthetic vitamin B9, solely for cerebral folate deficiency, a rare genetic disorder. Earlier this year, President Trump and HHS Secretary Robert Kennedy Jr. promoted the drug as an autism cure, prompting a surge in...

Eli Lilly’s chief executive David Ricks announced a strategic push to sustain the company’s recent $1 trillion revenue milestone by accelerating AI‑driven drug discovery and manufacturing. He highlighted a new partnership with Nvidia to embed generative‑AI models across the R&D pipeline, aiming...

Jay Bregman, CEO of Andel, unveiled a direct‑to‑employer medication platform that lets companies purchase drugs straight from manufacturers, starting with Eli Lilly’s Zepbound KwikPen. The model bypasses PBMs and insurers, aiming to lower out‑of‑pocket costs and curb rising spend on high‑priced...
Eli Lilly announced a $3 bn, ten‑year investment to expand its Chinese supply chain for the oral GLP‑1 weight‑loss drug orforglipron. The plan adds oral solid‑dosage capacity in Beijing, expands the Suzhou injection hub, and includes a $200 m CDMO partnership with Pharmaron....

Researchers have engineered an antibody‑drug conjugate that simultaneously triggers HIV’s entry‑facilitating conformational shift and binds the newly exposed epitopes. In vitro tests showed the dual‑action molecule achieved seven‑ to ten‑fold greater neutralization than the antibody alone and outperformed separate mixtures...

The FDA has granted approval for leucovorin, a folinic‑acid formulation, to treat a rare metabolic disorder despite the absence of new clinical trial data. The decision leans on decades of off‑label use and historical safety records rather than prospective studies....
Veristat announced an expansion of its regulatory and clinical‑trial services aimed at Chinese pharmaceutical and medical‑device companies seeking faster entry into European markets, as well as the United Kingdom, Switzerland, the United States, Canada and Australia. The CRO highlighted recent...
Parabilis Medicines announced that the FDA has granted both Orphan Drug and Fast Track designations to zolucatetide, the first direct inhibitor of the β‑catenin:TCF interaction, for desmoid tumors. Early Phase 1/2 data showed a 100 % disease‑control rate in ten patients and...

Ira Studin warns that while current policies such as TrumpRx and Medicare negotiations exempt orphan drugs, emerging mechanisms like most‑favored‑nation (MFN) pricing and pharmacy‑benefit‑manager (PBM) net pricing could introduce downward pressure. He argues manufacturers should evaluate risk‑contracting to safeguard pricing,...

Life Biosciences has secured FDA IND approval for ER-100, the first partial epigenetic reprogramming therapy to enter human trials. The gene‑therapy delivers OCT‑4, SOX‑2 and KLF‑4 to retinal ganglion cells via a single intravitreal injection, aiming to reset age‑related epigenetic...
DNA‑encoded libraries (DELs) enable billions of compounds to be screened, but X‑Chem argues that size alone does not predict success. By limiting synthesis to two‑three cycles, the company creates lead‑like molecules (350‑450 Da) with high three‑dimensional diversity, and its analysis of...

Astellas Pharma leveraged the Trump administration’s push for lower U.S. drug prices to argue for higher reimbursement for its eye drug Izervay in Japan, winning a more generous price level. Meanwhile, a review of FDA adverse event reports found that...

DNA damage repair gene alterations influence the tumor immune microenvironment in advanced non-small cell lung cancer [Feb 14, 2025] @KamyaSankar et al. @ReckampK Lung Cancer https://t.co/mXZ80zYke3 #lcsm #ImmunoOnc #PrecisionMedicine @TempusAI https://t.co/UJwZnw4qnc
Development & Validation of The Immune Profile Score (IPS), a Novel Multiomic Algorithmic Assay For Stratifying Outcomes in a Real-World Cohort of Pts w/ Adv Solid Cancer Tx'd w/ Immune Checkpoint Inhibitors [May 30, 2025] Zander etal @jitcancer https://t.co/rN5KIoVgPy #ImmunoOnc

The FDA is rolling out the Adverse Event Monitoring System (AEMS), replacing the legacy FAERS platform and consolidating reporting across drugs, biologics, devices, cosmetics, food, tobacco, and veterinary products. AEMS introduces standardized electronic submissions, AI‑driven redaction and analytics, and a...
Real-world clinical genomic analysis of Pts w/ BRAF mutated cancers identifies BRAF class II & III as a population of unmet medical need [3/7/22] Severson et al #ESMO2240P @TempusLabs https://t.co/s5PEBEczlv @Annals_OncologyDOI: https://t.co/c3wxWyUuX0 #PrecisionMedicine #caxtx

GoodRx announced a partnership with Viatris to slash prices on 17 of the latter’s established brand drugs, offering up to 85% off cash prices and as little as $0‑$4 for eligible commercially insured patients. The discounts are available at more...

Mitochondria and Aging: Redox Balance Modulation as a New Approach to the Development of Innovative Geroprotectors (Fundamental and Applied Aspects) https://t.co/TcG9GjMVg7 @IJMS_MDPI https://t.co/gxCC9SsO7z