ASGCT 2026: AI-Optimized Cas12l Gene Editor Offers Compact Cas9 Alternative
Researchers at Caszyme and Vilnius University unveiled an AI‑engineered Cas12l variant, M82, that delivers 67% indel editing efficiency—essentially on par with the industry‑standard Cas9. The 867‑amino‑acid nuclease is markedly smaller, recognizing C‑rich PAMs and showing up to 56% homology‑directed repair rates. By merging cryo‑EM structure data with the ESM‑2 protein language model, the team achieved a 7‑fold boost over wild‑type Cas12l and minimal off‑target activity. The compact size positions M82 as a strong candidate for AAV‑based therapeutic delivery.
Heat-Treated Postbiotic Linked to Skin Elasticity Gains in Clinical Trial
Japanese researchers conducted an eight‑week, double‑blind, placebo‑controlled trial of heat‑treated *Latilactobacillus sakei* KABP‑065 in 60 healthy women aged 30‑50. Participants taking the postbiotic capsule showed statistically significant improvements in skin elasticity, with the most pronounced gains in women in their...
Former Ovid CEO Dr. Jeremy Levin’s Rallying Cry for Biotech
Former Ovid Therapeutics CEO Dr. Jeremy Levin is launching a new book, *Biotech in the Balance*, slated for May 19. The book outlines a roadmap for positioning biotech as a strategic U.S. industry, calling for tax incentives, stronger patient engagement, and...
Protara Therapeutics Reports P-II (ADVANCED-2) Trial on TARA-002 in BCG-Naïve Non-Muscle Invasive Bladder Cancer (NMIBC)
Protara Therapeutics presented 12‑month data from Cohort A of its Phase II ADVANCED‑2 trial evaluating TARA‑002 in patients with carcinoma in situ or CIS‑plus‑Ta/T1 non‑muscle‑invasive bladder cancer (NMIBC) who are BCG‑naïve. Of the 31 enrolled, 29 were efficacy‑evaluable, yielding an overall...
CAR-T Therapy for Stiff Person Syndrome Nears Approval
Kyverna Therapeutics is close to securing regulatory approval for its CAR‑T cell therapy aimed at treating stiff person syndrome (SPS), a rare autoimmune neurological disorder with no approved drugs. The therapy uses engineered T cells to eliminate the B‑cell populations...
Making Eyes ‘Photosynthetic’ Could Treat Common Vision Problem
A team from the National University of Singapore has created a light‑activated particle called LEAF, derived from whole spinach thylakoids, that boosts NADPH and ATP production in mammalian eye cells. When applied as eye drops to mice with chemically induced...
Arna Pharma and Slate Run Complete JV for New US Company
Arna Pharma and Slate Run Pharmaceuticals have completed a joint venture to create a U.S.-based specialty pharmaceutical company focused on branded products, 505(b)(2) medicines, and specialized generics. The partnership launched its first collaborative product, Aridol, and plans to add more...
NICE Backs Stomach Cancer, PAH Drugs for NHS Use
The UK’s health technology assessment body NICE has endorsed AstraZeneca’s immunotherapy Imfinzi for peri‑operative treatment of aggressive stomach and gastro‑oesophageal junction cancers, and approved Merck & Co.’s Winrevair as an add‑on therapy for pulmonary arterial hypertension (PAH). More than 1,500 patients in...
CAR T-Cell Therapy Bolstered by Stiffening up Cancer Cells First
Researchers have discovered that pre‑treating cancer cells to increase their stiffness markedly improves the effectiveness of CAR T‑cell therapy in mouse models of aggressive melanoma. The physical alteration of tumor mechanics enhances immune cell infiltration and tumor killing, offering a novel...
Discovery Could Reshape RNA Editing with DNA-Guided CRISPR
University of Florida engineers unveiled the first DNA‑guided CRISPR system, allowing Cas12 enzymes to locate and edit RNA targets using stable DNA guides. The approach cuts guide‑synthesis costs, boosts specificity, and delivers near‑perfect diagnostic accuracy for viruses like HIV and...
Kyowa Kirin Secures FDA Approval for Crysvita Dosing Update
Kyowa Kirin has secured FDA approval to expand Crysvita’s dosing regimen for adults with X‑linked hypophosphataemia (XLH). The update allows a 0.5 mg/kg dose up to 90 mg every two weeks, with the option to increase to 1 mg/kg after four weeks if serum...
Kyowa Kirin Secures FDA Approval for Crysvita Dosing Update
Kyowa Kirin has secured FDA approval for a dosing update to Crysvita (burosumab‑twza) for adults with X‑linked hypophosphataemia (XLH). The new regimen permits a 0.5 mg/kg dose every two weeks, with the option to increase to 1 mg/kg after four weeks if serum...
Biogen Reports P-II (CELIA) Trial Results on Diranersen in Early Alzheimer’s Disease
Biogen disclosed phase‑II (CELIA) results for intrathecal diranersen in 416 patients with early Alzheimer’s disease. The trial did not meet its primary dose‑response endpoint on CDR‑SB at week 76, but all dosing arms, especially 60 mg every‑four‑weeks, showed a slowing of clinical...
Parkinson’s Disease in Women: Research Gaps, Treatment Challenges, and New Hope Through GEM-PD
In Women’s Health Month, Bio.News highlighted the systemic gaps affecting women with Parkinson’s disease, from delayed diagnoses to under‑representation in clinical trials. Ragasudha Botta, senior scientific director at C‑Path’s Critical Path for Parkinson’s (CPP), explained how biological sex differences influence...
Racura Oncology Advances CPACS Trial to Next Dose Level
Racura Oncology (ASX:RAC) received Safety Review Committee clearance to double the RC220 dose from 40 mg/m² to 80 mg/m² for Cohort 2 of its CPAPS Phase 1 trial, pairing the drug with doxorubicin. The first three Cohort 1 patients showed no dose‑limiting toxicities, and all...
Archer Materials Advances Biosensor Prototype Toward Preclinical Validation
Archer Materials announced that its alpha Biosensor prototype, built on its Biochip platform, has reached clinical‑grade potassium‑sensing accuracy. The company is now engineering a beta prototype designed for pre‑clinical validation and eventual patient trials, with the first beta system expected...
Biogen Finds Alzheimer’s Path for Tau ASO Despite Mixed Data
Biogen reported Phase 2 data for its tau‑targeting antisense oligonucleotide (ASO) in Alzheimer’s disease, showing a roughly 30% reduction in cerebrospinal fluid tau levels. Cognitive endpoints, however, delivered only modest, statistically non‑significant improvements versus placebo. The safety profile remained clean,...
Viagra Shows Promise as Potential Treatment to Halt Peyronie’s Disease
A Phase‑II trial of 133 men with acute Peyronie’s disease tested an off‑label combination of a PDE5 inhibitor (sildenafil or tadalafil) and the SERM tamoxifen. After three months, 43% of participants reported a meaningful reduction in penile curvature versus 15%...
60 Degrees Pharmaceuticals Inc (SXTP) Q1 2026 Earnings Call Transcript
United Therapeutics reported $782 million in Q1 2026 revenue, with Tyvaso contributing $458 million and showing 9% year‑over‑year growth despite seasonal headwinds. Phase III data revealed ralinepag delivering a threefold reduction in PAH disease progression and meeting all primary endpoints. The company announced a...
Gossamer Bio Inc (GOSS) Q1 2026 Earnings Call Transcript
Gossamer Bio reported an interim analysis of its Phase 2b LEDA study for GB001, which the independent data monitoring committee endorsed, allowing the company to begin Phase 3 planning. The firm also disclosed promising Phase 1b data for GB004 in...
Precision Therapies Offer New Hope Against Drug-Resistant Bacteria
Precision medicine is reshaping the fight against drug‑resistant bacteria as researchers unveil engineered phages and AI‑designed antimicrobial peptides that selectively eradicate superbugs. Early‑stage animal studies reported near‑complete clearance of MRSA and carbapenem‑resistant Enterobacteriaceae without harming beneficial microbes. A first‑in‑human Phase 1 trial...
New Radiopharmaceutical Achieves Remission in Difficult-to-Treat Pancreatic Cancer
Researchers have unveiled a novel radiopharmaceutical, ¹⁷⁷Lu‑AKIR001, that homes in on the CD44v6 protein variant prevalent in pancreatic ductal adenocarcinoma (PDAC). In mouse xenograft studies, a single 12 MBq dose achieved complete remission in 40% of subjects when used alone and...
Encoded's Gene Therapy Reduced Seizures in Dravet Syndrome
Encoded Therapeutics reported that its experimental gene therapy cut seizure frequency by 76 % in three children with Dravet syndrome, a severe childhood epilepsy. The effect was seen in patients receiving the second‑highest dose among four dose levels in a small...
ASGCT Dispatch: In Vivo CAR-T Is Everywhere
At the American Society of Gene & Cell Therapy (ASGCT) meeting in Boston, developers showcased a wave of in vivo CAR‑T programs, signaling a shift from traditional ex‑vivo manufacturing. Companies unveiled preclinical and early‑phase clinical data demonstrating tumor shrinkage using...
Materializing Safe, On-Demand Living Therapeutics
Harvard’s Wyss Institute unveiled an Implantable Living Materials (ILM) platform that embeds genetically engineered E. coli within a polyvinyl‑alcohol matrix to deliver therapeutics on demand. The bacteria are programmed to detect pathogenic Pseudomonas aeruginosa and release a killing molecule, successfully treating...
A Pill Can Stop People From Developing COVID After Being Exposed to the Virus, Trial Finds
Japanese drug ensitrelvir, already approved in Japan for treating mild COVID‑19, has demonstrated post‑exposure prophylactic efficacy in a double‑blind phase III trial. Among more than 2,000 household contacts, the five‑day oral regimen cut symptomatic infection rates from 9% to 2.9% and...
Clinical Trial Challenges Long Held Beliefs About Treating Brittle Bone Disease
A large eight‑year clinical trial (TOPaZ) involving 350 adults with osteogenesis imperfecta showed that drugs designed to increase bone density did not reduce fracture rates. Participants receiving the bone‑density regimen experienced a 37% fracture incidence, virtually identical to the 36%...
Roche Rolls Out Tecentriq SC Immunotherapy for Lung Cancer with the Promise of Less Treatment Time
Roche has launched its sub‑cutaneous form of Tecentriq (atezolizumab) in India, offering an immunotherapy for lung cancer that can be administered in about seven minutes versus several hours for traditional IV infusions. The SC version is priced at roughly ₹3.7 lakh...
Generative AI System Could Cut Animal Testing by Up to 50% in Preclinical Research
Researchers at Goethe University Frankfurt, Philipps University of Marburg and the Fraunhofer Institute for Translational Medicine and Pharmacology have unveiled genESOM, a generative AI tool that creates synthetic preclinical data. Trained on small experimental sets, it can mimic real laboratory...
AstraZeneca Follows Merck with Phase 3 Win in Bladder Cancer
AstraZeneca announced that its checkpoint inhibitor Imfinzi, co‑developed with Pfizer, combined with Astellas’ antibody‑drug conjugate Padcev, significantly extended overall survival in a Phase 3 trial for advanced bladder cancer. The regimen outperformed the current standard of care, marking the company’s second...
Biogen Takes Tau Alzheimer’s Therapy to Phase 3 Despite Mid-Stage Fail, Prompting Cautious Optimism
Biogen is advancing its tau‑targeting Alzheimer’s drug diranersen (BIIB080) to Phase 3 despite a failed primary endpoint in the Phase 2 CELIA trial. The study of over 400 early‑stage patients showed a strong reduction in cerebrospinal‑fluid tau and modest slowing of cognitive...
AstraZeneca Reports the P-III (VOLGA) Trial Data on Imfinzi + Enfortumab Vedotin (EV) for Muscle-Invasive Bladder Cancer
AstraZeneca presented Phase III VOLGA data showing that the combination of Imfinzi (durvalumab) and Enfortumab Vedotin improves event‑free survival and overall survival in muscle‑invasive bladder cancer patients who cannot receive cisplatin. The trial enrolled 695 patients undergoing radical cystectomy and compared two...
Why AI Fails in Drug Development and How to Build Tools That Actually Deliver Real Value
Artificial intelligence can add real value to drug development only when it is tightly woven into existing expert‑led workflows and respects real‑world constraints. The authors, drawing on their experience at Intrepid Labs and Quotient Sciences, illustrate how AI tools that...
BeOne Wins Mantle Cell Lymphoma Approval, Opening New Therapy Class
The FDA granted accelerated approval to BeOne Medicines’ BCL2 inhibitor sonrotoclax, marketed as Beqalzi, for patients with relapsed or refractory mantle‑cell lymphoma who have failed at least two prior therapies, including a BTK inhibitor. The drug is the first BCL2...
Alembic Pharma Gets USFDA Tentative Approval for Generic Prostate Cancer Drug
Alembic Pharmaceuticals received tentative USFDA approval for its 300 mg generic Darolutamide tablets, deemed therapeutically equivalent to Bayer’s Nubeqa. The approval applies to an Abbreviated New Drug Application, clearing a major regulatory hurdle for the Indian firm. Darolutamide targets prostate cancer...
STAT+: Biogen’s Tau-Targeting Alzheimer’s Drug Posts Mixed Results in Mid-Stage Study
Biogen’s Phase 2 trial of the tau‑targeting Alzheimer’s drug diranersen (BIIB080) showed that the compound lowered tau protein in cerebrospinal fluid and brain tissue and was linked to a modest slowing of cognitive decline. The study evaluated three escalating dose levels,...
FDA Hands BeOne, Taiho Approvals for Blood Cancers
BeOne Medicines’ BCL‑2 inhibitor Beqalzi (sonrotoclax) earned FDA accelerated approval as a third‑line treatment for mantle‑cell lymphoma, marking the first U.S. approval of a BCL‑2 drug for this indication. The approval is based on a phase 1/2 trial showing a 52%...
Regulatory Round-Up: A Month of Key FDA Approvals for Cancer Drugs
The FDA approved four oncology drugs in May 2026, marking a focused regulatory push in cancer therapeutics. Taiho Oncology received clearance for the oral Inqovi‑venetoclax regimen for elderly acute myeloid leukemia patients, while BeOne Medicines earned accelerated approval for Beqalzi...
Imfinzi Set to Become First Immunotherapy for Stomach Cancer Patients on NHS
AstraZeneca’s immunotherapy Imfinzi (durvalumab) has received NICE approval, becoming the first immunotherapy available on the UK NHS for patients with resectable gastric and gastro‑oesophageal junction cancers. The approval follows the Phase III MATTERHORN trial, which showed that adding Imfinzi to standard...
BeOne’s Next-Gen BCL2 Inhibitor Wins FDA Approval, Taking Aim at Venclexta
BeOne Medicines received accelerated FDA approval for sonrotoclax, its next‑generation BCL‑2 inhibitor, for mantle cell lymphoma. The decision rests on a single‑arm trial that delivered a 68% overall response rate in heavily pre‑treated patients. Sonrotoclax is positioned to compete directly...
AZ Cues up Broader Use of Imfinzi in Bladder Cancer
AstraZeneca’s immunotherapy Imfinzi (durvalumab) demonstrated a significant survival benefit in the phase‑3 VOLGA trial for muscle‑invasive bladder cancer (MIBC) patients who cannot receive platinum chemotherapy. The study combined Imfinzi with Pfizer‑Astellas’ antibody‑drug conjugate Padcev before surgery, extending both event‑free and...
Osel and Partners Launch Phase IIb FLIP-2 Trial of Lactin-V
Osel, Imperial College London and the March of Dimes have launched the Phase IIb FLIP‑2 trial to evaluate Lactin‑V, a live‑biotherapeutic containing Lactobacillus crispatus, for preventing preterm birth in high‑risk pregnancies. The study will enroll 360 women across four UK maternity...
Pfizer Receives EC Approval for Hympavzi to Treat Haemophilia
Pfizer secured European Commission marketing authorisation for Hympavzi (marstacimab) to treat hemophilia A or B patients with factor inhibitors aged 12 and older who weigh at least 35 kg. The once‑weekly subcutaneous therapy eliminates routine lab monitoring and delivered a 93%...
Pfizer Receives EC Approval for Hympavzi to Treat Haemophilia
Pfizer secured European Commission marketing authorisation for Hympavzi (marstacimab) to treat hemophilia A or B with inhibitors in patients aged 12 years and older weighing at least 35 kg. The once‑weekly subcutaneous therapy showed a 93% reduction in mean annualised bleeding rate versus on‑demand...
Taiho Oncology (Part of Taiho Pharmaceutical) Reports the US FDA Approval of Inqovi + Venetoclax for Newly Diagnosed AML
The U.S. Food and Drug Administration approved Inqovi (decitabine and cedazuridine) in combination with venetoclax for newly diagnosed acute myeloid leukemia patients who are 75 years or older or otherwise ineligible for intensive induction chemotherapy. The decision is based on...
BeOne Medicines Receives US FDA Accelerated Approval for Beqalzi (Sonrotoclax) to Treat R/R Mantle Cell Lymphoma
BeOne Medicines received FDA accelerated approval for its BCL‑2 inhibitor Beqalzi (sonrotoclax) to treat adults with relapsed or refractory mantle‑cell lymphoma after at least two prior lines, including a BTK inhibitor. The decision was based on a global Phase I/II...
Providing a Cellular ‘All-Clear’ Signal to Resume Protein Synthesis
Scientists at EMBL and the University of Virginia identified a previously unknown protein, SNOR, that binds to ribosomes in glucose‑starved yeast and induces a dormant, low‑translation state. Using in‑situ cryo‑electron tomography and visual proteomics, they mapped SNOR at the ribosomal...
Argenica Therapeutics Establishes Expert Committee to Advance Phase 2b Stroke Trial
Argenica Therapeutics has created a Clinical Advisory Committee to steer the design of its Phase 2b trial of the neuroprotective agent ARG‑007 for acute ischemic stroke. The committee, featuring leading stroke neurologists such as Professors Geoffrey Donnan and Jeffrey Saver, will...
Atrium Therapeutics Inc (RNA) Q1 2026 Earnings Call Transcript
Sarepta Therapeutics reported Q1 2026 revenue of $731 million, a modest 2% decline, while posting GAAP operating profit of $358 million and ending the quarter with $748 million in cash. The company reiterated full‑year revenue guidance of $1.2‑$1.4 billion and highlighted non‑cash collaboration income...
Sangamo Therapeutics Inc (SGMO) Q1 2026 Earnings Call Transcript
Sangamo Therapeutics announced that the FDA reaffirmed the use of eGFR slope as an endpoint for an accelerated Biologics License Application for its Fabry gene therapy ST-920, positioning a filing as early as Q1 2026. The STAR study showed a mean...