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The Swiss drugmaker is initiating a Phase 3 study of the gene therapy after a regulatory setback in Europe.
Patients who saw substantial hair regrowth in Nektar's trial experienced continued improvement in a follow-up study.
Amazon Web Services introduces a new AI platform designed to accelerate pharmaceutical research using biological foundation models.
Traditional randomized clinical trials are impractical for ultra-rare diseases with patient populations as small as 10, prompting calls for flexible evidence generation approaches.
A roundup from Genetic Engineering News covers AI-driven oncology progress, CRISPR screens for HIV, liver tissue engineering, and pharma business updates from recent conferences.
Phase I trial data for investigational antibody-drug conjugate QLS5132, presented at AACR, demonstrated over 50% response rates in platinum-resistant ovarian cancer patients regardless of CLDN6 expression.
At the AACR 2026 conference, experts highlight AI's growing role in cancer research from organoid models to pathology, though adoption and trust barriers remain.
Researchers examine whether weight reduction drives semaglutide's cardiovascular benefits in the SELECT trial.
Researchers used base editing to correct a cystic fibrosis mutation in cell and organoid models that does not respond to current therapies.
Otarmeni, the first gene therapy cleared under the FDA's national priority voucher program, will be offered free to eligible patients with a rare inherited hearing loss.
A phase 2 trial finds L9LS safe and effective against malaria in young children in western Kenya, though higher doses may be needed for high-level efficacy.
Research reveals that mechanical forces from the heart's beating alter gene regulation to prevent cancer cell proliferation in cardiac tissues.
Health Secretary Robert F. Kennedy Jr. is moving to loosen federal restrictions on peptides, potentially igniting a wellness trend similar to weight-loss drugs despite scant human evidence.
The contract research organization's shares fell sharply after new business bookings disappointed investors.
A clinical trial in China for a gene therapy targeting a rare form of deafness reported significant hearing gains in the vast majority of participants.
A novel AI tool called Path-IO accurately stratified patient outcomes in metastatic NSCLC, validated across international cohorts and a Phase III trial.
Chinese firms accounted for six of 26 major licensing and acquisition deals over 16 months, representing a significant portion of the sector's headline value.
The company has established a high-grade controlled environment for manufacturing sterile single-use assemblies used in bioprocessing and vaccine production.
Researchers have mapped key human genes that either promote or block HIV infection using CRISPR technology.
A new executive order seeks to streamline research and development of psychedelic therapies for mental health conditions.
The biotech company's experimental therapy shows potential in early studies for treating inflammation linked to cardiovascular risk and obesity.
The new biotech startup aims to create a novel class of genetic medicines by combining strengths from multiple existing approaches.
The pharmaceutical giant's second recent deal in the space centers on an early-stage therapy for multiple myeloma.
Researchers are engineering laboratory models to systematically decode how cancers evade targeted treatments.
A new company has launched to provide fluid management components for biologic drug manufacturing and cell and gene therapy production.
A novel gene delivery approach curbed harmful lung inflammation in infected mice without triggering systemic immune effects.
Analysts report nearly 1,400 prescriptions written for the newly launched weight-loss drug in its first two days.
A new seminar details the progression, cancer risks, and treatment challenges of the rare chronic liver condition.
A phase 3 trial found adding relacorilant to nab-paclitaxel significantly improved overall survival without requiring biomarker selection.
The obesity drug developer's massive offering will fund a portfolio of medicines licensed from China's Hengrui Pharma.
A new commentary highlights the need for genomic integrity monitoring in regenerative medicine using human pluripotent stem cells.
Researchers have developed an improved method for creating insulin-producing cells from human stem cells that reversed diabetes in a mouse model.
The biotech firm is reducing its workforce following another regulatory setback for its lead candidate.
Researchers have discovered new genetic markers for hyperemesis gravidarum, potentially revealing biological mechanisms and treatment pathways.
Regulators have asked Eli Lilly to conduct a post-approval study of its weight-loss drug Foundayo's potential liver safety concerns.
The California biotech secured funding to progress its pipeline of drugs targeting the AKT protein, with one candidate already in Phase 1 trials.
The startup aims to overcome bottlenecks in cryo-EM workflows for antibody-antigen analysis.
A reverse merger and $350 million private placement will fund clinical development of a novel cell therapy platform.
A new single-cell tracking method systematically reveals how genetic variations reshape cells in complex diseases like cancer.
The pharmaceutical giant's leader is taking a board seat at the AI lab, signaling deeper collaboration between the two sectors.
A newly characterized Cas12f nuclease variant demonstrates markedly improved efficiency, advancing compact genome editors for targeted delivery.
Regulators have asked Eli Lilly for additional information to assess potential heart and liver risks associated with its newly approved weight-loss drug.
The biotech firm is cutting staff and confronting existential questions after receiving another complete response letter for its cancer therapy.
The pharmaceutical giant announced a broad collaboration to embed artificial intelligence throughout its drug discovery and development processes.
The agency has warned more than 2,200 entities to report trial results or face potential fines.
The pharmaceutical giant is buying antibody-drug conjugate startup CrossBridge Bio in a deal that could reach $300 million.
The Parker Institute for Cancer Immunotherapy is increasing its focus on mRNA-based cancer vaccines while other developers grow wary of the politically charged technology.
Ben Sasse offers a personal account of his experience in a clinical trial for a new pancreatic cancer treatment.
A STAT News opinion piece argues that adults with rare diseases are often excluded from accessing breakthrough treatments approved for children.
Spyre Therapeutics' inflammatory bowel disease candidate demonstrated remission rates suggesting a 'best-in-class' profile in an early study.
A new targeted therapy for pancreatic cancer nearly doubled survival in a pivotal trial, offering a potential breakthrough for a notoriously difficult-to-treat disease.
Connected medical devices are driving advances in patient care and shaping the development of next-generation therapies.
Pharma experts advocate for trial designs that better measure long-term durability and real-world patient outcomes.
The search for a new director at the FDA's biologics division highlights the role's significant pressures and complex regulatory landscape.
Two biotech companies have filed for initial public offerings, signaling renewed activity in the sector.
A new study provides the first detailed structural insight into Bornavirus replication, uncovering unique RNA binding and assembly mechanisms.
New research suggests the interplay between gut bacteria and epigenetic markers at birth may influence the development of conditions like ASD and ADHD.
A new wave of autonomous AI systems is reshaping drug discovery and laboratory workflows.
The Shanghai-based biotech is advancing a GPC3-targeted CAR-T therapy for liver cancer as it prepares for a public listing.
Pharmaceutical innovation for menopause symptoms faces persistent scientific and commercial hurdles.
A correspondence in The Lancet highlights a critical difference between raw measurements and standardized scores for a vascular inflammation marker.
A new software portal aims to streamline workflows and reduce errors between labs and their external partners.
The biotech, backed by Novo Holdings, aims to develop improved treatments for idiopathic pulmonary fibrosis.
The long-established venture capital playbook for funding biotech startups is being upended by new challenges and market forces.
San Diego startup with seven employees secures funding from blue-chip investors to develop antibody-drug conjugates with reduced toxicity.
Harvard biologist David Sinclair's startup secures funding for one-time treatment aimed at reversing cellular aging.
The pharmaceutical company has made the 7.2 mg injectable form of its GLP-1 agonist available to US patients.
Pharmaceutical industry sees increased approvals of covalent drugs across multiple therapeutic areas, building on century-old foundation established by aspirin and penicillin.
Federal health agency funds Duke University and other academic centers to test experimental regenerative medicines for osteoarthritis in clinical trials.
Researchers develop compounds that reduce harmful inflammation while preserving infection-fighting capabilities.
Leading CRISPR researcher Professor Rodolphe Barrangou explores the technology's evolution from discovery to clinical applications in inaugural industry keynote.
The collaboration combines single-cell technology with machine learning to accelerate high-performance clone selection for biomanufacturing.
The AI company is expanding into life sciences with its largest acquisition to date.
Kezar Life Sciences closed despite reaching FDA agreement, highlighting timing pressures facing small biotech firms.
BioPharma companies explore reward strategies to address ongoing enrollment challenges in clinical studies.
Telehealth startup MEDVi faces FDA scrutiny despite founder's billion-dollar valuation claims.
New therapeutic approach targets metabolic dysfunction in red blood cells to treat the world's most common genetic disorders.
The annual genomics conference delivered significant technology announcements and scientific presentations showcasing new applications.
Researchers develop method to assemble conductive polymers in vivo using the body's natural enzymes.
The biotech's experimental eye treatment missed primary endpoints in late-stage trials, sending stock down alongside other companies with disappointing clinical updates.
Eli Lilly's once-daily oral obesity drug receives fastest FDA approval since 2002, cleared in just 50 days under priority program.
Utah biotech's LPCN 1154, an oral version of brexanolone, failed to meet primary endpoints in clinical testing.
The biotech will pursue registrational trials for its PRC2 inhibitor but faces questions over combination therapy strategy.
Study finds damaging variants in innate immunity genes accelerate breast cancer onset in women with BRCA1 mutations.
The California-based drugmaker terminated the study after placing it on hold for safety reasons.
New program creates diverse iPSC models from ALS patients to capture both genetic and sporadic disease forms.
Paragon Therapeutics offshoot pursues public listing to fund Alzheimer's disease research amid sector momentum.
Computational research shows protein families remain limited by ancestral starting points despite vast theoretical sequence possibilities.
Partnership leverages somatic genomics platform to identify novel therapeutic targets for inflammatory bowel disease.
The FDA flagged severe safety issues with Amgen's autoimmune treatment, though the company says it previously disclosed liver risks.
Two major pharmaceutical acquisitions announced, with Lilly targeting Centessa and Biogen acquiring rare disease specialist Apellis.
Excessive social media marketing for psychedelic treatments could jeopardize the industry's efforts to gain regulatory approval.
UK biotech will continue development with nonprofit funding after pharma giant declines to exercise option.
The startup's RNA-based treatment platform could potentially be reused across multiple diseases.
The pharmaceutical giant is making its largest acquisition in years to gain access to experimental sleep disorder and neurological treatments.
Six companies raised a median $287.5 million, surpassing historical first-quarter fundraising levels.
Amlitelimab hit primary endpoints in three Phase 3 trials but triggered analyst downgrades due to efficacy and safety questions.
The Ron Renaud-led company is developing a late-stage obesity drug to compete with Novo Nordisk's Wegovy and Eli Lilly's Zepbound.
The collaboration spans multiple therapeutic areas as Lilly seeks to maintain its industry leadership position.
WVE-007 showed minimal weight loss of 0.9% in Phase I trial, far below investor expectations for obesity treatment.