BioSpace

REGENXBIO Secures Legal Win in Gene Therapy Patent Battle Vs. Sarepta

An appellate court reversed a lower‑court ruling, siding with REGENXBIO in its patent dispute against Sarepta Therapeutics over an adeno‑associated virus (AAV) vector used in Duchenne muscular dystrophy gene therapy. The judge held that the patented composition, as a whole,...

Novo’s CagriSema Falls to Lilly’s Zepbound in Daring Head-to-Head Test

Novo Nordisk’s experimental obesity drug CagriSema delivered a 23% average weight loss in the 84‑week REDEFINE 4 Phase 3 trial, but fell short of Eli Lilly’s tirzepatide (Zepbound) which achieved 25.5%. The head‑to‑head study failed the primary non‑inferiority endpoint, prompting a 15% drop...

Sickle Cell Gene Therapies Casgevy and Lyfgenia Still Lacking Traction 2 Years In

In February 2024 Vertex and CRISPR Therapeutics launched Casgevy, and bluebird bio (now Genetix Biotherapeutics) launched Lyfgenia as the first FDA‑approved gene therapies for sickle cell disease. Two years later, uptake remains modest—only 64 patients received Casgevy and just over 100...

Beyond Rezdiffra: The 4 Most Promising MASH Pipelines in Play

Madrigal’s Rezdiffra became the first approved therapy for metabolic dysfunction‑associated steatohepatitis (MASH), generating $817 million in revenue within six quarters and prompting a wave of big‑pharma acquisitions focused on FGF21 analogs. In 2025, GSK, Roche and Novo Nordisk spent nearly $10 billion acquiring...

Redefining Obesity Treatment Beyond GLP-1 Limits

BioSpace’s Denatured podcast featured Verdiva Bio’s R&D head Jane Hughes and MitoRx CEO Jon Rees discussing next‑generation obesity therapies that move beyond the limitations of GLP‑1 agonists. They highlighted how GLP‑1 treatments can cause muscle loss and suffer from poor...

9 Companies Hiring Now in Pennsylvania

Pennsylvania’s Greater Philadelphia corridor remains a biotech hotbed, housing over 1,200 life‑science firms including AstraZeneca, GSK and Johnson & Johnson. Eli Lilly announced a new injectable weight‑loss manufacturing plant in the Lehigh Valley, projected to create roughly 850 jobs by 2031....

Extending Scientific Rigor From Bench to Boardroom

Researchers who launch biotech startups often abandon evidence‑based decision‑making once they enter the boardroom, falling prey to technology myopia, base‑rate neglect, and confirmation bias. Their lack of formal training in commercialization, financial modeling, and competitive analysis amplifies these pitfalls, leading...

Korsana Joins Alzheimer's Amyloid Arena With $150M Debut, Next-Gen Antibody

When Senior Leaders Are Asked To Carry Too Much

Biopharma senior leaders are increasingly shouldering operational work left behind by downsizing, stretching their capacity beyond strategic duties. The overload fragments focus, slows decisions, and erodes communication, while burnout remains hidden behind continued presence. As pressure builds, the organization’s decision‑making...

Early-Stage Biotechs Suffer in 2025 As VC Shuns Risk: PitchBook

Venture capital in biopharma rebounded in 2025, delivering $33.8 billion across 1,171 deals, but the capital is increasingly concentrated in late‑stage companies. Early‑stage startups received only 32% of the funding, down from over 40% during the pandemic surge, while median deal...

Ongoing Regulatory Uncertainty Means Innovation Is Now Higher Risk

Regulatory uncertainty is rising as the FDA shifts leadership, adopts a single‑trial approval pathway, and tightens its benefit‑risk framework. While the one‑trial standard promises faster, cheaper market entry, recent surprise complete response letters (CRLs) show that even successful Phase 3 results...

Ionis Paves Its Own Path as Initial Tryngolza Launch Defies Expectations

Ionis Therapeutics announced that its GSK‑partnered antisense drug bepirovirsen achieved a statistically significant functional cure rate of roughly 15‑20% in a Phase 3 trial, far surpassing the 1‑3% rate of existing hepatitis B therapies. The company also reported a strong first‑year launch...

Proliferating Patents, Lawsuits Stave Off Pharmas’ Generic Competitors

A new analysis in Health Affairs Scholar reveals that brand‑name drugmakers are exploiting the Hatch‑Waxman framework through serial patent litigation and continuation patents to extend market exclusivity. By filing multiple overlapping patents, companies can repeatedly sue generic challengers, triggering 30‑month...

Korsana Raises $150M in Series A to Develop Next‑gen Alzheimer’s Antibody

Korsana, a biotech focused on Alzheimer’s disease, announced a $150 million Series A funding round to advance its next‑generation amyloid‑targeting antibody. The capital will fund clinical development and expansion of its pipeline. Investor identities were not disclosed.

Does Vaxart Hold the Key to the Elusive Norovirus Vaccine?

Vaxart is advancing an oral, bivalent norovirus vaccine pill (VXA‑G1.1‑NN) that demonstrated a 30% reduction in infection in a Phase 2b challenge study. The vaccine uses a non‑replicating adenovirus vector loaded with a dsRNA adjuvant to stimulate gut immunity, and a...

As FDA Moves Away From Animal Testing, AI and Organoids Can Shine

The FDA is actively phasing out animal‑testing requirements for certain therapies, a move echoed by the NIH and codified in the pending FDA Modernization Act. This regulatory shift opens the door for artificial intelligence and human‑derived organoid models to become...

Biopharma Must Cope With The Fetal Bovine Serum Squeeze

Biopharma faces a tightening fetal bovine serum (FBS) market as U.S. cattle inventories hit record lows and European disease outbreaks curb supply. Prices have surged more than 300% over five years and release timelines have lengthened from weeks to months...

2025 Q4 Job Market Report: Positive Signs Emerge for Job Seekers

The fourth quarter of 2025 marked the first quarter‑over‑quarter rise in biopharma job postings, with a 10% increase and a 4% uptick in average listings, despite a 14% year‑over‑year decline. Science/research and development roles grew 14% while clinical positions surged...

‘Just Agree to It:’ Pazdur Said He Was Told To Cosign FDA’s Reduced Trial Requirements

Richard Pazdur, longtime FDA oncology leader, resigned after being pressured to endorse a controversial policy that would reduce required drug approval trials from two to one. He said Commissioner Marty Makary breached the traditional independence between the commissioner’s office and...

2026 Could Mark a Turning Point for American Innovation

The United States faces a potential decline in biotech leadership as recent Supreme Court decisions blur patent eligibility and congressional price‑control measures under the Inflation Reduction Act force program cancellations. Proposals to seize university licensing revenue further strain tech‑transfer offices,...

How DIA Is Helping Regulators Turn AI Principles Into Everyday Review Practice

At the 2026 DIA Global Annual Meeting, the DIA AI Consortium will showcase how regulators such as the FDA are embedding artificial intelligence into medical product review workflows. The FDA’s 2025‑2026 roadmap uses AI to automate routine tasks, accelerate timelines,...

Vertex, CRISPR Set Lofty Goal for Casgevy Gene Therapy as Patient Starts Ramp

Vertex Pharmaceuticals and CRISPR Therapeutics forecast combined Casgevy gene‑therapy and Journavx pain‑drug sales of $500 million in 2026, a 185 % jump from 2025. The therapy generated $115.8 million in 2025, with Q4 revenue of $54.8 million, and treated 111 patients that year, up...

All Eyes on Vertex’s Kidney Franchise, as Painkiller Journavx Tops Half a Million Scripts

Vertex Pharmaceuticals reported that its late‑stage RAINIER trial of povetacicept in IgA nephropathy is proceeding without safety concerns, with primary data expected in the first half of 2026. The company is leveraging a priority‑review voucher for the upcoming FDA filing...

Evommune Soars as Dermatitis Treatment Rivals Dupixent in Mid-Stage Trial

Evommune’s shares surged 75% after its Phase 2a trial showed the therapeutic protein EVO301 reduced atopic dermatitis severity by 33% versus placebo. The study enrolled 70 patients, with a 55% reduction in the treatment arm compared to 22% in the placebo...

HER2’s Digital Rebirth Is Unlocking the Full Potential of ADCs

The latest wave of antibody‑drug conjugates (ADCs) hinges on precise HER2 quantification, and digital pathology combined with AI is delivering that accuracy. By analyzing gigapixel slides, AI models can detect low and ultra‑low HER2 expression that traditional microscopy often misses,...

Facing Talent Crunch, Radiopharma Field Casts a Wide Net

Investment in radiopharmaceuticals is driving a fierce talent shortage as big‑pharma players like AstraZeneca, Novartis and Eli Lilly expand into the modality. The success of lutetium‑177 therapies such as Lutathera and Pluvicto has sparked a surge in clinical studies, intensifying demand...

Biogen’s Much Anticipated Tau Readout in Alzheimer’s Will Spur More Questions

Biogen expects Phase 2 readout for its tau‑targeting antisense oligonucleotide BIIB080 in the second or third quarter of 2026. The CELIA trial evaluates three dose levels through week 76 and uses cognition as the primary endpoint. A positive signal could lift Biogen’s...

Liver Toxicities Force Pause on Two Dose Groups of UniQure’s Mid-Stage Fabry Study

UniQure paused dosing in the mid- and high-dose arms of its Phase 1/2a Fabry gene‑therapy trial after two patients experienced grade 3 liver enzyme elevations. The affected cohorts received 4 × 10¹³ and 6 × 10¹³ genome copies per kilogram of AMT‑191, while the low‑dose group...

Ultragenyx’s Gene Therapy Shows Long-Term Efficacy in Two Sanfilippo Studies

Ultragenyx reported that its investigational AAV gene therapy UX111 delivers sustained cognitive improvements in Sanfilippo A patients, with follow‑up extending up to 8.5 years. Data from 33 dosed participants—27 receiving the highest dose—showed a 23.3‑point gain on the Bailey‑III scale...

FDA Policy Tracker 2026: Priority Vouchers Questioned, PRVs Return

2026 regulatory landscape sees the FDA reviving the rare‑pediatric disease priority review voucher (PRV) program, launching the PreCheck manufacturing pilot, and issuing new guidance for multiple myeloma trials and GLP‑1 label warnings. The PRV reinstatement restores a $150 million voucher market,...

BMS Beats Again Despite Eliquis and Cobenfy Disappointments

Bristol Myers Squibb posted fourth‑quarter 2025 sales of $12.5 billion, topping the $12.28 billion consensus. Eliquis generated $3.4 billion, falling short of expectations, while the new schizophrenia drug Cobenfy earned $51 million, missing forecasts by 3 %. Despite the miss, BMS forecasts 10‑15 % Eliquis growth...

Novartis Cuts 6 Early Cancer Candidates, Adds 2 to Refine Oncology Strategy

Novartis announced the removal of six Phase 1 oncology candidates—including KFA115, HRO761, MGY825, and AAA802—from its pipeline and introduced two new Phase 1 assets, AMO959 for prostate cancer and GCJ904 for solid tumors. The reshuffle aligns with a strategic review aimed at...

Stem Cell Specialist PrimeGen Takes SPAC Track to Nasdaq

PrimeGen US is set to go public on Nasdaq in 2026 by merging with SPAC DT Cloud Star Acquisition Corp., valuing the stem‑cell biotech at roughly $1.5 billion. The $69 million‑raised SPAC will provide capital to advance PrimeGen’s triple‑activated mesenchymal stem cell...

Programmable RNA 2.0: Beyond the First mRNA Revolution

BioSpace’s Denatureed podcast episode "Programmable RNA 2.0" spotlights the next wave of RNA therapeutics beyond the COVID‑19 mRNA vaccine success. Host Jennifer Smith‑Parker interviews Erik Digman Wiklund of Circio and Jacob Becraft of Strand Therapeutics about emerging platforms such as circular RNA...

3 Top Challenges Facing Regulatory Professionals Right Now

Regulatory professionals are grappling with three pressing challenges: vague FDA and EMA AI guidance, the mismatch between rapidly evolving AI models and static regulatory frameworks, and a constrained supply chain for radiopharmaceuticals. Experts stress that AI‑driven submissions must rest on...

Employers Warm Up to Remote Workers Again: BioSpace Report

Biopharma employers are warming to remote hiring again, with 28% saying they will recruit candidates regardless of location in 2026 – up from 20% in 2024 and 16% in 2023. The share of firms focused solely on local talent fell...

Sarepta Saga Has 'Gone on Too Long' As Competitors Catch Up

Sarepta Therapeutics’ one‑time gene therapy Elevidys, priced at $3.2 million, is under intense scrutiny after three patient deaths in 2025 and a steep 80% stock decline. The company’s three‑year efficacy data failed to reassure analysts, and quarterly sales missed expectations, leaving...

AbbVie’s I&I Portfolio Sells $30 Billion but Execs Again Underline Other Areas

AbbVie’s immunology portfolio generated roughly $30 billion in 2025, accounting for nearly half of its $61.1 billion total sales, driven by Skyrizi, Rinvoq and residual Humira revenue. Executives used the earnings call to spotlight under‑appreciated neuroscience and oncology assets, highlighting Parkinson’s drug...

Amgen Wants MariTide To Change Obesity Paradigm With Longer Dosing Periods

Amgen is positioning its investigational bispecific antibody‑peptide, MariTide, as a differentiated obesity therapy by offering monthly, bimonthly, or quarterly dosing without compromising efficacy. Phase II data revealed an average weight loss of up to 20 % after 52 weeks, and the company...

GSK Says No to GLP-1s, Prioritizes ‘Downstream Effects’ of Obesity

GSK’s new CEO Luke Miels announced the company will not pursue GLP‑1 obesity drugs, citing a crowded market and pipeline misalignment. Instead, GSK is concentrating on the downstream complications of obesity, particularly liver disease, after acquiring Boston Pharmaceuticals’ efimosfermin alfa...

Lilly’s GLP-1s Mounjaro, Zepbound Push Revenue up 46% in Q4

Eli Lilly reported fourth‑quarter revenue of $19.3 billion, driven by a 46 % surge in volume of its GLP‑1 drugs Mounjaro and Zepbound. Earnings per share rose to $7.54, comfortably beating the $6.91 consensus. The two drugs together generated $11.7 billion, lifting the company’s...

From Awareness to Acceleration: Rare Disease Drug Development Enters a Pivotal Era

Rare disease drug development is poised for rapid expansion, with the market projected to reach $400‑600 billion by the early‑to‑mid 2030s. Recent FDA guidances, including the plausible‑mechanism approval pathway, aim to accelerate cell and gene therapy approvals for ultrarare conditions. Executives...

2021 Sparked a Banner Year for Biotech IPOs. Where Are They Now?

In 2021 a pandemic‑driven gold rush produced 99 biotech IPOs that raised $15.6 billion, dwarfing the combined proceeds of 2023‑24. The surge created a “logical gap,” with many companies going public before their science was mature, leading to bankruptcies, pivots, and...

PrimeGen US to Merge with DT Cloud Star Acquisition Corp in $1.5B SPAC Deal

California-based biotech PrimeGen US announced a merger with SPAC DT Cloud Star Acquisition Corporation, valuing PrimeGen at approximately $1.5 billion in equity. The transaction, disclosed in a February 4 SEC filing, is expected to close in the second half of 2026 pending...

Novo Slides 14% as Early Q4 Results Predict Sales Decline for 2026

Novo Nordisk previewed its early Q4 results, showing a 5% sales decline forecast for 2026 driven by lower U.S. drug prices. The company beat analyst expectations in the quarter, with Ozempic and Wegovy outperforming by 9% and 3% respectively. Shares...

Pfizer’s Early Metsera Data Leaves Analysts Wanting More

Pfizer disclosed early Phase IIb data for its Metsera‑acquired obesity drug PF’3944, showing a 12.3% average weight loss at week 28 in the 3.2‑mg and 4.8‑mg monthly dosing arms. Analysts noted the placebo‑adjusted loss is roughly 12%, trailing Eli Lilly’s Zepbound 16%...

Merck Bats Away ‘Modest Growth’ Accusations, Touts Broad Pipeline

Merck met its 2025 sales guidance, reporting fourth‑quarter revenue of $16.4 billion, slightly above expectations. The company’s HPV vaccine Gardasil saw a 35% global decline, driven by weak demand in China and Japan. CEO Robert Davis countered modest‑growth criticism by highlighting...

BioNTech’s Multi-Modality Play Outpaces Moderna’s mRNA-Focused Pipeline

BioNTech is outpacing Moderna by expanding beyond mRNA into a multi‑modality oncology pipeline, highlighted by its $3.5 billion pumitamig partnership and several Phase III candidates. Moderna, still anchored to mRNA, has seen COVID‑19 vaccine revenues plunge from $18.4 billion in 2022 to $3.1 billion...

As Amgen and Lilly Recommit, Puerto Rico Seeks To Regain Manufacturing Momentum

Amgen and Eli Lilly have pledged more than $1.8 billion to expand their Puerto Rico facilities, with Amgen allocating $650 million to its Juncos biologics plant and Lilly committing over $1.2 billion to modernize its Carolina site for oral GLP‑1 production. These investments arrive as...

Moderna Partners with Recordati on Rare Disease mRNA Therapy in up to $160M Deal

Moderna and Italy’s Recordati announced a partnership to develop and commercialize mRNA‑3927 for propionic acidemia. Recordati will pay up to $160 million in upfront and milestone payments and receive royalties, while Moderna will lead clinical development and manufacturing and Recordati will...

Novo Nordisk Invests in Aspect Biosystems, Expands Partnership to Transfer Cell‑therapy Technologies

Novo Nordisk announced an expanded partnership with Aspect Biosystems, transferring its stem‑cell‑derived islet cell and hypoimmune cell‑engineering technologies. As part of the deal, Novo will make an equity investment and provide research funding to Aspect, though financial terms were not...

SpyGlass Pharma and AgomAb Therapeutics Launch Nasdaq IPOs

California‑based SpyGlass Pharma and New York‑based AgomAb Therapeutics announced the launch of their Nasdaq IPOs last week, joining the 2026 IPO class. Both firms have not disclosed the amount they expect to raise. SpyGlass will list on the Nasdaq Global Select...

Eli Lilly to Acquire Ventyx Biosciences for $1.2B

Eli Lilly announced a $1.2 billion cash acquisition of California‑based Ventyx Biosciences, gaining its oral drug pipeline targeting inflammatory diseases. The deal, expected to close in the first half of 2026 pending approvals, expands Lilly’s portfolio beyond its obesity drugs.

Amgen to Acquire Dark Blue Therapeutics for Up to $840M

Amgen announced on Tuesday that it will acquire England‑based Dark Blue Therapeutics in a deal valued at up to $840 million, aiming to bolster its oncology pipeline with Dark Blue’s pre‑clinical protein‑degradation assets. The acquisition will integrate Dark Blue into Amgen’s...

Corsera Raises $80M as First Preventative Heart Disease Program Moves Into Clinic

Cardiovascular biotech Corsera Health announced an $80 million Series A round co‑led by Forbion and Population Health Partners to advance its siRNA therapeutics, including the COR‑1004 program now entering Phase I trials. The funding will support the development of AI‑enabled tools for ASCVD...

Crinetics Pharmaceuticals Announces $350M Public Offering

Crinetics Pharmaceuticals announced a $350 million public offering of 7.6 million shares, with an underwriters’ option for an additional $52 million, to fund the development of its atumelnant drug after strong Phase II results that outperformed Neurocrine’s Crenessity. The offering was announced on Monday,...