Neuromuscular Disorders

Business

Korro Bio Unveils RNA-Editing Therapy KRRO-121, Aiming to Redefine Ammonia Control for Rare Metabolic and Liver Disorders

At its Analyst Day, Korro Bio detailed its lead candidate KRRO-121, an RNA-editing therapeutic designed to tackle hyperammonemia in urea cycle disorders and hepatic encephalopathy by stabilizing a key liver enzyme, positioning it as a potential paradigm shift from current burdensome treatments.

Jan 30, 2026

Business

Rhythm Pharmaceuticals: The Under-the-Radar Biotech Outpacing Tech Giants

While investors chase AI and big tech, a niche biotech firm focused on rare genetic obesity disorders has quietly delivered staggering returns, with Wall Street seeing further 40% upside. Rhythm Pharmaceuticals' targeted approach and upcoming catalysts have analysts bullish, despite its relative anonymity.

Feb 2, 2026

Business

Sanofi's Pipeline Gains Momentum with Key Phase 3 Successes in Rare Disease and Immunology

Sanofi reports a pivotal Phase 3 win for its investigational therapy venglustat in a rare neurological disorder, alongside positive EU regulatory news for Rezurock and progress for amlitelimab, signaling a strategic shift toward high-value specialty medicines.

Feb 2, 2026

Business

Quince Therapeutics Plummets After Late-Stage Trial of A-T Therapy Fails to Hit Targets

Quince Therapeutics' lead candidate, eDSP, has failed to meet primary and secondary endpoints in its pivotal Phase III NEAT trial for the rare neurological disorder Ataxia-Telangiectasia (A-T), triggering a catastrophic drop in the company's share price.

Feb 2, 2026

Business

Harmony CFO's Complete Stock Sale Raises Eyebrows Amid Upbeat Revenue Forecasts

Harmony Biosciences CFO Sandip Kapadia has liquidated his entire direct stake in the company, a move that contrasts sharply with the firm's robust financial performance and bullish $1 billion revenue target for its flagship drug WAKIX by 2026. While the sales were pre-planned, the timing fuels debate on insider confidence versus corporate optimism.

Feb 2, 2026

Sanofi's Genetic Disorder Drug Shows Promise in Gaucher Trial, Stumbles in Fabry Study

Sanofi's experimental oral drug venglustat demonstrated significant neurological benefits for patients with Type 3 Gaucher disease in a late-stage trial, but failed to meet its primary endpoint in a separate study for Fabry disease, casting uncertainty on its regulatory path.

Feb 2, 2026

Business

Navigating Market Volatility: Three ASX Penny Stocks With Potential for February 2026

As the ASX braces for a turbulent week amid rate hike fears, investors are scouring the market for resilient opportunities. We delve into three penny stocks—spanning energy, retail, and healthcare—that combine financial stability with distinct growth narratives for the discerning investor.

Feb 2, 2026

Business

Sarepta's Gene Therapy Shows Sustained Slowing of DMD Progression in Landmark Three-Year Data

New long-term results from the pivotal EMBARK trial indicate ELEVIDYS, Sarepta's gene therapy, continues to alter the disease trajectory for boys with Duchenne muscular dystrophy, with no new safety concerns emerging over three years.

Jan 31, 2026

Business

Citi Lifts Bristol-Myers Squibb Price Target, Citing Improved Biopharma Outlook Through 2026

Citi analyst Geoff Meacham raised his price target on Bristol-Myers Squibb (BMY) to $60, citing a more favorable setup for the sector. The move comes as the drugmaker also announces a partnership with Microsoft to leverage AI for earlier lung cancer detection.

Jan 30, 2026

Business

ResMed Posts Strong Q4 on Mask Innovation and Digital Health Surge, Beats Estimates

Medical device maker ResMed (RMD) reported fourth-quarter revenue of $1.42 billion, an 11% year-over-year increase that surpassed Wall Street forecasts. Non-GAAP earnings per share reached $2.81, also topping analyst expectations.

Jan 30, 2026

Business

Analysts Bullish on Ovid Therapeutics as Pipeline Progresses, Penny Stock Gains Attention

Ovid Therapeutics (NASDAQ: OVID) has garnered analyst optimism following positive Phase 1 trial results for its novel neurological drug candidate and a recent 'Outperform' initiation. The biotech firm is now shifting focus to a more potent oral therapy, with key regulatory milestones ahead.

Jan 30, 2026

Business

Novo Nordisk Pivots: Oral Wegovy Launch, Job Cuts, and a Cell Therapy Bet Reshape Its Future

Novo Nordisk charts a bold new course, launching a pivotal oral obesity drug while restructuring its workforce and deepening its investment in next-generation cell therapies for metabolic diseases.

Jan 30, 2026

Business

PTC Therapeutics Gains Analyst Favor: Barclays Upgrade Fuels Valuation Debate Amid Rare Disease Focus

Barclays' upgrade of PTC Therapeutics to Overweight has spotlighted the biotech firm's valuation, with shares showing strong momentum but questions lingering over its path to profitability and reliance on a narrow drug portfolio.

Jan 30, 2026

Business

Sanofi Bolsters Pipeline with Atopic Dermatitis Win and Genetic Therapy Foray

Sanofi announces promising late-stage results for its immunology candidate amlitelimab and expands into genetic therapies via an investment in Sensorion, signaling a strategic push into high-value therapeutic areas.

Jan 30, 2026

Business

Regeneron Aims to Redefine Obesity Treatment with Dual-Action Drug Targeting Weight and Cholesterol

Regeneron Pharmaceuticals is positioning its experimental obesity drug, olatorepatide, as a potential game-changer by combining significant weight loss with a dramatic reduction in 'bad' cholesterol. The company believes this dual benefit could carve out a unique niche in the competitive $150 billion market, currently led by Novo Nordisk and Eli Lilly.

Jan 30, 2026

Business

Beyond GLP-1: Eli Lilly Diversifies Pipeline with Autoimmune, Gene Editing Deals and Cancer Therapy Win

Eli Lilly bolsters its long-term growth strategy through two new R&D partnerships and a key regulatory milestone for an ovarian cancer drug, signaling a strategic push beyond its blockbuster diabetes and obesity treatments.

Jan 30, 2026

Business

Cathie Wood Sees Buying Opportunity in Market Dip, Adds to Stakes in Joby, Kodiak AI, and CRISPR

ARK Invest's founder capitalized on recent market weakness to increase holdings in three of her flagship disruptive innovation picks, signaling continued conviction despite sector volatility.

Jan 30, 2026

Business

Lilly Bets Big on Repertoire's Immune Decoding Tech in Up to $1.9B Autoimmune Pact

Eli Lilly commits $85 million upfront and over $1.8 billion in potential milestones to partner with Repertoire Immune Medicines, aiming to develop a new class of 'tolerizing' therapies that reset the immune system without broad suppression.

Jan 30, 2026

Business

Bank of America Lifts Target on Xencor Amid Broader Biotech Sector Optimism

Bank of America has raised its price target for clinical-stage biopharma firm Xencor (XNCR), highlighting a cautiously improving landscape for the sector driven by data catalysts, M&A, and capital access.

Jan 30, 2026