SNAPSHOT

Sanofi’s rare disease drug sees the light at the end of the tunnel, but only for Gaucher disease

Sanofi said its experimental drug venglustat succeeded in a Phase 3 trial for a neurological form of Gaucher disease but failed a separate Phase 3 study in Fabry disease, going “one-for-two” in rare disease trials.

Why it matters: The results could revive a drug that has struggled for years and potentially give Gaucher patients a long-awaited treatment option for brain-related symptoms that existing therapies don’t address.

Backstory: Sanofi inherited venglustat through its acquisition of Genzyme more than a decade ago and has since tested the drug across multiple diseases. Designed to reduce toxic substrate buildup inside cells, venglustat has previously failed trials in Parkinson’s disease, a rare kidney disorder, and GM2 gangliosidosis.

Big picture: Rare disease drugs are central to Sanofi’s strategy, but recent pipeline disappointments have raised pressure on its R&D organization. A regulatory filing for Gaucher could help offset broader concerns about the company’s late-stage portfolio.

Zoom in: In Type 3 Gaucher disease, a rare form that causes progressive neurological damage, venglustat met its primary endpoint and three of four key secondary endpoints. While it came with side effects such as nausea, diarrhea and spleen enlargement, venglusat was generally considered well tolerated. In Fabry disease, venglustat missed its primary endpoint, showing no meaningful improvement in patient-reported pain compared with the placebo.

What’s next: Sanofi plans to submit the Gaucher data to global regulators and will present detailed results at a medical meeting this week. A second Phase 3 Fabry study is still ongoing, with additional analyses planned. If approved, venglusat would be a daily oral pill, a potential breakthrough for Gaucher patients whose neurological symptoms aren’t helped by enzyme replacement therapies like Sanofi´s Cerezyme, and a rare late-stage win after years of clinical setbacks.

SNIPPETS

What’s happening in biotech today?

🧬 RNA revival: Roche is strengthening its renewed focus on RNA therapeutics through a deal with SanegeneBio that includes a $200M upfront payment and up to $1.5B in potential milestones for a worldwide license to an undisclosed RNA interference program. Sanegene will handle early development, after which Roche’s Genentech unit will lead clinical development and commercialization. The agreement builds on growing Big Pharma interest in Sanegene’s RNAi platform, following a recent $1.2B pact with Eli Lilly. Roche’s move continues its gradual return to RNA after exiting the field in 2010 and reentering via acquisitions and licensing deals.

🤝 Degrader deal: Ipsen has entered a global collaboration and option agreement with Origami Therapeutics to advance a small-molecule protein degrader program for a rare inherited neurodegenerative disorder. Origami will lead the research phase using its Oricision platform, while Ipsen holds an exclusive option to license the program worldwide once a candidate is selected, at which point Ipsen would assume clinical development and commercialization. Origami is eligible for upfront payments, milestones, and royalties. The deal expands Ipsen’s early neuroscience pipeline and reflects growing interest in protein degradation as a disease-modifying approach in neurology, where toxic protein accumulation drives progressive, largely untreatable conditions.

🛑 ADC stunted: Daiichi Sankyo is facing renewed challenges in its antibody-drug conjugate (ADC) pipeline, halting internal development of its CLDN6-directed ADC, DS-9606, after a portfolio review while leaving the door open to partnering the asset. At the same time, the company has again delayed a key phase 3 readout for Datroway, pushing top-line data from the Avanzar trial in first-line non-small cell lung cancer to the second half of 2026 due to slower event accumulation. Additional complications include biomarker-driven trial redesigns, halted patient recruitment in one adjuvant study, and a recently lifted FDA clinical hold on another ADC following safety concerns.

🌍 Global grab: China-based Newsoara has expanded its collaboration with vTv Therapeutics by paying $20M upfront to secure global rights outside its previously licensed territories to HPP737, an oral PDE4 inhibitor in phase 1 development for psoriasis. The agreement includes up to $115M in potential development and sales milestones for vTv, plus tiered royalties. HPP737 is positioned as a differentiated PDE4 inhibitor that may offer improved tolerability compared with existing therapies. The deal builds on a regional licensing agreement signed in 2018 and highlights sustained industry interest in PDE4 inhibitors across inflammatory and respiratory diseases.

💰 Capital confidence: Early-stage investor Santé Ventures has closed its fifth and largest fund to date, raising $330M to invest across biotechnology, medtech and digital health. The Austin-based firm will continue focusing primarily on early-stage companies, particularly those addressing clinically complex areas, capital inefficiencies or outdated care models, while applying a hands-on investment approach. The fund surpasses Santé’s original $300M target and comes amid signs of a stabilizing biotech financing environment in 2026.

SNAP AGAIN

GSK hands back RNA editing frontrunner, leaving Wave to go it alone

GSK has returned global rights to WVE-006, the most advanced RNA editing drug in clinical testing, allowing Wave Life Sciences to independently advance the therapy for alpha-1 antitrypsin deficiency (AATD). Despite the setback, GSK and Wave’s broader collaboration remains intact, with up to $2.8B still at stake.

Why it matters: The move reshapes one of the most closely watched programs in RNA editing, a still-unproven but highly promising drug modality, and puts pressure on Wave to show the drug can deliver transformative benefits on its own.

Backstory: GSK gained rights to WVE-006 through a broad 2022 collaboration with Wave and kept them through early clinical readouts. On Monday, Wave said the companies agreed the biotech is better positioned to rapidly develop a rare disease therapy, while GSK’s respiratory strategy prioritizes large, mass-market conditions.

Big picture: RNA editing is viewed as a potentially safer and more flexible alternative to DNA editing, but no such therapy has yet been approved. WVE-006, the first RNA editing drug to reach human testing, has become a key test case for whether the technology can live up to its promise. Unfortunately, GSK exited before the end of March data readout and so will not be able to benefit from any positive results.

Zoom in: WVE-006 targets AATD, a rare liver and lung disease caused by the inability to produce a functional protein. Wave has reported data from multiple dose levels and repeat dosing showing that patients can produce the missing protein. The company argues the drug could represent a “functional cure,” but investors have been seeking stronger effects. Skepticism has grown after rival Korro Bio dropped its own RNA editing AATD program due to a lack of potency.

What’s next: Wave plans to meet with U.S. regulators to discuss accelerated approval requirements, with feedback expected midyear. The company says it is accelerating its regulatory strategy and has a cash runway into the third quarter of 2028. Analysts note GSK’s decision may reflect competitive pressure from Beam Therapeutics, which recently aligned with regulators on a fast approval pathway for its gene editing AATD drug.

TOUR OPERATOR

Upcoming events

• 🇳🇱 Amsterdam, 3-4 March 2026 - BioCapital

• 🇪🇸 Barcelona, 10-12 March 2026 - Bioprocessing Summit Europe

• 🇳🇱 Utrecht, 26 March 2026 - Innovation for Health

• 🇦🇹 Vienna, 27-30 March 2026 - BioProcess International

• 🇩🇪 Munich, 17-21 April - ESCMID Global

• 🇺🇸 San Diego, 17-22 April 2026 - AACR Annual Meeting

• 🇩🇪 Leipzig, 21-22 April 2026 - German Biotech Days

• 🇨🇳 Shanghai, 28-29 April 2026 - ChinaBio

• 🇸🇦 Riyadh, 11-13 May 2026 - BIO Middle East

• 🇩🇪 Berlin, 09 – 11 June 2026 - bio:cap