The Investor Relations website contains information about Ultragenyx Pharmaceutical Inc.'s business for stockholders, potential investors, and financial analysts. “As our first gene therapy approval, GENGLYCOS represents an important achievement for our company and the realization of the promise of a powerful new tool to deliver transformative medicines for people living with rare diseases.” · GSDIa is an ultra-rare genetic metabolic disorder caused by a deficiency of the enzyme needed to release glucose from the liver to the bloodstream. About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. About Glycogen Storage Disease Type Ia (GSDIa) GSDIa is an ultra-rare, serious, and life-threatening disease due to an inborn error of carbohydrate metabolism caused by pathogenic variants of the G6PC gene, which encodes G6Pase, an enzyme that is critical for the release of glucose from glycogen and other metabolic sources. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

GENGLYCOS is the first gene therapy approval, and fifth FDA approval overall, for the company Approval provides a long-awaited first-ever option to reduce... “As our first gene therapy approval, GENGLYCOS represents an important achievement for our company and the realization of the promise of a powerful new tool to deliver transformative medicines for people living with rare diseases.” · GSDIa is an ultra-rare genetic metabolic disorder caused by a deficiency of the enzyme needed to release glucose from the liver to the bloodstream. About Ultragenyx Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. About Glycogen Storage Disease Type Ia (GSDIa) GSDIa is an ultra-rare, serious, and life-threatening disease due to an inborn error of carbohydrate metabolism caused by pathogenic variants of the G6PC gene, which encodes G6Pase, an enzyme that is critical for the release of glucose from glycogen and other metabolic sources. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

CSL Canada is proud to recognize this important milestone of the first Canadian patient to receive HEMGENIX® , the one-time gene therapy treatment for eligible patients living with hemophilia B, at... This milestone marks a significant advancement in hemophilia B care, reflecting how clinical innovation and health system collaboration are enabling real-world access to gene therapy for eligible patients in ... Hemophilia B is a rare genetic bleeding disorder caused by a deficiency of clotting Factor IX, which prevents blood from clotting properly, often resulting in a lifelong dependence on medication to manage bleeding.2 People living with hemophilia B may experience spontaneous or prolonged bleeding episodes that can lead to joint damage, chronic pain, reduced mobility, and other long-term complications that affect quality of life.3 "Gene therapy introduces a new approach to treatment, and delivering it requires close coordination across clinical teams and systems. It's encouraging to see this level of readiness translating into real-world care for eligible patients." CSL Canada continues to advance innovation and is working to help ensure all eligible Canadians can access and benefit from advances in hemophilia care and innovation. About Hemophilia B Hemophilia B is a life-threatening rare disease. This milestone marks the first HEMGENIX® treatment administered to a Canadian patient outside of a clinical trial, demonstrating real‑world access to gene therapy in People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage.6 The constant worry of a bleed means that their daily activities can be restricted, even for things as simple as going up and down stairs.6-8 Current treatments for moderate to severe hemophilia B include life-long prophylactic infusions of Factor IX to temporarily replace or supplement low levels of the blood-clotting factor.6 Many people with hemophilia find themselves continually confronted with the mental and emotional impact of managin

Both Seaport Therapeutics and Hemab Therapeutics are hoping to bring in around $180 million each from their public listings this week, in the latest sign that biotech IPOs are regaining momentum. Both Seaport and Hemab are hoping to bring in around $180 million from their listings, in the latest sign that biotech IPOs are regaining momentum. The biotech has already tested sutacimig in Glanzmann thrombasthenia, a rare genetic bleeding disorder caused by a deficiency of the platelet integrin alpha IIb beta3, which prevents blood from properly clotting. Hemab has previously pointed to phase 2 study as demonstrating that sutacimig achieved clinically meaningful bleeding reduction in these patients. The IPO funds would help finance a phase 3 study in Glanzmann thrombasthenia, as well as an ongoing phase 2 trial in another bleeding disorder called Factor VII deficiency. Should the final share price fall in the middle of this range, the biotech expects the IPO to bring in $183.5 million in net proceeds—rising to $211.4 million if underwriters fully take up their 30-day offer to buy an additional 1.77 million shares at the same price. The biotech employs 58 people and was originally assembled by PureTech Health—where Zohar was previously CEO—and supplied with $100 million when it launched in the spring of 2024. Seaport followed that up with a $225 million series B a few months later. Out of the $325 million raised to date, Seaport entered 2026 with a hefty chunk of funds—$233.7 million, to be precise—still intact. When combining these funds with the IPO proceeds, Seaport said today that it has earmarked $121 million to advance its lead depression candidate SPT-300, also known as GlyphAllo.