(NASDAQ:BEAM) reported initial ... storage disease type Ia, according to a press release statement. The trial enrolled six patients who received a single intravenous infusion of BEAM-301 at doses of either 20 mg or 40 mg. Median follow-up was 7.6 months as of the August 17, 2026 data cutoff. The data was presented today at the European Society of Gene and Cell Therapy congress in ... (NASDAQ:BEAM) reported initial data from its Phase 1/2 clinical trial evaluating BEAM-301, an investigational treatment for glycogen storage disease type Ia, according to a press release statement. The trial enrolled six patients who received a single intravenous infusion of BEAM-301 at doses of either 20 mg or 40 mg. Median follow-up was 7.6 months as of the August 17, 2026 data cutoff. The data was presented today at the European Society of Gene and Cell Therapy congress in Hamburg, Germany. Beam reports early data on gene therapy for rare metabolic disease BEAM-301 is designed to correct the R83C mutation in the G6PC1 gene. This article was generated with the support of AI and reviewed by an editor.

Mereo BioPharma Group plc, a ... therapeutics for the treatment of oncology and rare diseases in the United Kingdom, the United States, and internationally. It develops Etigilimab (MPH-313), an antibody T-cell immunoreceptor with Ig and ITIM domains, which is in Phase 1b clinical trial for the treatment ... Mereo BioPharma Group plc, a biopharmaceutical company, develops and commercializes therapeutics for the treatment of oncology and rare diseases in the United Kingdom, the United States, and internationally. It develops Etigilimab (MPH-313), an antibody T-cell immunoreceptor with Ig and ITIM domains, which is in Phase 1b clinical trial for the treatment of tumors. Mereo BioPharma Group (NASDAQ:MREO – Get Free Report) and Ultragenyx Pharmaceutical (NASDAQ:RARE – Get Free Report) are both small-cap healthcare companies, but which is the superior investment? We will compare the two companies based on the strength of their earnings, profitability, dividends, analyst recommendations, valuation, institutional ownership and risk. The company is also developing Navicixizumab (OMP-305B83), which has completed Phase 1b clinical trials for the treatment of the late line ovarian cancer; Acumapimod (BCT-197), a p38 MAP kinase inhibitor that is in Phase II clinical trials to treat acute exacerbations of chronic obstructive pulmonary disease; and Leflutrozole (BGS-649), an oral aromatase inhibitor for the treatment of hypogonadotropic hypogonadism which is in Phase 2 trials. In addition, it develops rare disease product candidates, including Setrusumab (BPS-804), an antibody for the treatment of osteogenesis imperfecta; and Alvelestat (MPH-966), an oral small molecule that is in Phase II clinical trial to treat Alpha-1 anti-trypsin deficiency. The company's products candidatures include DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III.

The FDA has approved Genglycos ... gene therapy, to reduce cornstarch dependence in patients eight and older with GSDIa, the first approved treatment to target the disease's underlying genetic cause. ... The FDA has granted accelerated approval to pariglasgene brecaparvovec-opnr (Genglycos) for adults and pediatric patients eight years of age and older with glycogen storage disease type Ia (GSDIa), a rare inherited disorder caused by ... The FDA has approved Genglycos (pariglasgene brecaparvovec-opnr), a one-time AAV8 gene therapy, to reduce cornstarch dependence in patients eight and older with GSDIa, the first approved treatment to target the disease's underlying genetic cause. ... The FDA has granted accelerated approval to pariglasgene brecaparvovec-opnr (Genglycos) for adults and pediatric patients eight years of age and older with glycogen storage disease type Ia (GSDIa), a rare inherited disorder caused by deficiency of the enzyme glucose-6-phosphatase.¹ Genglycos, formerly known in development as DTX401, is indicated to reduce daily cornstarch intake as an adjunct to nutritional management and is the first approved therapy for the condition.¹,² Ultragenyx announces U.S. FDA approval of Genglycos gene therapy, the first-ever FDA-approved treatment designed to treat the underlying cause of glycogen storage disease type Ia (GSDIa). News release. Ultragenyx Pharmaceutical Inc. Published August 19, 2026. Accessed August 20, 2026. RARE rises on completion of rolling submission for AAV gene therapy. "This accelerated approval reflects our confidence in the clinical evidence to date and our commitment to bringing innovative treatments to patients with rare genetic diseases while we continue to gather data to confirm long-term benefit." — Megha Kaushal, MD, MSc, acting deputy director, CBER Office of Therapeutic Products, US Food and Drug Administration · Genglycos is a one-time, AAV8-based gene therapy administered as a single intravenous infusion, designed to deliver a functional G6PC gene to liver cells to restore glucose-6-phosphatase activity and stabilize blood sugar during fasting Serious adverse reactions reported across two clinical studies included anaphylaxis, adrenal insufficiency, elevated lactate levels, and hypoglycemia, and the most common adverse reactions were increased liver transaminases, nausea, headache, constipation, and hyperglycemia.¹ Genglycos-treated patients also showed a higher rate of hypertriglyceridemia than placebo-treated patients (29% versus 8%).¹ The prescribing information carries warnings for anaphylaxis, liver toxicity, adrenal insufficiency, and tumorigenicity risk, and the therapy should not be used during pregnancy.¹ "Today's approval is a great milestone in using a gene therapy to treat this disease and improve the quality of life for people with this condition," said Karim Mikhail, B Pharm, MS, acting director of the FDA's Center for Biologics Evaluation and Research.¹

Duchenne Cell Therapy Decision Slips to November After FDA Labels New Arm Function Data a Major Amendment · Hunter Syndrome Gene Therapy Halted by FDA After Spine Scans Show Small Masses in Five Children · Gene Therapy for Glycogen Storage Disease Type Ia Wins Accelerated Approval Priced at 2.7 Million Dollars · What are inborn errors of immunity? A group of more than 500 rare genetic disorders... Duchenne Cell Therapy Decision Slips to November After FDA Labels New Arm Function Data a Major Amendment · Hunter Syndrome Gene Therapy Halted by FDA After Spine Scans Show Small Masses in Five Children · Gene Therapy for Glycogen Storage Disease Type Ia Wins Accelerated Approval Priced at 2.7 Million Dollars · What are inborn errors of immunity? A group of more than 500 rare genetic disorders, also called primary immunodeficiencies, in which a faulty gene leaves the immune system unable to work properly. Mayo Clinic joined the federal AEGIS project to build CRISPR treatments for children with inherited immune disorders. Here is what is real. Mayo Clinic has signed on as one of three clinical sites for a federally funded project that aims to do something no gene therapy program has managed yet: build a single, reusable system to treat children with rare inherited immune disorders, rather than developing an expensive therapy for each mutation one at a time. The project is called AEGIS, short for Affordable Gene Editing Therapies for Immune System Diseases of Children. It is funded by an award of up to $27.7 million from the Advanced Research Projects Agency for Health, the federal agency created to pursue high-risk medical research, and it is part of the agency's THRIVE program, which targets hereditary rare diseases with in vivo genetic medicines.

The clearance of Genglycos for a rare glycogen storage disease gives Ultragenyx a sellable voucher and momentum ahead of an eagerly anticipated study readout.