We will continue to prioritize expense management and focus our investments on the execution of our upcoming commercial launches and advancing our phase three programs.

Feb 13, 2025 / receipt, needs a key / Howard Horn, CFO | earnings call 2025-02-13 | transcript 8e26868c-7051-4408-bc1e-f6263f7688bb | chars 12717-12885 | segment 12

First, perhaps for 111 for central liposyndrome, can you discuss some major topics that you plan to discuss with the FDA in the upcoming meeting? And what do you plan to achieve coming out of the meeting? Yeah, well, in the NPS3A program, our main focus of these discussions is on how to qualify epinephrine sulfate as a biomarker for accelerated approval. I point out to you we're also measuring clinical data and we're encouraged by the clinical data as well. I mean, the patients have continued to gain ground, gain development skills over time, and I think that this shows that the gene therapy is working. While we could potentially get approval by just following these patients clinically, Our hope is to be able to get the biomarker accepted. There's been a challenge with the agency. I think Peter Marks has publicly supported the use of the biomarker, including this biomarker at the recent MPS conference. But we have to get them through the details of how that's done. I think as a company, we're as well versed as anyone in how to explain the biomarker and how to analyze the results. But it is a situation where you have a neurodegenerative disorder with a relatively slower progression through multiple years. And therefore, the qualification takes a little more work to figure out and support. But I'd say everything I've seen in our program and multiple other clinical programs from other parties shows that these markers represent the underlying disease and their reduction through e

Nov 2, 2023 / receipt, needs a key / Thomas Yip, other | earnings call 2023-11-02 | transcript 83fb89db-44a8-47d5-88ef-6f59d33febab | chars 54725-56225 | segment 55

UltraCare Liaison | {"ats": "greenhouse", "city": null, "state": null, "job_id": "4732383005", "remote": true, "category": "other", "function": "other", "location": "Germany - Remote", "job_title": "U

company_statement / Sep 11, 2026 / receipt

Director, Development AI Workflow Transformation Lead

company_statement / Sep 2, 2026 / receipt

Director, Development AI Workflow Transformation Lead | {"ats": "greenhouse", "city": null, "state": null, "job_id": "4728300005", "remote": true, "category": "other", "function": "data", "location":

company_statement / Sep 2, 2026 / receipt

Associate Director, Business Development | {"ats": "greenhouse", "city": "Somerville", "state": "MA", "job_id": "4728292005", "remote": false, "category": "sales_expansion", "function": "sales", "loca

company_statement / Sep 1, 2026 / receipt

Senior Clinical Trial Manager | {"ats": "greenhouse", "city": null, "state": null, "job_id": "4726626005", "remote": true, "category": "other", "function": "clinical", "location": "United States - Rem

company_statement / Aug 31, 2026 / receipt

Senior Specialist, Quality Assurance Operations | {"ats": "greenhouse", "city": "Bedford", "state": "MA", "job_id": "4727955005", "remote": false, "category": "other", "function": "operations", "locat

company_statement / Aug 31, 2026 / receipt

Senior Litigation Paralegal, eDiscovery & AI

company_statement / Aug 27, 2026 / receipt

Senior Litigation Paralegal, eDiscovery & AI | {"ats": "greenhouse", "city": "Bedford, MA; Miami, FL; Novato, CA; Somerville, MA; Woburn", "state": "MA", "job_id": "4726658005", "remote": false, "cate

company_statement / Aug 27, 2026 / receipt

Rare Disease, Field Sales (Florida) | {"ats": "greenhouse", "city": null, "state": null, "job_id": "4727058005", "remote": true, "category": "sales_expansion", "function": "sales", "location": "Miami,

company_statement / Aug 27, 2026 / receipt

Associate Director, Regulatory Operations, EMEA | {"ats": "greenhouse", "city": null, "state": null, "job_id": "4727895005", "remote": false, "category": "other", "function": "legal", "location": "Bas

company_statement / Aug 27, 2026 / receipt

Associate Director Supply Chain - Gene Therapy | {"ats": "greenhouse", "city": null, "state": null, "job_id": "4726956005", "remote": false, "category": "other", "function": "operations", "location":

company_statement / Aug 26, 2026 / receipt

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company_statement / Aug 24, 2026 / receipt