South Carolina’s life sciences industry is the fastest-growing in the state. Keeping that momentum will require more than recruiting new talent. It will require strong partnerships between industry, education and workforce development organizations. Helping lead that effort is the South Carolina Technical College System (SCTCS), connecting employers with the talent and training needed to support continued growth.

SCbio’s Midlands Technical College Roundtable on Aug. 27 at their Northeast Campus in Columbia highlighted what that partnership can look like in practice. The discussion brought together leadership from the college and regional life sciences companies to explore workforce challenges and the resources available to help address them.

Three industry needs emerged: strengthening the entry-level talent pipeline, creating more opportunities to upskill existing workers and developing structured internship pathways that can help students transition into full-time careers.

South Carolina’s technical colleges are uniquely positioned to help meet all three.

Here’s how.

SCTCS comprises 16 technical colleges strategically located across the state, putting a campus within a 30-minute drive of every South Carolinian. The colleges offer two-year degree programs connected to life sciences careers, along with industry-specific training that helps employers develop the skills their workforce needs.

That makes the technical college system more than an educational resource. It is a workforce partner that can help companies develop new talent while strengthening the skills of their existing employees.

The system also offers programs designed to make workforce development more accessible and affordable for South Carolina companies.

• readySC supports companies moving to or expanding in South Carolina with recruiting, assessment, training development, management and hiring implementation at no charge.

• retrainSC provides qualifying companies with a rebate for eligible employee training. The program offers a 1:1 match on eligible training costs, with companies able to receive up to $1,000 per eligible employee each year. Eligible training can include upskilling, management development, recertification, new technology, new processes and more.

• Apprenticeship Carolina works alongside companies to establish federally recognized apprenticeship programs, helping employers build long-term talent pipelines.  

The value of these programs was clear during the roundtable. Anthony White of The Ritedose Corporation put it plainly: “If you are in this room and have not taken advantage of any of these programs, you are missing out on a lot of money.”

And he’s right. These programs can help companies offset the cost of training today while building the skills and talent that are needed for future growth.

In addition, workforce development is about more than filling an open position. It’s about creating a sustainable talent pipeline that grows alongside South Carolina’s life sciences ecosystem.

South Carolina’s technical colleges create earlier connections between students and careers through dual-enrollment programs, allowing high school students to earn college credit while exploring pathways into the workforce.

The Midlands Technical College Roundtable reinforced a simple point: South Carolina already has much of the workforce infrastructure its growing life sciences industry needs. What matters now is connecting more companies, students and workers to these resources.

As the Palmetto State continues to strengthen its position as a life sciences hub, SCTCS will be an essential partner in preparing the people who will drive that growth forward.

Biotech cohort powered by SCbio targets ALS, Alzheimer’s, and rare pediatric disease, alongside programs in liver cancer and obesity

Techbio cohort powered by MassBio spans generative design, causal inference, and virtual cell modeling, alongside therapeutic programs in oncology and neuroscience

CHARLESTON, S.C. and CAMBRIDGE, M.A. (September 1, 2026) —SCbio, in collaboration with MassBio, today announced the 10 companies selected for the Fall 2026 cohorts of Drive, an accelerator designed to propel breakthrough science while equipping scientific founders with the tools for long-term success. As part of a strategy to resource emerging startups wherever they are based, Drive will feature two parallel cohorts this fall: biotech, powered by SCbio, and techbio, powered by MassBio.

The biotech cohort takes on ALS, Alzheimer’s disease, a rare pediatric neurological disorder, liver cancer and obesity. The techbio cohort combines computational platforms and therapeutic programs, with targets that include glioblastoma, pancreatic cancer, Parkinson’s disease and drug resistance, and methods that include generative protein design, causal inference and virtual cell modeling. Across both, the 10 companies span nearly every major modality in drug development, from small molecules and antibodies to gene editing, gene therapy, oncolytic viruses and cell therapy.

“Drive is the catalyst for these companies with therapies that will change the quality of life for the next generation of patients,” said James Chappell, Ph.D., president and CEO of SCbio. “The founders in this biotech cohort are working to solve some of the hardest problems in neuroscience, metabolic disease and cancer. We, and our experts, are ready to help get their treatments to those who need them most.”

“This cohort is tackling some of the most difficult challenges in medicine, from glioblastoma and pancreatic cancer to Parkinson’s disease. These are areas where progress has been hard-won, and these techbio founders are bringing new approaches to both the technology and therapeutics needed to address them,” said Kendalle Burlin O’Connell, CEO & president of MassBio. “Pre-seed and seed are the hardest dollars in biotech to raise right now, and good science stalls waiting for them. Drive exists to get these companies to their next milestone.”

Biotech Cohort (SCbio)

Techbio Cohort (MassBio)

More details about each company and the teams involved can be found on the Drive website.

Over an eight-week hybrid program running September 14 through November 6, these startups will participate at no cost in six expert-led, industry-specific curriculum modules, weekly sessions with a tailored team of mentors, and networking opportunities across the life sciences ecosystem. Curriculum experts for SCbio’s biotech track include Eli Lilly and Company, Maynard Nexsen, Novartis, The Matchstick Group and VentureSouth, with premier sponsorship from Charleston County Economic Development and the South Carolina Department of Commerce. Curriculum experts for MassBio’s techbio track include Citrin Cooperman, F-Prime Capital, Thermo Fisher Scientific, Voisin Consulting Life Sciences, and Wilson Sonsini, with SandboxAQ serving as premier sponsor and Mispro as supporting sponsor. Participants will also receive in-kind prizes from Premier Sponsors specific to SCbio and MassBio. Through a partnership with the ADA Forsyth Institute, one graduate of the fall cohort will receive a sponsored lab bench for one year at the nonprofit research organization’s Somerville facility, with all other graduates eligible for a discounted rate.

Both cohorts will present at MassBio’s Align Summit in Boston on October 15, putting their new pitch decks to use in front of an audience of potential funders.

Since launching in 2022, Drive has supported 70 early-stage companies, 80 inclusive of the current cohorts, that have raised $137 million in funding and created nearly 100 jobs after completing the program.

# # #

About SCbio

SCbio is the definitive, unifying leader in South Carolina’s life sciences sector, fostering authentic collaboration and innovation across academia, startups, government and established enterprises. As a member-driven economic development organization, SCbio represents an industry that generates $20.1 billion in annual economic impact and supports 63,000 jobs across South Carolina. Our members are unified by a mission to positively impact humanity through science across the ecosystem of advanced and specialty manufacturing; digital health and the data frontier; and cutting-edge health specializations. For additional information about SCbio and the life sciences industry, or to become a member, visit SCbio.org.

About MassBio

MassBio is the driving force behind Massachusetts’s life sciences ecosystem, supporting innovation and industry growth by offering best-in-class resources to over 1,700 member organizations at all stages of the biopharma lifecycle. Founded in 1985, MassBio aspires to extend Massachusetts’s impact as the global center of excellence in biomedical breakthroughs. Through strategic cost-saving initiatives, robust business partnerships, educational and networking opportunities, and proactive advocacy, MassBio empowers its members to launch the next generation of medical advancements to deliver the cures and therapies that enhance patient lives.

New device can help people with diabetes detect warning signs of a dangerous complication before it becomes a medical emergency

For Immediate Release: August 25, 2026

The U.S. Food and Drug Administration authorized the Libre Duo 10 Day Continuous Dual Glucose Ketone Monitoring System for people aged 2 years and older living with diabetes. The Libre Duo 10 Day is the first wearable device in the U.S. that continuously monitors ketone levels, and the first in the world to continuously monitor both ketones and blood sugar (glucose) together in a single device.

“Today’s authorization is a breakthrough for the safety of children and adults living with diabetes,” said Michelle Tarver, M.D., Ph.D., Director of the FDA’s Center for Devices and Radiological Health. “Knowing that ketone levels are rising, and having that information in real time, around the clock, can be the difference between early intervention and a life-threatening emergency.”

According to the Centers for Disease Control and Prevention, an estimated 40.1 million Americans are living with diabetes, a condition that requires careful monitoring of blood sugar levels. For the estimated 2.1 million Americans with type 1 diabetes, blood sugar is only part of the picture. When the body produces too much ketone, which is made by the body when it is using fat instead of glucose for energy, it can lead to diabetic ketoacidosis, or DKA — a serious complication that can develop quickly and become life-threatening if left untreated.

Early detection of rising ketone levels is critical. Until now, people who needed to monitor ketones had to do so separately, using tests that could only provide a single measurement at one point in time which could not tell users if ketones were rising or falling. The Libre Duo 10 Day addresses that gap by continuously measuring both ketone and glucose levels in the fluid just beneath the skin, every minute, day and night. Readings are sent wirelessly to a compatible smartphone, where users can see their current ketone and glucose levels and whether they are trending up or down. The device can send automatic alerts if ketone levels reach a concerning threshold, which may help patients and caregivers recognize rising ketones before DKA becomes a medical emergency. Ketone information should always be interpreted in the context of glucose readings and symptoms.

The FDA’s authorization is based on a comprehensive review of clinical and performance data submitted by the device sponsor. The clinical program included data from six clinical studies that together enrolled more than 600 participants aged 2 years and older to evaluate the accuracy of the device’s ketone and glucose monitoring functionality across a range of ketone and glucose levels. The results demonstrated that the device accurately tracked clinically meaningful differences in ketone levels across its 10-day wear period, including identifying elevated ketone levels before the onset of diabetic ketoacidosis.

This authorization is also aligned with the FDA’s Home as a Health Care Hub Initiative, which focuses on advancing the development of innovative, patient-centered devices that fit more seamlessly into people’s daily lives at home.

Prior to authorization, the FDA granted Breakthrough Device designation for the Libre Duo 10 Day Continuous Dual Glucose Ketone Monitoring System. A breakthrough designation is meant to expedite the development and review of devices that provide for more effective treatment or diagnosis of life-threatening or irreversibly debilitating diseases or conditions.

The FDA granted marketing authorization to Abbott Diabetes Care for the Libre Duo 10 Day through the De Novo premarket review pathway, a regulatory pathway for low- to moderate-risk devices of a new type. Along with this De Novo authorization, the FDA is establishing special controls that define requirements including those related to labeling and performance testing. When met, the special controls, in combination with general controls, provide a reasonable assurance of safety and effectiveness.

Media:
FDA Request for Comment
202-690-6343

Consumer:
888-INFO-FDA

###

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products.

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 the burden of care associated with GSDIa

Ultragenyx received a Priority Review Voucher upon approval

NOVATO, Calif., August 2026 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the U.S. Food and Drug Administration (FDA) granted accelerated approval for GENGLYCOS™ (pariglasgene brecaparvovec-opnr), also known as DTX401, in adult and pediatric patients eight years and older with glycogen storage disease type Ia (GSDIa).

“The approval of GENGLYCOS fulfills our commitment to provide the first therapy that directly targets the root cause of GSDIa. The reduced reliance on cornstarch, experienced by patients in our clinical studies, demonstrates this gene therapy’s ability to establish the normal breakdown of glycogen to produce glucose during fasting or episodes of metabolic stress. This ability to regulate glucose has alleviated the disease burden and has the potential to mitigate the risk of severe or life-threatening hypoglycemia for these patients,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “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. The deficiency reduces the liver’s ability to control glucose levels and is associated with potentially life-threatening hypoglycemia episodes and other serious complications, requiring rigorous nutritional management that involves a burdensome, around-the-clock regimen of raw cornstarch intake as an oral glucose replacement therapy. Glucose control with cornstarch is crude with large swings in glucose, and patients instead end up spending a large fraction of their day significantly hyperglycemic to avoid hypoglycemic episodes. GSDIa affects 1,500-2,500 patients in the U.S. and 6,000-8,000 worldwide within commercially accessible geographies.

“Day-to-day management of GSDIa requires a relentless regimen of raw cornstarch and strict dietary management that can be extraordinarily demanding for patients and families. Even with meticulous adherence to this regimen, patients must be perfect. Any missed cornstarch puts patients at risk of severe hypoglycemia, seizures, and even death,” said David Weinstein, M.D., MMSc, one of the world’s leading GSDIa experts. “The approval of GENGLYCOS represents a major step forward for the GSDIa community and reflects almost 30 years of work and scientific progress aimed at improving safety and the quality of life of people living with this disease.”

“For families affected by GSDIa, every day revolves around strict schedules, overnight vigilance, and the constant worry that a missed meal or dose of cornstarch could trigger life-threatening hypoglycemia,” said David and Wendy Feldman, co-founders and current Board members at The Children’s Fund for Glycogen Storage Disease Research. “This approval is an incredibly meaningful milestone for a community that has spent decades hoping, advocating, and helping advance the research for new treatment options that could ease the burdens of this disease.” 

Clinical Program and Post-Marketing Study Requirements Supporting Accelerated Approval of GENGLYCOS

The approval of GENGLYCOS is based on positive data from the 48-week randomized, double-blind, placebo-controlled Phase 3 GlucoGene study which treated 46 participants aged eight years and older with DTX401 (1.0 x 10^13 GC/kg dose) or placebo, showing a reduction in the cornstarch requirements in the treated group (p<0.001). There were 44 participants in the modified intention-to-treat (mITT) population providing efficacy data within the Week 48 analysis period following treatment with DTX401 (n=20) or placebo (n=24). At Week 48, eligible participants crossed over and received the alternate treatment. After crossover, participants continued to be followed, with analyses conducted at Week 96 and Week 144.

As part of accelerated approval, Ultragenyx has agreed to provide two years of safety and efficacy clinical data from open-label commercial treatment of 50 patients and 20 control patients through enhancement of its existing GSDIa Disease Monitoring Program (DMP). The control group will consist of patients who sought commercial treatment but cannot be treated with GENGLYCOS due to the presence of anti-AAV8 antibodies. The study will provide more data to support the reduction in cornstarch clinical burden, fasting tolerance, and other measures in a post-marketing setting where patients can know their immediate glucose levels, and their cornstarch and diet can be managed more promptly by their physician. The DMP will also evaluate previously treated clinical trial participants as well as these new commercial patients for a total of 10 years.  

Enabling Access for Eligible Patients

Ultragenyx will provide support to help enrolled patients and caregivers navigate access to treatment through its UltraCare^® program, which now includes specially trained UltraCare^® Gene Therapy Guides to help understand insurance coverage, assist in obtaining treatment support, and answer questions about the treatment process. Dedicated in-house UltraCare Gene Therapy Guides are available Monday through Friday from 9 a.m. to 8 p.m. Eastern Time at 888-756-8657. More information is available at www.ultracaresupport.com. 

GENGLYCOS will be available through a national network of Qualified Treatment Centers (QTCs) with specialized expertise and training to safely administer gene therapy.

GENGLYCOS is manufactured entirely at Ultragenyx’s Gene Therapy Manufacturing Facility (GTMF) in Bedford, Mass., strengthening the Company’s ability to scale production of high-quality gene therapy products and deliver them to patients as efficiently as possible. 

More information will be available at www.genglycos.com.

This approval reflects the work of a remarkable team spanning many years and organizations. Ultragenyx is deeply grateful to the patients, families, and clinical investigators who made our clinical studies possible; Dr. David Weinstein, whose scientific and clinical leadership laid critical groundwork across early research and clinical trials; Dr. Janice Chou for her early work at NIH, and the team from Dimension Therapeutics. Their collective commitment and perseverance have transformed a scientific vision into a new therapeutic option for patients.

Investor Conference Call and Webcast Information

Ultragenyx will host a conference call today at 6:00 p.m. Eastern Time / 3:00 p.m. Pacific Time to discuss the approval of GENGLYCOS. The live and replayed webcast of the call will be available through the company’s website at https://ir.ultragenyx.com/events-presentations. 

INDICATION

GENGLYCOS (pariglasgene brecaparvovec-opnr) is indicated to reduce daily cornstarch intake as an adjunct to nutritional management in adult and pediatric patients 8 years of age and older with glycogen storage disease type Ia (GSDIa).

This indication is approved under accelerated approval based on reduction in daily cornstarch intake. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

GENGLYCOS is contraindicated in patients with known severe hepatic fibrosis or cirrhosis.

WARNINGS AND PRECAUTIONS

Hypersensitivity and Infusion Reactions (IRs)

• Hypersensitivity reactions including anaphylaxis and IRs have occurred with GENGLYCOS treatment. Severe reactions have been reported. Monitor for signs and symptoms of hypersensitivity and IRs, including urticaria, flushing, hypotension, bronchospasm, dyspnea, chest tightness, nausea, vomiting, headache, abdominal pain, lightheadedness, flu-like symptoms, shivering, rash, and hypertension.
• Premedicate with acetaminophen and non-sedating antihistamines and administer GENGLYCOS according to recommended infusion rates. Monitor patients during and after completion of GENGLYCOS infusion as clinically indicated. If anaphylaxis or severe IR occurs, pause GENGLYCOS infusion immediately and initiate medical treatment as clinically indicated, monitoring as needed. For mild to moderate IRs, consider slowing or temporarily interrupting the infusion, and administer symptomatic treatment as clinically indicated. The infusion may be restarted at half the prior rate upon resolution of symptoms.
• Medical support measures, including cardiopulmonary resuscitation equipment and medications for the treatment of anaphylaxis (e.g., epinephrine, antihistamines, corticosteroids), should be available during GENGLYCOS administration.

Hepatotoxicity

• Immune-mediated hepatotoxicity, with elevated alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) levels, has occurred with GENGLYCOS. Avoid use in patients with preexisting hepatic impairment or acute hepatic viral infection.
• Prior to GENGLYCOS infusion, evaluate liver-related medical history and assess liver function by clinical examination and laboratory testing. Advise patients to immediately report signs and symptoms of hepatotoxicity, including fatigue, jaundice, dark urine, nausea, vomiting, and right upper quadrant pain. Administer corticosteroids to all patients after GENGLYCOS infusion in order to mitigate hepatic reactions. Elevated transaminases may require adjustment of the corticosteroid treatment regimen, including increased dose or prolongation of the corticosteroid taper.
• Monitor transaminase levels for the first 6 months after GENGLYCOS administration. Continue to monitor transaminases in all patients who develop transaminase elevations, until transaminases return to baseline or as clinically indicated.

Adrenal Insufficiency

• Adrenal insufficiency, including serious events, has been reported in patients receiving GENGLYCOS during corticosteroid use and tapering.
• Signs and symptoms of adrenal insufficiency include fatigue, weakness, anorexia, nausea, vomiting, hypotension, hyponatremia, and hypoglycemia. Adrenal crisis may present as severe hypotension, acute abdominal pain, or loss of consciousness.
• Monitor patients for signs and symptoms of adrenal insufficiency and adrenal crisis after GENGLYCOS administration during and after corticosteroid therapy and tapering. Taper corticosteroid therapy gradually. Do not abruptly discontinue corticosteroid therapy.

AAV Vector Integration and Risk of Tumorigenicity

• There is a theoretical risk of tumorigenicity due to integration of AAV vector DNA into the genome.
• GENGLYCOS is composed of a recombinant, non-replicating AAV8 vector whose DNA persists largely in episomal form. Random integration of recombinant AAV-vector DNA into human DNA has been reported with AAV gene therapies. The clinical relevance of individual integration events is unknown, but it is acknowledged that individual integration events could potentially contribute to a risk of tumorigenicity. If a tumor develops in a patient receiving GENGLYCOS, health care providers should contact and report the tumor to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657.

Adverse Reactions

• Seven serious adverse events were observed in the Primary Efficacy Analysis Period (PEAP) of Study 1 (Weeks 1-48), including anaphylaxis/infusion reaction (2), adrenal insufficiency (2), high lactate level (2) and hypoglycemia (1).
• The most common adverse reactions during the PEAP of Study 1 (occurring in ≥10% of patients) with higher frequency in GENGLYCOS compared to placebo were ALT/AST Enzyme elevated (71%), Nausea (38%), Headache (24%), Hypertriglyceridemia (29%), Adrenal Insufficiency (24%), Constipation (19%), Hyperglycemia (14%), Acne/Dermatitis Acneiform (19%), Cushingoid Features (14%), and Anaphylaxis (10%).

DRUG INTERACTIONS

Vaccinations

• Vaccine schedules may need to be adjusted for immunosuppressive therapy, and vaccines should be avoided 1 month prior to GENGLYCOS administration.

USE IN SPECIFIC POPULATIONS

Pregnancy

• GENGLYCOS should not be used during pregnancy. There are no data on the use of GENGLYCOS in pregnant women. It is unknown whether GENGLYCOS can cause fetal harm when administered to a pregnant woman or can affect reproductive capacity.

Contraception

• Women of childbearing potential should use effective contraception for at least 12 months after administration of GENGLYCOS.
• For 6 months after administration of GENGLYCOS, men must not donate semen, and men of reproductive potential and their female partners must prevent or postpone pregnancy using an effective form of contraception.

ADDITIONAL PATIENT COUNSELING INFORMATION

Vector Shedding

• Inform patients/caregivers that vector distribution in blood and vector shedding in urine, stool, and saliva can occur after GENGLYCOS infusion. Advise patients/caregivers on proper hygiene when handling patient body waste. These precautions should be followed for 3 months after GENGLYCOS infusion.

Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657. 

Please see the full Prescribing Information for GENGLYCOS.

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. Deficiency of G6Pase activity results in severe hypoglycemia during periods of fasting between meals and during the night along with excess hepatic glycogen storage, metabolic derangements, and other disease-related complications. Cornstarch is critical in the management of GSDIa throughout the day and night in providing an exogenous source of glucose to help avoid sudden and severe drops in plasma glucose levels; however, current management strategies carry a significant burden to patients and families. GSDIa affects 1,500-2,500 patients in the U.S. and 6,000-8,000 worldwide within commercially accessible geographies.

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. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.

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.

For more information on Ultragenyx, please visit the company’s website at: www.ultragenyx.com.

Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements regarding the commercial launch, availability and market acceptance of GENGLYCOS; Ultragenyx’s ability to manufacture GENGLYCOS at its gene therapy manufacturing facility, scale production and supply GENGLYCOS to Qualified Treatment Centers; patient access to GENGLYCOS, including insurance coverage and reimbursement; the safety, efficacy, durability and potential benefits of GENGLYCOS; estimates of the number of patients with GSDIa and the potential commercial opportunity for GENGLYCOS; the design, enrollment, timing, conduct and results of the post-marketing Disease Monitoring Program and other post-marketing requirements; and Ultragenyx’s ability to satisfy FDA requirements and maintain accelerated approval for GENGLYCOS, are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause actual results to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, risks and uncertainties related to the commercial launch and market acceptance of GENGLYCOS; the ability to identify eligible patients and establish and support a network of Qualified Treatment Centers; uncertainty related to insurance coverage and reimbursement; risks related to serious or undesirable side effects, including risks associated with AAV gene therapy; manufacturing risks, including Ultragenyx’s limited experience operating its own manufacturing facility and the ability to manufacture and supply GENGLYCOS in sufficient quantities and in compliance with regulatory requirements; Ultragenyx’s ability to complete the post-marketing Disease Monitoring Program and other post-marketing requirements within required timeframes and to confirm clinical benefit; the risk that the FDA may modify the approved indication or impose additional requirements, or may withdraw accelerated approval if clinical benefit is not confirmed or post-marketing requirements are not satisfied; smaller than anticipated market opportunities; competition from other therapies or products; product liability; regulatory scrutiny; and other matters that could affect the availability or commercial potential of Ultragenyx’s products and product candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.

For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 5, 2026, and its subsequent periodic reports filed with the SEC.

In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).

Ultragenyx Contacts

Investors
Joshua Higa
ir@ultragenyx.com

Media
Jess Rowlands
media@ultragenyx.com

In 2019, Joey Thames attended an SCbio event as a representative of Clemson University. He didn’t know at the time, but a conversation that evening would eventually lead him to his next career.

At that event, Thames met Corporate Manager Sheila Dela Cruz and President Dave Shalaby of Poly-Med, who also serves as SCbio’s Fiduciary and Operations Board Secretary, where he had the opportunity to talk with them about his research, interests and experience. He was already familiar with Poly-Med through the company’s Chief Technology Officer, Scott Taylor, but the conversation gave him a chance to learn more about the company and share what he could bring to the team.

Two years later, Thames received a phone call from Poly-Med about an open position.

Today, he serves as the company’s recruiting manager.

His story is one example of what nearly 14 years of SCbio membership has meant to Poly-Med: connections that continue long after an event ends.

More Than a Membership: Investing in People, Partnerships and Relationships

Based in Anderson, Poly-Med is an industry leader in bioresorbable medical technology. The company develops biomaterials and implantable devices designed to support the body as it heals and then safely dissolve once they are no longer needed.

Poly-Med brings the entire process under one roof, including raw polymer synthesis and fiber extrusion to finished implantable device manufacturing. Their in-house expertise allows the company to turn complex ideas into medical technologies that improve patient care.

But Poly-Med’s impact on South Carolina’s life sciences extends beyond the products it develops.

Since its founding in 1993, the company has invested in people, partnerships and relationships that help drive innovation. Its long-standing membership with SCbio has been one part of that effort, giving Poly-Med opportunities to connect with industry leaders, engage with students, support workforce development, participate in policy conversations and stay informed about the issues shaping South Carolina’s life sciences industry.

Simply put, SCbio membership has given Poly-Med a way to stay connected to people and ideas driving the state’s life sciences ecosystem.

Building Connections that Can Change a Company’s Future

In any industry, relationships matter. In life sciences, they can change the company’s future.

Poly-Med has seen that firsthand through its participation in SCbio events, including Academia + Industry: Bridging the Gap, regional programs such as Breakfast and Bio, the Workforce Roundtable Luncheon and at the Life + Science Annual Conference.

“SCbio conferences are much more than opportunities to hear from leaders across the life sciences industry,” said Thames. “They foster meaningful collaboration, the exchange of ideas and lasting professional relationships.”

Those relationships have become particularly valuable as Poly-Med works to build its talent pipeline.

Over the past year, the company participated in three Academia + Industry events, connecting with more than 100 college students and learning about their interests, skills and career goals.

Some of those conversations have continued well beyond the event.

Turning Conversations into Careers

Kylie King is one example of an SCbio student member who has a career success story with Poly-Med.

Thames first met King, a Clemson University biomedical engineering Ph.D. student, at SCbio’s Life + Science Conference. They crossed paths again at an Upstate Academia + Industry: Bridging the Gap event, where Thames saw the ambition and curiosity that Poly-Med looks for in its employees.

Later, he came across King’s profile on LinkedIn and remembered their conversations.

Today, King is a full-time member of the Poly-Med team.

Another Poly-Med hire came through the SCbio Foundation Internship Grant.

Haley Pepper joined Poly-Med as an intern after receiving support from the SCbio Foundation.

“When we met Haley Pepper, we found someone who was engaging and curious,” Thames said.

After Pepper connected with SCbio President and CEO James Chappell, Ph.D., and Executive Vice President and COO Erin Ford, they referred her to Poly-Med for an internship opportunity.

These connections illustrate one of the most valuable parts of SCbio’s role in the industry: bringing the right people together.

Staying Connected to the Industry

Talent is only one part of Poly-Med’s engagement with SCbio.

The company also uses its membership to stay connected to the issues affecting its business and to better understand where South Carolina’s life sciences industry is headed.

Poly-Med representatives have participated in SCbio’s legislative events and conversations with policymakers. The company has also participated in the BIO Fly-In in Washington, D.C., a roundtable discussion with the office of Sen. Tim Scott and the Life Sciences Legislative Breakfast.

This involvement gives Poly-Med a broader view of the opportunities and challenges facing the industry.

“Being a part of SCbio for almost 14 years has provided us with a birds-eye view of the life sciences ecosystem of experts and technologies operating all over the state of South Carolina,” Thames said.

That perspective has helped Poly-Med stay informed on policy and workforce issues while also giving the company opportunities to share its own experiences and ideas.

Growing With South Carolina’s Life Sciences Industry

Poly-Med’s continued growth in South Carolina reflects many of the strengths that make the state attractive to life sciences companies: a growing talent pipeline, strong research partners and a cost-effective operational environment.

The company expanded to Powdersville in 2019 and again to Liberty in 2022, while continuing to develop biomaterial platforms and engineering capabilities designed to improve patient care.

SCbio has helped Poly-Med stay connected to the people and opportunities surrounding that growth.

Over the past year, Poly-Med has reached more than 775 people through SCbio events and received more than 69,000 brand impressions through SCbio communications.

But those numbers tell only part of the story.

The more meaningful measure is what happens after the event ends, like a researcher meeting a future colleague, a student meeting an employer and a company connecting with a policymaker.

That is what has kept Poly-Med engaged with SCbio for nearly 14 years.

“Membership with SCbio has truly allowed us to engage directly with industry partners and share experiences and ideas that allow member companies a leg up in the highly competitive life sciences industry,” Thames said.

As Poly-Med continues to develop new biomaterial platforms and the engineering expertise to support them, the company plans to keep investing in the people and partnerships that have helped shape its growth.

About Poly-Med, Inc.

Poly-Med’s mission is to design, develop, and manufacture custom components and implantable medical devices using next-generation absorbable biomaterials, propelling customers’ specialty product visions into tangible, highly competitive and unique product offerings.

SCbio, and other healthcare and life sciences leaders, joined the United Nations Foundation to highlight South Carolina’s distinct contributions to global health during the Foundation’s Global Health Celebration on Wednesday in Columbia.

Throughout August, the Foundation will showcase the vital importance of U.S. leadership in global health programs, sharing how these global investments drive local innovation and foster economic vitality within specific states and districts.

The event began with a dedication to Senator Lindsey Graham, who served South Carolina in Congress from 2002 until his passing in July 2026.

Representative Joe Wilson was recognized for his steadfast support for global polio eradication and Gavi, the Vaccine Alliance, the largest purchaser of U.S.-produced vaccines, which helps vaccinate over 50% of children worldwide.

James Chappell, Ph.D., President and CEO of SCbio, thanked Rep. Wilson for his service and explained the importance of his support of healthcare initiatives and programs. “That type of work and support is so important for the life sciences industry I represent in South Carolina. We know that before a treatment reaches those patients, someone has to do the research. Someone has to discover it. 
Someone has to commercialize it. Someone has to manufacture. What I’m most excited about is that someone, more and more, is right here in South Carolina.”

Deputy Secretary of the S.C. Department of Commerce Ashely Teasdel emphasized the large role the South Carolina life sciences industry serves in the intersection of healthcare and global health, where innovation translates into new treatments and medical technologies for patients.

“We’re seeing a thriving and growing sector of the life sciences across the state,” said Teasdel. “What that translates to is our commitment to our mission every single day, which is to create economic opportunities and choices for our South Carolinians.”

Jody Chastain, president and CEO of The Ritedose Corporation, a leading Blow-Fill-Seal CDMO for generic pharmaceuticals, shared how patients in 13 countries are being impacted by products manufactured at the Richland County facility.

“When I think about the globalization effort on the eradication of disease through vaccines and biologics, Ritedose is here in Columbia, South Carolina. And we should have an immense impact on the world.”

The United Nations Foundation has built novel innovations and partnerships to support the United Nations and help solve global problems at scale for over 25 years. As an independent charitable organization, the Foundation was created to work closely with the United Nations to address humanity’s greatest challenges and drive global progress.

About SCbio

SCbio is the definitive, unifying leader in South Carolina’s life sciences sector, fostering authentic collaboration and innovation across academia, startups, government and established enterprises. As a member-driven economic development organization, SCbio represents an industry that generates $20.1 billion in annual economic impact and supports 63,000 jobs across South Carolina. Our members are unified by a mission to positively impact humanity through science across the ecosystem of advanced and specialty manufacturing; digital health and the data frontier; and cutting-edge health specializations. For additional information about SCbio and the life sciences industry, or to become a member, visit SCbio.org.