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WATERTOWN, Mass.--(BUSINESS WIRE)--Avilar Therapeutics, a biopharmaceutical company pioneering extracellular protein degradation through its ATAC (ASGPR Targeting Chimera) platform, today announced the appointment of Leslie Meltzer, Ph.D. as Chief Executive Officer. Dr. Meltzer will also join Avilar's Board of Directors. Dr. Meltzer joins Avilar as the company advances its lead ATAC program toward the clinic. The program is an ATAC designed to rapidly and selectively degrade circulating sFlt1, a key driver of preeclampsia, an important women’s health issue of high unmet need. The sFlt1 ATAC is progressing alongside additional programs targeting IgM, ApoB, and IgG. “Avilar has built a broad extracellular protein degradation platform with the potential to address a wide range of diseases driven by pathogenic circulating proteins, from our lead ATAC program targeting sFlt1 in preeclampsia to our IgM-targeting ATAC program for anti-MAG neuropathy and other IgM-mediated diseases, and our work developing oral ATAC degraders,” said Milind Deshpande, Chair of Avilar’s Board of Directors. “Leslie has spent her career advancing innovative therapies across the full spectrum from development through approval and commercialization, and we are confident this experience makes her the right leader for Avilar’s next phase.” Dr. Meltzer brings deep expertise developing treatments for complex diseases across a range of therapeutic areas and modalities and is a veteran of two strategic acquisitions. Most recently, she was Chief Research and Development Officer at Apellis Pharmaceuticals, where she led strategic prioritization of the company’s immunology pipeline assets and helped lead the acquisition of Apellis by Biogen in 2026. Previously, she was Chief Medical Officer at Orchard Therapeutics, where she advanced the company's gene therapy pipeline for rare pediatric neurometabolic diseases and secured FDA approval of LENMELDY™ for metachromatic leukodystrophy. Dr. Meltzer was a key figure in Orchard’s acquisition by Kyowa Kirin in 2024. Dr. Meltzer has also held leadership roles at Keryx Biopharmaceuticals, Biogen, and Actelion Pharmaceuticals, building and leading Medical Affairs teams that shaped launch strategies and expanded scientific engagement. Dr. Meltzer holds a Ph.D. in neuroscience from Stanford University and a bachelor's degree in biology and neuroscience from Brandeis University. “I’ve spent my career advancing therapies for complex diseases where patients and their families have significant unmet needs. Avilar is pursuing extracellular targets and diseases where current treatments fall short or are not available, and this is exactly where Avilar’s ATAC degradation approach has the potential to offer meaningful advantages. I am excited to work with the Avilar team to advance the company’s innovative pipeline into the clinic and, ultimately, bring new treatment options to patients,” said Dr. Meltzer. About Avilar Therapeutics Avilar Therapeutics is a biopharmaceutical company pioneering the discovery and development of extracellular protein degraders. Avilar develops ATACs (ASGPR Targeting Chimeras) that harness the liver's natural asialoglycoprotein receptor (ASGPR)-mediated degradation pathway to selectively remove pathogenic extracellular proteins from circulation. Avilar is advancing a pipeline of ATAC programs across multiple therapeutic areas with significant unmet need. Its lead program targets circulating sFlt1, a key driver of preeclampsia. Additional programs include an IgM-targeting ATAC for IgM-mediated diseases, with an initial focus on anti-MAG neuropathy, an oral ApoB-targeting ATAC for cardiovascular disease, and an oral IgG-targeting ATAC for autoimmune diseases. Across the pipeline, Avilar employs biologics, peptides and small molecules as target-binding modalities. These capabilities extend the platform to a broad range of extracellular pathogenic protein targets. Avilar is based in Watertown, Massachusetts. For more information, please visit www.avilar-tx.com and follow Avilar on LinkedIn.

iStock, weiyi zhu The market approval for Fayuvi, which was rejected in 2025 due to manufacturing problems, comes shortly after Ultragenyx got the FDA nod for its first gene therapy last month. The FDA has given the go-ahead to Ultragenyx’s rebisufligene etisparvovec—now to be marketed under the brand name Fayuvi—for the treatment of Sanfilippo syndrome type A, an ulta-rare, progressive neurological condition in children. Fayuvi is the first-ever FDA-approved therapy for the fatal condition, according to a company news release on Thursday afternoon. Sanfilippo syndrome type A causes children to gradually lose cognitive and other developmental abilities, with Ultragenyx’s new therapy indicated for patients with preserved neurodevelopmental function. The approval represents a comeback for the one-time gene therapy, which was initially rejected in July 2025 due to manufacturing issues. Ultragenyx expects Fayuvi will be available to ship within 30 to 60 days, with a wholesale acquisition cost of $3.95 million, Jefferies reported in a note on Thursday evening. That would make it one of the most expensive gene therapies of all time, trailing Orchard Therapeutics’ Lenmeldy for pediatric metachromatic leukodystrophy, which costs $4.25 million , according to a BioSpace analysis. Ultragenyx was up 13% by the end of Thursday’s trading session, hitting $14.50 per share. Drug Delivery Deep Dive: Drug Price Reform In this deep dive, BioSpace takes a closer look at the drug price crisis in the U.S. As President Joe Biden and former President Donald Trump gear up for a rematch in the 2024 election, we explore how federal reforms to lower costs could be leveraged on the campaign trail. April 18, 2024 · 4 min read · Neil Versel Read more Fayuvi is Ultragenyx’s second gene therapy to cross the FDA finish line, behind Genglycos, which was cleared last month for a rare genetic disease called glycogen storage disease type Ia or Von Gierke disease. . Genglycos, also a one-time therapy, is expected to cost $2.7 million. Also called mucopolysaccharidosis type IIIA, Sanfilippo syndrome type A is a rare and heritable disorder characterized by progressive damage to the brain and nervous system, with treatment options up to this point limited to managing symptoms, according to the FDA’s announcement of Fayuvi’s approval. Ultragenyx estimates around 3,000 to 5,000 treatable patients in “commercially accessible geographies. With a median life expectancy of around 15 years for patients with the disease, Jefferies expects the high unmet need to facilitate Fayuvi’s uptake. Ultragenyx said they were already receiving requests already for the drug, including outside of the U.S., according to the analyst. Gene therapy Ultragenyx bags first gene therapy approval as previously rejected Sanfilippo asset awaits decision The accelerated greenlight for Ultragenyx’s gene therapy for glycogen storage disease has raised analyst expectations for approval of UX111, which the FDA rejected last summer and is currently reviewing for a second time. A decision is expected by mid-September. August 20, 2026 · 2 min read · Nick Paul Taylor Read more Also going in Ultragenyx’s favor is the company’s existing manufacturing infrastructure, which the firm contended can drive down production costs to “biologics-like levels.” Jefferies projects peak sales of around $250 million for Fayuvi. Fayuvi was initially developed at Abeona Therapeutics as ABO-102. In 2022, Ultragenyx picked up exclusive licensing rights to the gene therapy, with Abeona on the line to collect up to $30 million in commercial milestones, plus tiered royalties. The adeno-associated virus gene therapy works by delivering a functional copy of the SGSH gene, which encodes an enzyme that is deficient in Sanfilippo, leading to the buildup of a certain molecule in cells that in turn progressively damage the nervous system. Positive data from the pivotal Phase 1/2/3 Transpher A trial supported Fayuvi’s approval, showing biomarker and cognitive improvements as compared with untreated patients from an external national history control group. Fayuvi’s approval gives Ultragenyx a second priority review voucher (PRV), adding to the one it got for Genglycos’ approval. Ultragenyx intends to sell these vouchers, which William Blair analysts in a Friday morning note said “will significantly bolster the company’s balance sheet given the market rate for PRVs is ~$200 million.” As of June 30, the biopharma had $436 million in cash, equivalents and marketable securities. The approval follows a Phase 3 fail in Angelman syndrome earlier this month that prompted Ultragenyx to “assess its planned operations” and initiate “significant expense reductions.” The biotech reported that its investigational antisense oligonucleotide apazunersen was unable to significantly improve cognition , an update that sent the company’s stock crashing more than 43% at the time. Rare diseases Priority Review Vouchers: By the Numbers A BioSpace analysis of all 80 priority review vouchers that have been handed out across the three FDA programs that offer them found that 2024 was the busiest year yet. Companies have disclosed spending $513 million on vouchers that were earned in 2024 so far. March 5, 2025 · 1 min read · Annalee Armstrong Read more