Voretigene Neparvovec

The University of Pennsylvania has spun out multiple life sciences companies over the years, including Interius BioTherapeutics / iStock, f11photo The Philadelphia market has gained recognition not only for its cell and gene therapy sector but also its real estate scene and talent pool. Vittoria Biotherapeutics, Interius BioTherapeutics and Chamber of Commerce for Greater Philadelphia executives share why the area is a life sciences hot spot. As a life sciences hub within the Pharm Country hotbed , the Philadelphia market has much to offer, although it wasn’t always that way. Just ask Nick Siciliano, CEO, co-founder and director of Philadelphia-based Vittoria Biotherapeutics. He told BioSpace that Philadelphia’s life sciences scene has grown tremendously during his 20-year career in the city and is more sophisticated than it’s ever been. “You have a stable of entrepreneurs and life science executives that I think are committed now to bringing Philadelphia up to par with the Bostons, the Bay Areas,” said Siciliano, whose clinical-stage immunotherapy company develops cell therapies for hard-to-treat diseases. “They’re committed to building companies here. The technology that we’ve seen just in the last decade has been inspirational coming out of some of these institutions for what I believe are going to be transformative modalities in medicine—cell therapy, gene therapy, etc.” The supporting infrastructure has also noticeably changed during Siciliano’s 20-year career. As an example, he noted the growth of incubator University City Science Center. Established in 1963 as a research park, the nonprofit eventually partnered with Wexford Science & Technology and Ventas to create uCity Square , a mixed-use development that takes up 11 city blocks and is home to over 200 life sciences companies. The Science Center has full or partial ownership in six buildings there. Philadelphia’s life sciences businesses and jobs have grown significantly in the past five to 10 years, according to Claire Greenwood, senior vice president of economic competitiveness at the Chamber of Commerce for Greater Philadelphia and executive director of the chamber’s CEO Council for Growth. A 2024 chamber report reflects that growth. It noted that more than 5,000 people work in biotech in Philadelphia, an eight times increase from a decade ago, while lab space square footage inside the city has increased by 150% since 2018. Also noteworthy: According to the chamber , Greater Philadelphia—which it defines as an 11-county region across northern Delaware, southern New Jersey and southeastern Pennsylvania—has over 1,200 life sciences establishments, including more than 450 biotech research and development and manufacturing companies. Cell and Gene Therapy Remains Key Part of Identity The Philadelphia market is best known for its innovative cell and gene therapy sector, which has more than 60 companies and institutions in Greater Philadelphia, according to the chamber’s report. The report also noted that the sector represents over 7,000 jobs and $1 billion in estimated wages. Notable milestones include development of Kymriah and Luxturna , which in 2017 were the first FDA-approved cell and gene therapies. The Philadelphia market goes beyond cell and gene therapy, however. mRNA technology is a growing area, according to Greenwood and Cushman & Wakefield. The commercial real estate services firm’s 2024  research noted, “Pioneered in Philadelphia, mRNA technology presents a budding future for Philadelphia’s life sciences ecosystem.” An April 2024 Colliers life sciences report had similar findings, noting that the Philadelphia market remains on the forefront of gene, cell and mRNA research and development. Real Estate Scene Earns Recognition The Colliers report also named the area as the No. 4 life sciences market based on 2020 to 2023 venture capital funding, fiscal year 2023 National Institutes of Health funding, 2022 biomed degree completions, size of real estate inventory and space absorbed—the rate at which property is leased or sold—over the past three years. Regarding real estate specifically, Philadelphia has started coming into its own, noted Dora Mitchell, senior vice president of operations and chief of staff at Interius BioTherapeutics, a Philadelphia-based clinical-stage biotech that’s developing genetic medicines. Mitchell told BioSpace that five or six years ago, she likely would have said, “Where are all the developers at?” Now, she said, the city has an excellent mix of space available, including larger and customizable offerings. Greenwood told BioSpace she’s seen significant real estate investment in the past three to four years. Because of that, she said, the issue of not having the space needed to serve the industry is no longer part of the dialogue. She noted that there’s a lot of new capacity either online or coming online, whether companies are looking for manufacturing facilities or traditional lab and office workspaces. According to the Colliers report, there is 2.5 million square feet under construction in the Philadelphia market, where the vacancy rate is 7.7%. As to the cost of office space, a key factor for life sciences companies, Mitchell noted that rent in Philadelphia is a fraction of what it is in Boston. That aligns with Cushman & Wakefield research, which stated that the cost per square foot is $53.53 in the Philadelphia market, compared to $90.39 in the Boston market. Proximity to Resources Benefits Companies A key advantage of the Philadelphia life sciences scene, according to Greenwood, Mitchell and Siciliano, is proximity to resources, including universities and contract development and manufacturing organizations (CDMOs). Siciliano noted that companies can get in front of third-party manufacturing groups, academic collaborators and other industry service providers all in an afternoon. Philadelphia universities include the University of Pennsylvania, which has spun out multiple life sciences companies, including Interius, whose co-founders are Saar Gill, an associate professor of medicine at Penn, and Bruce Peacock, who also co-founded Vittoria and has worked in biotech for over 35 years. Mitchell had high praise for Peacock’s contributions to Philadelphia’s biotech scene. “We all need to put a monument of that man out in Philly somewhere,” she said. Regarding proximity to resources, Mitchell noted that Interius is walking distance from the University of Pennsylvania, which houses Gill’s research lab, and near the Philadelphia College of Pharmacy, which is part of Saint Joseph’s University. Expand the perimeter, she said, and there’s also Drexel University , Temple University and Thomas Jefferson University. These institutions offer life sciences companies talent and the opportunity to collaborate, she noted. Mitchell also shared that Interius is just 10 minutes away from its contract manufacturing organization. “The benefit that that has brought to our program is just immeasurable,” she said. “Our folks can jump in a car and be right there, troubleshoot together.” Talent Pool Draws Praise As to the talent pool itself, a June 2024 CBRE  report noted that in 2023, the Philadelphia market ranked No. 8 for research and development talent and No. 7 for manufacturing talent. Cushman & Wakefield’s research found that total life sciences employment in the market is 50,300, with the 10-year growth rate at 20.7%. Regarding who’s in that talent pool, Siciliano noted that there are great scientists coming out of the universities. Mitchell agreed, sharing that academic institutions offer individuals who are at the forefront of clinical science. She also highlighted Philadelphia’s manufacturing professionals; nearby New Jersey’s Big Pharma employees, who include R&D and commercialization talent; and CDMOs’ process development professionals. “The combination of all of these inputs, I think, is unique,” Mitchell said. “And I don’t know that you can really replicate that anywhere else.” On the Wish List: More Resident Capital One area where there’s room for improvement, according to Mitchell, is resident capital—having more venture capital firms located in Philadelphia. “There is a benefit to an up-and-coming life sciences community if there is resident capital there, because then you really naturally have more opportunity to bump into each other, to have these sort of organic discussions and ideas flourish in a less sort of deliberate sort of way,” she said. Siciliano agreed there’s not a strong presence of resident capital in the area, noting, “We go to a biotech event here in Philadelphia, we’re not going to be rubbing shoulders with the men and women that run some of the biggest VC funds, where you get that in Boston, you’ll get that in the Bay Area.” He said that should change. “I think as the sector continues to grow here, and more companies are successful, you’ll start to see more and more venture funds set up shop here,” Siciliano said. Interested in more career insights?  Subscribe to  Career Insider  to receive our quarterly life sciences job market reports, career advice and more.

FORT WORTH, Texas, Jan. 21, 2025 (GLOBE NEWSWIRE) -- Nacuity Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to NPI-001 (N-acetylcysteine amide) tablets, Nacuity’s proprietary investigational therapy for the treatment of patients with retinitis pigmentosa (RP). A drug may receive Fast Track designation if it is intended to treat a serious or life-threatening disease and demonstrates the potential to address an unmet medical need. “Fast Track Designation represents an objective assessment by the FDA for the potential of NPI-001 tablets as a treatment for RP, a severe blinding disease,” said G. Michael Wall, Ph.D., Senior Vice President and Chief Scientific Officer of Nacuity Pharmaceuticals. “We are committed to advancing NPI-001 to address this significant unmet medical need for patients suffering from RP.” A drug that receives Fast Track designation may benefit from more frequent interactions with the FDA throughout drug development. In addition, the Fast Track program allows for Accelerated Approval and Priority Review, if relevant criteria are met. About Retinitis PigmentosaOften diagnosed in childhood or adolescence, retinitis pigmentosa (RP) is a heterogeneous group of inherited retinal diseases involving over 3,000 different mutations in more than 50 genes (Daiger et al., 2013), causing progressive night and peripheral vision loss. RP often leads to legal and sometimes complete blindness. Forms of RP and related diseases include Usher syndrome, Leber congenital amaurosis and Bardet-Biedl syndrome. An estimated 100,000 people in the U.S. have RP (https://www.fightingblindness.org/diseases/retinitis-pigmentosa). There are no FDA-approved drugs or no standard treatments for RP. A curative gene therapy, voretigene neparvovec (LUXTURNA®), exists for patients with the RPE65 mutation, about 1-6% of patients with RP (Cross et al., 2022). Nacuity researchers are working to develop a gene-agnostic treatment for RP, a significant unmet medical need. About NPI-001NPI-001 is a proprietary, GMP-grade formulation of N-acetylcysteine amide (NACA) tablets being developed to target oxidative stress associated with diseases such as retinitis pigmentosa. Preclinical studies indicate that NPI-001 boosts glutathione, the body’s most powerful endogenous antioxidant, to stop chemically aggressive oxygen molecules from damaging retinal cells. NPI-001 and related small molecules are potent antioxidants in several preclinical models that may lead to additional indications. In addition to Fast Track Designation, NPI-001 has also been granted Orphan Drug Designation for RP which provides seven years of U.S. FDA regulatory exclusivity for the product upon regulatory approval. About Nacuity Pharmaceuticals, Inc.Nacuity Pharmaceuticals is a clinical-stage leader in innovative treatments for oxidative stress. The company’s powerful, targeted therapies aim to attenuate oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has three highly differentiated clinical programs ongoing for retinitis pigmentosa, cataract and cystinosis. Nacuity has operations in Fort Worth, TX, USA, and Australia, and extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness (https://www.fightingblindness.org/) and its venture arm RD Fund (https://www.retinaldegenerationfund.org). For more information, please visit www.nacuity.com. Trademarks are the property of their owners. Nacuity Media ContactJulia Clements267.626.1085jclements@6degreespr.com References: Cross N, van Steen C, Zegaoui Y, Satherley A, Angelillo L. Retinitis Pigmentosa: Burden of Disease and Current Unmet Needs. Clin Ophthalmol. 2022 Jun 20;16:1993-2010. doi: 10.2147/OPTH.S365486. PMID: 35757022; PMCID: PMC9232096. Daiger SP, Sullivan LS, Bowne SJ. Genes and mutations causing retinitis pigmentosa. Clin Genet. 2013 Aug;84(2):132-41. doi: 10.1111/cge.12203. Epub 2013 Jun 19. PMID: 23701314; PMCID: PMC3856531 Guidance for Industry: Expedited Programs for Serious Conditions – Drugs and Biologics, May 2014. Wood JPM, Chidlow G, Wall GM, Casson RJ. N-acetylcysteine amide and di-N-acetylcysteine amide protect retinal cells in culture via an antioxidant action. Exp Eye Res. 2024 Nov; 248:110074. doi: 10.1016/j.exer.2024.110074. Epub 2024 Sep 7. PMID: 39251120.