We are a leader in the field of gene therapy, seeking to transform the lives of patients suffering from debilitating genetic diseases by developing potentially one-time, life-altering treatments. The goal of gene therapy is to overcome the effects of a malfunctioning, disease-causing gene. Gene therapies have the potential to provide long-lasting effects, dramatically and positively changing the lives of patients with conditions where no, or only palliative, therapies exist. We have built a pipeline of gene therapy product candidates that are directed to the retina, the liver and the central nervous system. In December 2017, the U.S. Food and Drug Administration, or FDA, approved LUXTURNATM (voretigene neparvovec-rzyl) for the treatment of patients with viable retinal cells and confirmed biallelic RPE65 mutation-associated retinal dystrophy, a genetic blinding condition caused by mutations in the RPE65 gene. LUXTURNA is the first FDA-approved gene therapy for a genetic disease, the first and only pharmacological treatment for an inherited retinal disease, or IRD, and the first adeno-associated virus, or AAV, vector gene therapy approved in the United States. LUXTURNA is manufactured at our manufacturing facility located in Philadelphia, which is the first licensed manufacturing facility in the United States for a gene therapy treating an inherited disease. LUXTURNA has received orphan drug status. In January 2018, we entered into a license and commercialization agreement with Novartis Pharma AG, or Novartis, for the development and commercialization of voretigene neparvovec outside the United States. In November 2018, we received approval from the European Medicines Agency, or EMA, for the marketing authorization of LUXTURNA in all 28 member states of the European Union, as well as Iceland, Liechtenstein and Norway.
Company profile
Ticker
ONCE
Exchange
Website
CEO
Jeffrey D. Marrazzo
Employees
Incorporated
Location
Fiscal year end
Industry (SIC)
SEC CIK
Corporate docs
ONCE stock data
Latest filings (excl ownership)
15-12B
Securities registration termination
27 Dec 19
S-8 POS
Registration of securities for employees (post-effective amendment)
17 Dec 19
S-8 POS
Registration of securities for employees (post-effective amendment)
17 Dec 19
S-8 POS
Registration of securities for employees (post-effective amendment)
17 Dec 19
S-8 POS
Registration of securities for employees (post-effective amendment)
17 Dec 19
S-8 POS
Registration of securities for employees (post-effective amendment)
17 Dec 19
25-NSE
Exchange delisting
17 Dec 19
8-K
Termination of a Material Definitive Agreement
17 Dec 19
SC 14D9/A
Tender offer solicitation (amended)
17 Dec 19
SC TO-T/A
Third party tender offer statement (amended)
17 Dec 19
Latest ownership filings
SC 13G/A
Beneficial ownership report (amended)
14 Feb 20
3
Initial statement of insider ownership
18 Dec 19
4
Lota S. Zoth
17 Dec 19
4
Lisa Dalton
17 Dec 19
4
Stephen W Webster
17 Dec 19
4
ELLIOT SIGAL
17 Dec 19
4
ROBERT J PEREZ
17 Dec 19
4
VINCENT MILANO
17 Dec 19
4
Anand Mehra
17 Dec 19
4
Federico Mingozzi
17 Dec 19
Institutional ownership, Q3 2021
13F holders | Current |
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Total holders | 0 |
Opened positions | 0 |
Closed positions | 0 |
Increased positions | 0 |
Reduced positions | 0 |
13F shares | Current |
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Total value | 0.00 |
Total shares | 0.00 |
Total puts | 0.00 |
Total calls | 0.00 |
Total put/call ratio | – |
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