Company Information

  

Address: 1035 O'BRIEN DRIVE  
City: MENLO PARK 
State: CA 
Zip Code: 94025 
Telephone: 650-272-6269 

Description of Business

Primary SIC Code: 2836
(As filed with the SEC)  

We are a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Leveraging our next-generation adeno-associated virus ("AAV")-based directed evolution platform, we generate gene therapy product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Our core capabilities include clinical development and in-house manufacturing expertise, specifically in process development, assay development, and current Good Manufacturing Practices ("cGMP") quality control. Our leadership team has significant drug development and gene therapy expertise. We are advancing our robust pipeline of gene therapy product candidates designed to treat rare diseases, alpha-1 antitrypsin ("A1AT") deficiency and hereditary angioedema ("HAE"), as well as wet age-related macular degeneration ("wAMD"). Our pipeline of lead and partnered gene therapy programs is shown below.  ... More ...  

Per Share Overview

Date 12-mos Rolling EPS Dividend P/E Ratio
03/2018-1.21NAN/E
12/2017-1.29NAN/E
09/2017-1.45NAN/E
06/2017-1.29NAN/E
03/2017-2.95NAN/E
09/2016-3.21NAN/E
06/2016-3.60NAN/E
03/2016-2.05NAN/E

Key Financial Ratios and Statistics

FYE: 12/31

Profitability 2017 Leverage 2017
Net Inc/Comm Equity-0.13Total Liab/Total Assets0.09
Net Inc/Total Assets-0.28Total Liab/Inv Cap0.09
Net Inc/Inv Cap-0.29Total Liab/Comm Equity0.04
Pretax Inc/Net Sales-30.37Interest Coverage RatioNA
Net Inc/Net Sales-30.37Curr Debt/EquityNA
Cash Flow/Net Sales-24.57LTD/EquityNA
SG&A/NetSales11.28Total Debt/EquityNA
Asset Utilization   Liquidity  
Net Receivables Turnover4.17Quick Ratio18.15
Inventory TurnoverNACurrent Ratio18.15
Inventory Day SalesNANet Rec/Curr AssetsNA
Net Sales/Work Cap0.01Inv/Curr AssetsNA
Net Sales/PP&E0.61  

Income Statement (Millions)

  3/31/2018 12/31/2017 9/30/2017 6/30/2017
Total Revenues(Net Sales) 0.22 0.46 0.46 0.46
Cost of Goods Sold NA NA NA NA
Selling & Admin Exps 5.37 4.04 4.76 4.06
Operating Income -17.95 -15.60 -14.57 -12.09
Interest Exp NA NA NA NA
Pretax Income -17.20 -14.79 -13.83 -11.43
Other Income 0.75 0.81 0.74 0.66
Net Income Bef Extraordinary ... NA NA NA NA
Net Income -17.20 -14.79 -13.83 -11.43

Balance Sheet (Millions)

Assets 3/31/2018 12/31/2017 9/30/2017 6/30/2017
Cash & Short Term Investments 247.04 190.49 186.64 197.42
Receivables - Total NA 0.00 0.00 0.00
Inventories - Total NA NA NA NA
Total Current Assets 249.20 193.74 189.52 199.64
Net Property, Plant & Equipment 2.82 3.02 3.35 3.80
Total Assets 257.16 201.91 198.21 208.58
Liabilities        
Accounts Payable 7.89 8.70 7.64 6.35
Debt in Current Liabilities NA NA NA NA
Total Current Liabilities 9.27 10.67 9.61 8.31
Long-Term Debt NA NA NA NA
Total Liabilities 11.12 17.88 17.22 16.40
Stockholder's Equity        
Minority Interest NA NA NA NA
Preferred Stock 0.00 0.00 0.00 0.00
Common Stock 0.01 0.01 0.01 0.01
Retained Earnings -265.12 -254.06 -239.27 -225.44
Treasury Stock NA NA NA NA
Total Stockholders' Equity 246.04 184.03 180.99 192.18
Total Liabilities and Stockholders' Equity 257.16 201.91 198.21 208.58

Cash Flow Summary (Millions)

Categories 3/31/2018 12/31/2017 9/30/2017 6/30/2017
Net Cash Provided by Operating Activities -12.98 -12.20 -9.99 -11.42
Net Cash Provided by Investing Activities 25.38 34.69 2.63 -20.67
Net Cash Provided by Financing Activities 69.80 16.65 -0.24 0.16

Annual Summary Data (Millions)

Year Sales Net Income EPS
12/20130.48-5.28--
12/20140.57-25.40--
12/20152.32-47.45--
12/20161.46-113.75--
12/20171.85-56.15-1.29
Growth Rates40.10----

Stock Ownership

Type Date(Q,M) No. Owners Shares Held (000s) % Own
Institutional06/30/1811134,84255.95




Report Date : 7/16/2018

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