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Recent, highly favorable U.S. Patent and Trademark Office decision affirms and upholds fundamental CRISPR/Cas9 issued patents
Extended CRISPR technology leadership with addition of novel and differentiated Cpf1 system, advanced forms of Cas9
Achieved in vivo proof-of-editing of non-human primate retina in LCA10 program
“In three years, we have established an unparalleled position for developing genome editing medicines,” said
Bosley added, “2017 is already shaping up to be a transformative year for Editas. The recent, highly favorable decision by the U.S. Patent & Trademark Office affirms and upholds the broad and fundamental patents in this field granted to the
Driving Editas Medicine’s unparalleled platform for genome editing medicines
- Reinforced robust Cas9 intellectual property. Highly favorable decision by US Patent and Trademark Office (USPTO) in
February 2017affirmed and upheld the Broad Institute, Inc’s (Broad) fundamental CRISPR/Cas9 patents and reinforces Editas Medicine’s unmatched intellectual property portfolio for CRISPR medicines. The USPTO determined that Broad’s invention using CRISPR/Cas9 in eukaryotic cells was not obvious in view of University of California, University of Vienna, and Emmanuelle Charpentier’s claims in their patent application and thus ended the interference and upheld the Broad issued patents. Foundational claims covering the use of CRISPR/Cas9 for gene editing in eukaryotic cells have issued to the Broad as patents in each of the United States, Europe, and Australia.
- Expanded unparalleled and differentiated platform for genome editing medicines with exclusive licenses for the CRISPR/Cpf1 system and for advanced forms of Cas9. Editas Medicine’s platform now includes exclusive rights to both CRISPR systems that have been demonstrated to work in human cells: Cas9 and Cpf1. Cpf1 offers several potential advantages including expanding the range of genomic sites that can be edited, simplifying manufacture and delivery, and increased efficiency and accuracy of some forms of gene repair. Its intellectual property rights are independent of Cas9.
- Invented and deployed new proprietary technologies to potentially enable unprecedented medicines.
Editas Medicineachieved multiple technical advancements including novel compositions and methods for covalently-coupled dual guide RNAs and rigorous new empirical methods for assessing specificity referred to as Uni-directional Targeted Sequencing (UDITAS™).
Advancing a pipeline strategy to enable successful product development in the years ahead
- Achieved in vivo proof-of-editing in non-human primates in our Leber Congenital Amaurosis type 10 (LCA10) program. Our advancements in the LCA10 program establish the foundation for developing a wide range of potential treatments for inherited retinal diseases and diseases of the eye, as well as other systemic therapies.
- Demonstrated efficient editing of hematopoietic stem cells (HSCs) and long-term engraftment and reconstitution of the blood and bone marrow using edited cells in animal models.
Editas Medicine'sprogress in HSCs provides the foundation for potential CRISPR medicines to treat a broad range of diseases of the blood and bone marrow, including sickle cell disease and immunological diseases.
- Attained over 90% knock-out of PD-1 in T cells carrying a chimeric antigen receptor (CAR) with no detected off-target edits in our collaboration with
Juno Therapeutics, Inc.(Juno Therapeutics). In our wholly-owned T cell research outside of cancer, we attained similar high levels of editing and targeted integration that creates the potential to treat a broad array of immunological and infectious diseases.
Building the business for the long term by assembling the capabilities to fully develop and commercialize important medicines
- Successfully completed initial public offering resulting in
$109 millionin gross proceeds including full exercise of underwriters' over-allotment option.
- Expanded and strengthened executive team with multiple appointments in 2016 and early 2017, including:
- Established important alliances to further extend platform leadership and advance our pipeline. We are working with
Adverum Biotechnologiesto explore the use of Adverum's next-generation adeno-associated virus (AAV) vectors in up to five ophthalmic indications. Through our collaboration with San Raffaele Telethon Institute for Gene Therapy(TIGET), one of the world's leading institutes for genomic medicines, we aim to advance our pipeline of HSC and T cell therapies.
- Submit IND for LCA10 program by end of year;
- Initiate LCA10 clinical natural history study mid-year;
- Achieve pre-clinical proof-of-concept for additional programs;
- Establish additional alliances to enable successful product development; and
- Continue to build outstanding organization and culture.
Barclays Global Healthcare Conference, March 16, 9:30 a.m. ET, Miami; and UBS Global Healthcare Conference, May 22-24, New York City.
- Keystone Symposium on Genomic Instability and DNA Repair,
April 2-6, Santa Fe;
- Tides 2017: Oligonucleotide and Peptide Therapeutics,
April 30-May 3, San Diego; and American Society of Gene & Cell Therapy(ASGCT), May 10-13, Washington, DC.
Fourth Quarter and Full Year 2016 Financial Results
Cash and cash equivalents at
For the three months ended
- Collaboration and other research and development revenues were
$0.9 millionfor the three months ended December 31, 2016, compared to $0.8 millionfor the same period in 2015. The $0.1 millionincrease was due to an increase in revenue recognized pursuant to our collaboration with Cystic Fibrosis Foundation Therapeutics, Inc.
- Research and development expenses increased by
$21.0 million, to $26.8 millionfor the three months ended December 31, 2016, from $5.8 millionfor the same period in 2015. The $21.0 millionincrease was due to a $16.5 millionincrease in license fees, primarily related to new license agreements with Massachusetts General Hospitaland the Broad Institute, a $2.1 millionincrease in employee and non-employee related expenses, including stock-based compensation, resulting from an increase in the size of our workforce, a $1.2 millionincrease in our process and platform development expenses due to increased research activity, and a $1.2 millionincrease in facility-related costs resulting from additional office and laboratory space.
- General and administrative expenses increased by
$5.7 million to $13.0 millionfor the three months ended December 31, 2016, from $7.3 millionfor the same period in 2015. The $5.7 millionincrease in general and administrative expenses consisted of an increase of $3.5 millionin legal fees to support patents that we own or in-license, including costs for the prosecution and maintenance of patents that we own or in-license as well as to procure the application for and issuance of additional patents in the United Statesand other jurisdictions, an increase of $1.6 millionin employee compensation costs, and a $0.6 millionincrease in other general and administrative expenses including office and facility costs related to our new headquarters.
For the full year 2016, net loss attributable to common stockholders was
- Collaboration and other research and development revenues were
$6.1 millionfor 2016, compared to $1.6 millionfor 2015. The increase of $4.5 millionwas due to a $4.2 millionincrease in revenue recognized pursuant to our collaboration with Juno Therapeutics, and a $0.3 millionincrease in revenue recognized pursuant to our agreement with Cystic Fibrosis Foundation Therapeutics.
- Research and development expenses for 2016 were
$57.0 million, compared to $18.8 millionfor 2015. The increase of $38.1 millionwas due to a $14.6 millionincrease in research and development employee and non-employee compensation costs, including stock based compensation, a $13.1 millionincrease in license fees primarily related to new license agreements with Massachusetts General Hospitaland the Broad Institute, $6.4 millionin increased process and platform development costs, $3.9 millionin increased facilities costs, and $0.1 millionin increased other expenses.
- General and administrative expenses were
$46.3 millionfor 2016, compared to $18.1 millionfor 2015. The increase of $28.2 millionwas due to a $16.5 millionincrease in in legal fees to support patents that we own or in-license, including costs for the prosecution and maintenance of patents that we own or in-license, $7.2 millionin increased employee compensation costs, $2.4 millionin increased contractor consulting fees, and $2.1 millionin other general and administrative expenses.
- Other income (expense), net for 2016 was
$5 thousand, compared to $(37.6) millionfor 2015. The decrease in expense was primarily related to a $35.6 milliondecrease in our Series A preferred stock tranche right liability, which was settled in June 2015, resulting from mark-to-market adjustments attributable to an increase in the fair value of our Series A preferred stock during 2015, and to a $1.6 millionmark-to-market adjustment recorded in June 2015for the anti-dilution protection liability, which was also settled in June 2015, related to our issuance of common stock to our licensors.
Editas Medicine is a leading genome editing company dedicated to treating patients with genetically-defined diseases by correcting their disease-causing genes. The Company was founded by world leaders in genome editing, and its mission is to translate the promise of genome editing science into a broad class of transformative genomic medicines to benefit the greatest number of patients.
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s goals of submitting of an IND for the LCA10 program by the end of 2017, initiating an LCA10 clinical natural history study in mid-2017, achieving preclinical proof-of-concept for additional programs, and establishing additional alliances to enable successful product development. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the
|EDITAS MEDICINE, INC.|
|Consolidated Statement of Operations|
|(amounts in thousands, except share and per share data)|
|Three Months Ended||Twelve Months Ended|
|December 31,||December 31,|
|Collaboration and other research and development revenues||$||898||$||792||$||6,053||$||1,629|
|Research and development||26,835||5,826||56,979||18,846|
|General and administrative||13,047||7,339||46,262||18,095|
|Total operating expenses||39,882||13,165||103,241||36,941|
|Other income (expense), net:|
|Other expense, net||(35||)||(226||)||(57||)||(37,445||)|
|Interest income (expense), net||(357||)||(34||)||62||(143||)|
|Total other income (expense), net||(392||)||(260||)||5||(37,588||)|
|Net loss and comprehensive loss||$||(39,376||)||$||(12,633||)||$||(97,183||)||$||(72,900||)|
|Accretion of redeemable convertible preferred stock to redemption value||$||-||$||(99||)||$||(47||)||$||(394||)|
|Net loss attributable to common stockholders||$||(39,376||)||$||(12,732||)||$||(97,230||)||$||(73,294||)|
|Net loss per share attributable to common stockholders, basic and diluted||$||(1.10||)||$||(4.05||)||$||(3.02||)||$||(28.55||)|
|Weighted-average common shares outstanding, basic and diluted||35,731,230||3,145,380||32,219,717||2,566,916|
|EDITAS MEDICINE, INC.|
|Selected Consolidated Balance Sheet Items|
|(amounts in thousands)|
|December 31,||December 31,|
|Cash and cash equivalents||$||185,323||$||143,180|
|Deferred revenue, net of current||26,000||25,321|
|Construction financing lease obligation, net of current portion||35,096||-|
|Redeemable convertible preferred stock||-||199,915|
|Total stockholders' equity (deficit)||134,607||(83,114||)|
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