RNAi Company Ltd

Bunkyō-ku, Japan

RNAi Company Ltd

Bunkyō-ku, Japan
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News Article | May 12, 2017
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that it has granted stock options to Manmeet S. Soni, the Company's newly appointed Chief Financial Officer. The compensation committee of Alnylam's board of directors approved the grant, effective as of May 8, 2017, to Mr. Soni of non-qualified stock options to purchase an aggregate of 150,000 shares of Alnylam's common stock. These options were granted as an inducement material to Mr. Soni's entering into employment with the Company in accordance with NASDAQ Listing Rule 5635(c)(4). The stock options have a ten-year term and an exercise price equal to $52.61, the per share closing price of Alnylam's common stock as reported by NASDAQ on the date of grant. Options to purchase 125,000 shares will vest over four years, with 25% vesting on the first anniversary of the grant date and the remainder vesting ratably at the end of each three-month period thereafter over the remaining three years, and options to purchase 25,000 shares will vest upon the later of the one year anniversary of the date of grant and the launch of Alnylam's first internally developed product, assuming in each case Mr. Soni remains continuously employed by Alnylam as of such date. Alnylam is providing this stock option information in accordance with NASDAQ Listing Rule 5635(c)(4). About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of patients who have limited or inadequate treatment options. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust discovery platform and deep pipeline of investigational medicines, including three product candidates that are in late-stage development or will be in 2017. Looking forward, Alnylam will continue to execute on its “Alnylam 2020” strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam.


News Article | May 9, 2017
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, announced today the appointment of several experienced industry leaders to key leadership roles including: Manmeet S. Soni, Chief Financial Officer; Theresa Heggie, Senior Vice President, Head of Europe and Canada; Peter Smith, Ph.D., Senior Vice President, Early Development; and Alan Eisenberg, Vice President, Global Public Policy and Government Relations. "We are thrilled to welcome Manmeet, Theresa, Peter, and Alan to Alnylam at an exciting moment in our history. Each of these individuals brings a critical set of skills to the organization as we transition from a late-stage research and development company to a multi-product, commercial-stage company with a robust and sustainable pipeline of innovative medicines,” said John Maraganore, Ph.D. Chief Executive Officer of Alnylam. "This expansion of our leadership team solidifies and strengthens our path forward." “I couldn’t imagine a more exciting time to join Alnylam,” said Mr. Soni. “It’s an honor to have the opportunity to work with such a talented group of individuals focused on bringing forward a new class of medicines for the betterment of lives of patients in need. I look forward to using my experience in building and leading commercial finance teams and capabilities to help Alnylam execute on its strategy, goals and transition towards an independent commercial-stage company.” In this role Mr. Soni will provide strategic leadership in the overall financial management of Alnylam, including for the global finance, investor relations and communications teams. Manmeet is the former Chief Financial Officer and Treasurer of ARIAD Pharmaceuticals, Inc., where he played a central role in the strategic review, turnaround and subsequent acquisition of ARIAD Pharmaceuticals, Inc. by Takeda Pharmaceuticals Company Limited. Before joining ARIAD Pharmaceuticals, Inc., Manmeet worked at Pharmacyclics, Inc., where he served most recently as Chief Financial Officer and Treasurer. Mr. Soni also played a vital role in the acquisition of Pharmacyclics, Inc. by Abbvie, Inc. for $21 billion. Previously, Mr. Soni worked at ZELTIQ Aesthetics Inc., and PricewaterhouseCoopers San Jose, in the Life Science and Venture Capital Group. Prior to that, he worked at PricewaterhouseCoopers, India. Mr. Soni is currently a board member and audit committee chair at Genoscience Pharma. He graduated from Hansraj College at Delhi University in India. He is a Certified Public Accountant, licensed in the state of California and a Chartered Accountant from India. "Expanding Alnylam’s operations into Europe and Canada is being done in recognition of the broad commercial rights we have in these regions and the important role they will play in our global commercial strategy," commented Ms. Heggie. "Drawing on my expertise from numerous European and global leadership roles, I look forward to advancing this important phase of the Company’s commercial evolution and bringing new treatments to patients in Europe and Canada." In this role Ms. Heggie will be responsible for the strategic direction and activity of all of Alnylam’s operations in Europe and Canada including building the go-to-market strategy across multiple products and therapeutic areas. Most recently she served as the Chief Marketing and Strategy Officer at The British United Provident Association (Bupa). Previously, Ms. Heggie held various senior commercial positions at Shire Human Genetic Therapies (and formerly TKT) including the roles of Vice President and General Manager of EMEA, Chief Executive Officer of Jerini AG (a Shire acquisition), and Senior Vice President of Global Commercial Operations. Earlier, at Baxter Healthcare she held numerous roles including Vice President of Global Marketing. Early in her career, Ms. Heggie held a variety of sales and marketing positions at Janssen Pharmaceuticals. She formerly served as a member of the board of directors of Swedish Orphan Biovitrum AB. Theresa received a BSc from Cornell University. Ms. Heggie will report to Barry Greene, President and will be based at Alnylam’s European headquarters in Zug, Switzerland. “Having the ability to work at an organization with a product engine as productive as Alnylam’s is a very special opportunity,” added Dr. Smith. “The ability to grow and develop the Early Development team across the pipeline of RNAi therapeutics at Alnylam will be paramount as we advance the translation of promising science toward new medicines for patients.” In this role, Dr. Smith will be responsible for all aspects of non-clinical safety, drug metabolism and pharmacokinetics, bioanalysis and biomarker programs, providing both scientific and drug development leadership. Dr. Smith brings more than 30 years of pharma industry experience to Alnylam, most recently joining from Moderna, where he was Head of R&D Non-Clinical. He joined Moderna from Millennium Pharmaceuticals, where he most recently served as co-head of R&D and a member of the company’s management team. In this role, he was responsible for management of all Non-Clinical groups and Pharmaceutical Sciences. His extensive experience in drug discovery and development spans multiple therapeutic areas and therapeutic modalities. Over the course of Dr. Smith’s career, he has had oversight of the non-clinical development of multiple, currently marketed therapeutics including CELEBREX®, INSPRA®, VELCADE® and ENTYVIO®, and also deep involvement in the development of numerous other products. Dr. Smith has a B.S. in biology from Fairfield University and a Ph.D. in Pharmacology and Toxicology from the University of Arizona. His postdoctoral fellowship in biochemical toxicology was undertaken at SmithKline. He has published and presented extensively in the pharm/tox area as well as in the area of drug development. Dr. Smith will report to Akshay Vaishnaw, Executive Vice President of Research and Development. “In an era of intense scrutiny around value and access to innovation, I look forward to drawing on my political and policy experience both in the private and public sectors to help Alnylam achieve its objectives,” said Mr. Eisenberg. “I’m deeply aligned with the mission and vision of Alnylam and look forward to working on behalf of the company with our governmental stakeholders globally.” In this role, Alan will lead federal, state and local government affairs and public policy initiatives globally. Alan joins Alnylam from Celgene where he was the Vice President for Federal Government Relations. In this role, he led Celgene’s Federal Government Relations function and had direct responsibility for the Company’s public policy engagement with Congress, relevant Executive Branch agencies and other Washington, D.C. based stakeholders. Prior to Celgene, Mr. Eisenberg was Executive Vice President for Emerging Companies & Business Development at the Biotechnology Innovation Organization (BIO), leading BIO’s services and advocacy efforts for BIO’s pre-market and early stage commercial companies, in addition to serving in other senior leadership roles at BIO. Previously, Mr. Eisenberg served as Health and Economics Policy Advisor to Congressman Jim Greenwood and prior to that, he served on the staff of the Senate HELP Public Health Subcommittee, and also was a legislative assistant for Congressman John Shadegg. Earlier in his career he spent four years with Ford Motor Company. Mr. Eisenberg holds a Master in Public Policy degree from Harvard University, a Master of Science in Finance degree from George Washington University, and a Bachelor of Science degree from Union College. Mr. Eisenberg will report to Laurie Keating, Senior Vice President and General Counsel. Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of patients who have limited or inadequate treatment options. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically-validated approach for the treatment of a wide range of debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust discovery platform and deep pipeline of investigational medicines, including three product candidates that are in late-stage development or will be in 2017. Looking forward, Alnylam will continue to execute on its “Alnylam 2020” strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam. Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, Alnylam's views with respect to the potential for RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, Alnylam's ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of its product candidates, the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all, actions or advice of regulatory agencies, including actions by regulators concerning product candidates, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, Alnylam's ability to enforce its intellectual property rights against third parties and defend its patent portfolio against challenges from third parties, obtaining and maintaining regulatory approval, pricing and reimbursement for products, progress in establishing a commercial and ex-United States infrastructure, competition from others using technology similar to Alnylam's and others developing products for similar uses, Alnylam's ability to manage its growth and operating expenses, obtain additional funding to support its business activities, and establish and maintain strategic business alliances and new business initiatives, Alnylam's dependence on third parties for development, manufacture and distribution of products, the outcome of litigation, the risk of government investigations, and unexpected expenditures, as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today reported financial and operational results for the first quarter ended March 31, 2017. “While continuing to focus on our research and development activities during the first quarter, we announced a major $70.0 million convertible preferred stock financing, which subsequently closed on April 11, 2017,” said Douglas Fambrough, Ph.D., president and chief executive officer of Dicerna. “The financing, led by Bain Capital Life Sciences and a syndicate of current and new investors, lends an additional level of validation to the potential of our proprietary GalXC™ RNAi technology platform. Furthermore, the funds, when combined with cash already on-hand, provide us with the necessary resources to execute our stated strategy, which includes pursuing the advancement of our core therapeutic programs. Specifically, as we look forward from today and into 2019, we expect to be able to advance our first three development programs, including DCR-PHXC, a second undisclosed rare disease program and DCR-HBV, into proof of concept studies, while progressing DCR-PCSK9 toward formal preclinical development, resulting in a number of key, value-creating inflection points. In the nearer term, I look forward to presenting new preclinical data for DCR-PHXC later this year and to our expected Investigational New Drug (IND) or Clinical Trial Application (CTA) filing at the end of 2017.” For more detailed information and analysis, see Dicerna’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2017, which was filed with the Securities and Exchange Commission (SEC) on May 8, 2017. With the closing of its Preferred Stock transaction, Dicerna believes that it has sufficient cash to fund the execution of its current operating plan into 2019, which includes focusing its resources on advancing its first three development programs into proof of concept studies and a fourth program into formal preclinical development. This estimate assumes no additional funding from new partnership agreements or from additional financing events. Management will host a conference call at 4:30 p.m. ET today to review the Company's first quarter 2017 financial results and provide a general business update. The conference call can be accessed by dialing (855) 453-3834 or (484) 756-4306 (international), and referencing conference ID 7812180 prior to the start of the call. The call will also be webcast via the Internet and will be available under the “Investors & Media” section of the Dicerna website, www.dicerna.com. A replay of the call will be available beginning at 7:30 p.m. ET on May 8, 2017. To access the replay, please dial (855) 859-2056 or (404) 537-3406, and refer to conference ID 7812180. The webcast will also be archived on the Company's website. Dicerna Pharmaceuticals, Inc., is a biopharmaceutical company focused on the discovery and development of innovative RNAi-based therapeutics for diseases involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases. The Company is leveraging its proprietary GalXC™ RNAi technology platform to build a broad pipeline in these core therapeutic areas, focusing on target genes where connections between target gene and diseases are well understood and documented. The Company intends to discover, develop and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners. For more information, please visit www.dicerna.com. GalXCTM is a proprietary technology platform invented by Dicerna to discover and develop next-generation RNAi-based therapies designed to silence disease-driving genes in the liver. Compounds produced via GalXC are intended to be broadly applicable across multiple therapeutic areas, including rare diseases, chronic liver diseases, cardiovascular disease and viral infectious diseases. Using GalXC, Dicerna scientists attach N-acetylgalactosamine (GalNAc) sugars directly to the extended region of our proprietary Dicer substrate short-interfering RNA molecules, yielding multiple proprietary conjugate delivery configurations. Many of the conjugates produced via GalXC incorporate a folded motif known as a tetraloop in the extended region. The tetraloop configuration, which is unique to Dicerna’s GalXC compounds, allows flexible and efficient conjugation to the targeting ligands, and stabilizes the RNAi duplex which we believe will enable subcutaneous delivery of Dicerna's RNAi therapies to hepatocytes in the liver, where they are designed to specifically bind to receptors on target cells, potentially leading to internalization and access to the RNAi machinery within the cells. The technology may offer several distinct benefits, as suggested by strong preclinical data. These benefits include: potency that is on par with or better than comparable platforms; highly specific binding to gene targets; long duration of action; and an infrequent subcutaneous dosing regimen. This press release includes forward-looking statements, including, for example, our anticipated use of proceeds from the Preferred Stock transaction, expected timeline and plans for development, potential collaborations, and potential therapeutic benefits. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include risks relating to our clinical and preclinical research and other risks identified under the heading "Risk Factors" included in our most recent Form 10-Q filing and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Dicerna's current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to update any forward-looking statements.


DUBLIN, May. 10, 2017 /PRNewswire/ -- Research and Markets has announced the addition of Jain PharmaBiotech's new report "RNAi - Technologies, Markets and Companies" to their offering. The markets for RNAi are difficult to define as no RNAi-based product is approved yet but...


"During the first quarter of 2017, RXi has been integrating MirImmune into its existing R&D operations. Through careful planning and execution of this integration, the Company has been able to maintain its cash use in line with that of last year," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He also commented that, "RXi has added two new executives to its senior management team who will be instrumental in driving the development of novel therapeutics. Dr. Gerrit Dispersyn as Chief Development Officer and Dr. Alexey Eliseev as Chief Business Officer will provide significant leadership in the new and exciting space of immuno-oncology and cell therapy, while we continue to advance our dermatology and ophthalmology trials toward our planned readouts later this year."   He further added that, "Recently, RXi also announced that Dr. Pamela Pavco will retire this month. During her tenure as Chief Development Officer, she has been an integral part of the development of RXi's technology platform and clinical programs.  We are grateful that she will remain involved with the Company as part of its Scientific Advisory Board where her expertise in the field of oligonucleotides will continue to support the advancement of our development pipeline." The Company will host a conference call today at 4:30 p.m. EDT to discuss financial results and provide an update on the Company. The webcast link will be available under the "Investors – Event Calendar" section of the Company's website, www.rxipharma.com. The event may also be accessed by dialing toll-free in the United States +1 844-376-4678. International participants may access the event by dialing: +1 209-905-5958. An archive of the webcast will be available on the Company's website approximately two hours after the presentation. At March 31, 2017, the Company had cash of $10.2 million, compared with $12.9 million at December 31, 2016. The Company believes that its existing cash should be sufficient to fund operations for at least the next twelve months. Research and development expenses for the quarter ended March 31, 2017 was $1.3 million, which included less than $0.1 million of non-cash stock-based compensation expense, as compared with $1.3 million for the quarter ended March 31, 2016, which included $0.1 million of non-cash stock-based compensation expense. Research and development expenses were consistent quarter over quarter, but did see slight increases due to the commencement of the Company's new immunotherapy program with the acquisition of MirImmune Inc. ("MirImmune"), a privately-held biotechnology company that was engaged in the development of cancer immunotherapies, in January 2017, which were offset by a decrease in stock-based compensation expense. The Company had acquired in-process research and development expense of $3.0 million for the quarter ended March 31, 2017. There was no such expense for the three months ended March 31, 2016. The expense related to the Company's acquisition of MirImmune in January 2017. Per the terms of the acquisition, the Company acquired all of the outstanding capital stock of MirImmune in exchange for shares of the Company's common stock and Series C Convertible Preferred Stock, which were subject to a 3% holdback for any purchase price adjustments. The fair value of the consideration given during the quarter totaling $3.0 million was expensed as in-process research and development expense. The fair value of the securities subject to the 3% holdback, which were released and issued on April 12, 2017, will be recorded as in-process research and development expense during the second quarter of 2017. General and administrative expenses for the quarter ended March 31, 2017 were $1.1 million, which included $0.1 million of non-cash stock-based compensation expense, as compared with $1.0 million for the quarter ended March 31, 2016, which included $0.2 million of non-cash stock-based compensation expense. The increase in general and administrative expenses was due to an increase in employee headcount with the hire of the Company's Chief Business Officer as part of the acquisition of MirImmune and an increase in legal fees, offset by a decrease in stock-based compensation expense. Net loss for the three months ended March 31, 2017 was $5.5 million, compared with $2.2 million for the three months ended March 31, 2016. The increase in net loss was primarily driven by the one-time charge of $3.0 million of acquired in-process research and development expense related to the acquisition of MirImmune in January 2017. Management Team:  The Company announced that Dr. Pamela Pavco, RXi's Chief Development Officer, will retire effective May 19, 2017.  At that time, Dr. Pavco will join the Company's Scientific Advisory Board where her expertise in the field of oligonucleotides and the development of RNAi therapeutics will continue to support RXi's ongoing research and development initiatives. On April 24, 2017, Dr. Gerrit Dispersyn was appointed as RXi's new Chief Development Officer.  Dr. Dispersyn brings a wealth of experience to RXi as an accomplished leader in clinical, product and business development. Intellectual Property Estate: The Company has been diligent and proactive with its approach to broadly protect its valuable corporate assets by securing patent protection for its RNAi platform and Samcyprone™, a small molecule that is a proprietary ointment formulation of diphenylcyclopropenone (DPCP). Most recently, the Company was granted a patent by the Japan Patent Office (JPO) for the composition of matter of sd-rxRNAs targeting connective tissue growth factor (CTGF) for the treatment or prevention of fibrotic disorders, including but not limited to skin fibrosis and proliferative vitreoretinopathy.  This patent includes the Company's lead clinical candidate RXI-109, an sd-rxRNA® therapeutic compound, which is currently being evaluated in Phase 2 clinical trials. Our portfolio currently includes 79 issued patents and 60 pending applications.  This includes coverage in the United States, Canada, Europe, Japan and other markets. In addition, Samcyprone™ has been granted orphan-drug designation for malignant melanoma stage IIb to IV.  The Company's intellectual property estate provides for numerous commercial, regional and strategic partnering opportunities. Immuno-oncology:  The Company's ongoing program to develop cell-based immunotherapies to treat cancer is based on our proprietary self-delivering RNAi (sd-rxRNA) technology platform.  RXi's novel sd-rxRNA technology differs from natural and most synthetic RNA interference (RNAi) molecules in that they are chemically modified to allow for an easy internalization of the compounds by most types of cells and silencing of the targeted genes. sd-rxRNA offers unprecedented flexibility in targeting immunosuppressive pathways with the potential to modulate multiple checkpoint genes in a single therapeutic treatment.  The built-in delivery and therapeutic properties of sd-rxRNA lend themselves well for local therapeutic applications, such as ex vivo treatment of the immune cells.  The ex vivo use of sd-rxRNA to pre-treat immune cells prior to infusion may prove advantageous as an immuno-therapeutic in that there is the potential to simultaneously reduce multiple checkpoints or targets, including both intracellular and extracellular targets, with little change to current protocols. As outlined at the beginning of the year in our 2017 Corporate Goals, we are actively working on multiple checkpoint-inhibiting sd-rxRNA compounds co-transfected in CAR T-cells in mouse models for solid tumors and conducting preclinical studies on the use of sd-rxRNA with tumor infiltrating lymphocytes (TILs) in melanoma.  Data from this ongoing work is expected to be available in the second half of 2017. RXI-109-1402 – Hypertrophic Scarring:  The Company's ongoing Phase 2 clinical trial, RXI-109-1402, is being conducted to evaluate its first clinical candidate RXI-109, an sd-rxRNA compound targeting connective tissue growth factor (CTGF) to reduce scar formation in the skin following scar revision surgery. The Company will provide a full read-out, for Cohorts 3 and 4, in the second half of 2017. RXI-109-1501 – Retinal Scarring in Advanced Age-related Macular Degeneration (AMD):  As in dermal scarring, CTGF is known to play a role in retinal scarring.  Reduction of CTGF in the eye by RXI-109 treatment may reduce the formation of retinal fibrosis that often accompanies late stage AMD and contributes to permanent vision loss.  Enrollment in the first two cohorts in the Company's Phase 1/2 trial, RXI-109-1501, is complete.  RXI-109 has been well-tolerated in the eye to date; enrollment into the third cohort at the next higher dose level is ongoing. RXI-SCP-1502 – Treatment of Cutaneous Warts:  Samcyprone™, the Company's second clinical candidate, is being evaluated in a Phase 2a clinical trial. RXI-SCP-1502 is a multi-center, multi-dose trial conducted in subjects with at least one cutaneous, plantar or periungual wart.  The Company expects to share early read-outs in the second half of 2017. Consumer/Functional Health Care:  The Company presented an update on its consumer health program at the 76th annual meeting of the Society for Investigative Dermatology.  RXI-231, an sd-rxRNA targeting tyrosinase, is in development as a cosmetic ingredient that may improve the appearance of uneven skin tone and pigmentation.  RXi has developed a proprietary formulation that allows for the non-invasive penetration of sd-rxRNA compounds to the epidermal-dermal junction where the reduction of tyrosinase in the resident melanocytes would lead to a reduction of melanin. We are in the process of finalizing the first two protocols for testing in volunteers. RXI-185, an sd-rxRNA targeting MMP1, is in development as a cosmetic ingredient that may improve the appearance of wrinkles, skin laxity or photo-aging. A study conducted in collaboration with DSM, showed that RXI-185 is capable of reducing MMP1 mRNA levels following topical administration in an ex vivo human skin model.  A topical formulation of RXI-185 is currently in development to lessen the effects of photo-aging. RXi Pharmaceuticals Corporation (NASDAQ: RXII) is a clinical-stage company developing innovative therapeutics that address significant unmet medical needs. Building on the pioneering discovery of RNAi, scientists at RXi have harnessed the naturally occurring RNAi process which has the ability to "silence" or down-regulate the expression of a specific gene that may be overexpressed in a disease condition. RXi developed a robust RNAi therapeutic platform including self-delivering RNA (sd-rxRNA®) compounds, that have the ability to selectively block the expression of any target in the genome, thus providing applicability to many therapeutic areas. Our current programs include dermatology, ophthalmology and cell-based cancer immunotherapy. RXi's extensive patent portfolio provides for multiple product and business development opportunities across a broad spectrum of therapeutic areas and we actively pursue research collaborations, partnering and out-licensing opportunities with academia and pharmaceutical companies. Additional information may be found on the Company's website, www.rxipharma.com. This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements about: our ability to successfully develop RXI-109, Samcyprone™ and our other product candidates (collectively "our product candidates"); the future success of our clinical trials with our product candidates; the timing for the commencement and completion of clinical trials; our ability to enter into strategic partnerships and the future success of these strategic partnerships; and our ability to deploy our sd-rxRNA® technology through partnerships, as well as the prospects of these partnerships to provide positive returns. Forward-looking statements about expectations and development plans of RXi's product candidates and partnerships involve significant risks and uncertainties, including the following: risks that we may not be able to successfully develop and commercialize our product candidates; risks that product development and clinical studies may be delayed, not proceed as planned and/or be subject to significant cost over-runs; risks related to the development and commercialization of products by competitors; risks related to our ability to control the timing and terms of collaborations with third parties; and risks that other companies or organizations may assert patent rights preventing us from developing or commercializing our product candidates. Additional risks are detailed in our most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q under the caption "Risk Factors."  Readers are urged to review these risk factors and to not act in reliance on any forward-looking statements, as actual results may differ from those contemplated by our forward-looking statements. RXi does not undertake to update forward-looking statements to reflect a change in its views, events or circumstances that occur after the date of this release. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/rxi-pharmaceuticals-reports-first-quarter-2017-financial-results-and-recent-corporate-highlights-300455836.html


News Article | May 10, 2017
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that management will present company overviews at the Bank of America Merrill Lynch 2017 Healthcare Conference on Thursday, May 18, 2017 at 8:40 am PT (11:40 am ET) at the Encore at Wynn Las Vegas in Las Vegas, Nevada, and at the UBS Global Healthcare Conference on Wednesday, May 24, 2017 at 10:00 am ET at the Grand Hyatt New York in New York City. A live audio webcast of each presentation will be available on the Investors section of the company’s website, www.alnylam.com. A replay will be available on the Alnylam website within 48 hours after each event. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of patients who have limited or inadequate treatment options. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust discovery platform and deep pipeline of investigational medicines, including three product candidates that are in late-stage development or will be in 2017. Looking forward, Alnylam will continue to execute on its “Alnylam 2020” strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam.


Gene therapy can now combine with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression. Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included. The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2016-2026. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets Profiles of 187 companies involved in developing gene therapy are presented along with 232 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. Key Topics Covered: Part I: Technologies & Markets Executive Summary 12. References Part II: Companies 13. Companies involved in Gene Therapy For more information about this report visit http://www.researchandmarkets.com/research/9jzl3f/gene_therapy Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-gene-therapy-technologies-markets-and-companies-research-report-2017-2026---research-and-markets-300455149.html


A double-stranded nucleic acid molecule including (a) a sense strand which includes a nucleotide sequence corresponding to a target sequence indicated by any one of SEQ ID Nos.: 1 to 21, and (b) an antisense strand which includes a nucleotide sequence complementary to that of the sense strand specified in (a), wherein the double-stranded nucleic acid molecule is for suppressing the expression of at least one of APP and EBAG9 genes.


The present invention aims to provide a double-stranded nucleic acid molecule (e.g., siRNA) which can effectively inhibit the proliferation of uterine, breast and bladder cancer cells by suppressing the expression of at least one target gene of COX7RP and Efp genes (estrogen-responsive genes); a cancer cell proliferation inhibitor; and a pharmaceutical agent. Specifically, the present invention relates to a double-stranded nucleic acid molecule for suppressing the expression of at least one of COX7RP and Efp genes which includes (a) a sense strand which includes a nucleotide sequence corresponding to a target sequence indicated by any one of SEQ ID Nos.: 1 to 38 and (b) an antisense strand which includes a nucleotide sequence complementary to that of the sense strand specified in (a); a cancer cell proliferation inhibitor with the double-stranded nucleic acid molecule and against at least one of uterine, breast and bladder cancer cells; and a pharmaceutical agent with the cancer cell proliferation inhibitor and against at least one of uterine, breast and bladder cancers.


A double-stranded nucleic acid molecule including (a) a sense strand which includes a nucleotide sequence corresponding to a target sequence indicated by any one of SEQ ID Nos.: 1 to 21, and (b) an antisense strand which includes a nucleotide sequence complementary to that of the sense strand specified in (a), wherein the double-stranded nucleic acid molecule is for suppressing the expression of at least one of APP and EBAG9 genes.

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