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First Trust NYSE Arca Biotech ETF ( NYSEArca: FBT ) The investment seeks investment results that correspond generally to the price and yield (before the fund's fees and expenses) of an equity index called the NYSE Arca Biotechnology Index(SM). The fund will normally invest at least 90% of its net assets plus the amount of any borrowings for investment purposes in common stocks that comprise the index. The index is an equal-dollar weighted index designed to measure the performance of a cross section of companies in the biotechnology industry that are primarily involved in the use of biological processes to develop products or provide services. The fund is non-diversified.
iShares US Healthcare ( NYSEArca: IYH ) The investment seeks to track the investment results of an index composed of U.S. equities in the healthcare sector. The fund generally invests at least 90% of its assets in securities of the underlying index and in depositary receipts representing securities of the underlying index. It seeks to track the investment results of the Dow Jones U.S. Health Care Index (the "underlying index"), which measures the performance of the healthcare sector of the U.S. equity market. The fund is non-diversified.
iShares US Healthcare Providers ( NYSEArca: IHF ) The investment seeks to track the investment results of an index composed of U.S. equities in the healthcare providers sector. The fund generally invests at least 90% of its assets in securities of the underlying index and in depositary receipts representing securities of the underlying index. It seeks to track the investment results of the Dow Jones U.S. Select Health Care Providers Index (the "underlying index"), which measures the performance of the healthcare providers sector of the U.S. equity market. The fund is non-diversified.
iShares US Medical Devices ( NYSEArca: IHI ) The investment seeks to track the investment results of an index composed of U.S. equities in the medical devices sector. The fund seeks to track the investment results of the Dow Jones U.S. Select Medical Equipment Index (the "underlying index"), which measures the performance of the medical equipment sector of the U.S. equity market. The underlying index includes medical equipment companies such as manufacturers and distributors of medical devices such as magnetic resonance imaging (MRI) scanners, prosthetics, pacemakers, X-ray machines, and other non-disposable medical devices. The fund is non-diversified.
iShares Nasdaq Biotechnology ( NasdaqGIDS IBB ) The investment seeks to track the investment results of an index composed of biotechnology and pharmaceutical equities listed on the NASDAQ. The fund generally invests at least 90% of its assets in securities of the underlying index and in depositary receipts representing securities of the underlying index. The underlying index contains securities of NASDAQ® listed companies that are classified according to the Industry Classification Benchmark as either biotechnology or pharmaceuticals and that also meet other eligibility criteria determined by the NASDAQ OMX Group, Inc. The fund is non-diversified.
Market Vectors Biotech ETF ( NYSE MKT:BBH ) seeks to replicate as closely as possible, before fees and expenses, the price and yield performance of the Market Vectors® US Listed Biotech 25 Index. The fund normally invests at least 80% of its total assets in securities that comprise the fund&#39;s benchmark index. The Biotech Index is comprised of common stocks and depositary receipts of U.S. exchange-listed companies in the biotechnology sector. Such companies may include medium-capitalization companies and foreign companies that are listed on a U.S. exchange. It is non-diversified.
PowerShares Dynamic Biotech & Genome ETF ( NYSEArca: PBE ) The investment seeks investment results that generally correspond (before fees and expenses) to the price and yield of the Dynamic Biotechnology & Genome IntellidexSM Index. The fund generally will invest at least 90% of its total assets in common stocks of biotechnology companies and genome companies that comprise the underlying intellidex. The underlying intellidex was composed of common stocks of 30 U.S. biotechnology and genome companies. These companies are engaged principally in the research, development, manufacture and marketing and distribution of various biotechnological products, services and processes, etc. It is non-diversified.
4SC (XETRA: VSC.DE) is an innovative biotech company with a strong focus on clinical development. We discover and develop targeted small molecule drugs with an epigenetic mode of action for the treatment of cancer in indications with a high unmet medical need and major economic potential.
Aastrom Biosciences, Inc. (NasdaqCM: ASTM) is the leader in developing patient-specific expanded cellular therapies for use in the treatment of patients with severe diseases and conditions. Aastrom markets two autologous cell therapy products in the United States for the treatment of cartilage repair and skin replacement. Aastrom is also developing MACI(TM), a third-generation autologous chondrocyte implant for the treatment of cartilage defects in the knee, and ixmyelocel-T, a patient-specific multicellular therapy for the treatment of advanced heart failure due to ischemic dilated cardiomyopathy.
ABBOTT LABORATORIES (NYSE: ABT) is a global healthcare company devoted to improving life through the development of products and technologies that span the breadth of healthcare. With a portfolio of leading, science-based offerings in diagnostics, medical devices, nutritionals and branded generic pharmaceuticals, Abbott serves people in more than 150 countries and employs approximately 69,000 people.76
Abeona Therapeutics Inc. (NasdaqCM:ABEO) develops and delivers gene therapy and plasma-based products for severe and life-threatening rare diseases. Abeona's lead programs are AB0-101 (AAV NAGLU) and ABO-102 (AAV SGSH), adeno-associated virus (AAV)-based gene therapies for Sanfilippo syndrome (MPS IIIB and IIIA). We are also developing ABO-201 (AAV CLN3) gene therapy for juvenile Batten disease (JBD); and ABO-301 (AAV FANCC) for Fanconi anemia (FA) disorder using a novel CRISPR/Cas9-based gene editing approach to gene therapy program for rare blood diseases. In addition, we are also developing rare plasma protein therapies including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD using our proprietary SDF™ (Salt Diafiltration) ethanol-free process.
Ablynx (Brussels: ABLX.BR) is a biopharmaceutical company engaged in the discovery and development of Nanobodies®, a novel class of therapeutic proteins based on single-domain antibody fragments, for a range of serious and life-threatening human diseases, including inflammation, thrombosis, oncology and pulmonary disease.
Acadia Pharmaceuticals Inc. (NasdaqGS: ACAD) is a biopharmaceutical company focused on the development and commercialization of innovative medicines that address unmet medical needs in neurological and related central nervous system disorders. ACADIA has a pipeline of product candidates led by pimavanserin, for which we have reported positive Phase III trial results in Parkinson's disease psychosis and which has the potential to be the first drug approved in the United States for this disorder. We are currently completing NDA-enabling clinical and manufacturing activities for pimavanserin and are planning to submit an NDA with the FDA near the end of 2014. Pimavanserin is also in Phase II development for Alzheimer's disease psychosis and has successfully completed a Phase II trial in schizophrenia. ACADIA also has clinical-stage programs for chronic pain and glaucoma in collaboration with Allergan, Inc. and two preclinical programs directed at Parkinson's disease and other neurological disorders. All product candidates are small molecules that emanate from internal discoveries.
Access Pharmaceuticals, Inc. (OTC:ACCP) is an emerging biopharmaceutical company that develops and commercializes proprietary products for the treatment and supportive care of cancer patients. Access developed MuGard and ProctiGard and is developing multiple follow-on products. Access also has other advanced drug delivery technologies including CobaCyte™-mediated targeted delivery and CobOral-oral drug delivery, its proprietary nanopolymer delivery technology based on the natural vitamin B12 uptake mechanism.
AcelRx Pharmaceuticals, Inc. (NasdaqGM:ACRX) is a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of moderate-to-severe acute pain. The Company's late-stage pipeline includes ARX-04 (sufentanil sublingual tablet, 30 mcg), designed for the treatment of moderate-to-severe acute pain in medically supervised settings; and Zalviso® (sufentanil sublingual tablet system), designed for the management of moderate-to-severe acute pain in adult patients in the hospital setting. Zalviso delivers 15 mcg sufentanil sublingually through a non-invasive delivery route via a pre-programmed, patient-controlled analgesia device. Zalviso is approved in the EU as well as Norway, Iceland, and Liechtenstein and is investigational and in late-stage development in the U.S. Grunenthal Group holds the rights for Zalviso in Europe and Australia, while AcelRx retains all other world-wide rights.
Acorda Therapeutics, Inc. (NasdaqGS:ACOR) is a biotechnology company focused on developing therapies that improve the lives of people with neurological disorders. Acorda markets three FDA-approved therapies including: AMPYRA (dalfampridine) Extended Release Tablets, 10 mg, a treatment to improve walking in patients with multiple sclerosis (MS); ZANAFLEX CAPSULES® (tizanidine hydrochloride) and Zanaflex tablets, a short-acting drug for the management of spasticity; and QUTENZA® (capsaicin) 8% Patch, for the management of neuropathic pain associated with postherpetic neuralgia. AMPYRA is marketed outside the United States as FAMPYRA® (prolonged-release fampridine tablets) by Biogen Idec under a licensing agreement from Acorda. Acorda has one of the leading pipelines in the industry of novel neurological therapies. The Company is currently developing six clinical-stage therapies and one preclinical stage therapy that address a range of disorders including post-stroke deficits, epilepsy, stroke, peripheral nerve damage, spinal cord injury, neuropathic pain, and heart failure.
Acrux Limited (ASX:ACR.AX) is a dynamic Australian drug delivery business developing and commercialising a range of patient-preferred pharmaceutical products for global markets, using innovative, patented technology to administer drugs through the skin. Fast drying, non-occlusive topical sprays or liquids provide an enhanced transdermal delivery platform with low or no skin irritation, superior cosmetic acceptability, and simple, accurate and flexible dosing.See the full stock directory here
EMA Grants Orphan Designation
RESEARCH TRIANGLE PARK, N.C., May 24, 2018 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX), a pharmaceutical company focused on the development and commercialization of treatments for rare diseases, today announced that the European Medicines Agency’s (“EMA”) Committee for Orphan Medicinal Products (“COMP”) issued a positive opinion on BioCryst’s application for orphan designation of BCX7353 for the treatment of hereditary angioedema (“HAE”). In addition, the United Kingdom’s Medicines and Healthcare products Regulatory Agency (“MHRA”) has granted a Promising Innovative Medicine (“PIM”) designation to BCX7353.
The positive opinion issued by COMP is expected to be adopted by the European Commission within 30 days. Orphan Drug Designation in Europe is available to companies developing products intended to treat a life-threatening or chronically debilitating condition that affects fewer than five in 10,000 persons in the European Union (“EU”). This designation allows for financial and regulatory incentives that include a 10-year period of marketing exclusivity in the EU after product approval. BCX7353 has previously received Orphan Drug Designation in the United States from the Food and Drug Administration.
A PIM designation is an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme (“EAMS”) in the United Kingdom, which supports products intended for the treatment, diagnosis or prevention of a life-threatening or seriously debilitating condition, with the potential to address an unmet medical need.
“We are pleased to receive these important regulatory designations granted to BCX7353. These provide further support for the potential for BCX7353 to address a clear unmet medical need with an oral treatment for patients with HAE,” said Jon P. Stonehouse, President & Chief Executive Officer.
Discovered by BioCryst, BCX7353 is a novel, oral, once-daily, selective inhibitor of plasma kallikrein currently in development for the prevention and treatment of angioedema attacks in patients diagnosed with HAE. BCX7353 has been generally safe and well tolerated in the Phase 2 APeX-1 clinical trial. BioCryst is currently conducting the Phase 3 APeX-2 clinical trial and the long-term safety APeX-S clinical trial, both evaluating two dosage strengths of BCX7353 administered orally once-daily as a preventive treatment to reduce the frequency of attacks in patients with HAE. BioCryst is also conducting the ZENITH-1 clinical trial. ZENITH-1 is a proof-of-concept Phase 2 clinical trial testing an oral liquid formulation of BCX7353 for the treatment of acute angioedema attacks.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals designs, optimizes and develops novel small-molecule medicines that address both common and rare conditions. BioCryst has several ongoing development programs including BCX7353, an oral treatment for hereditary angioedema, galidesivir, a potential treatment for filoviruses, and a preclinical program to develop oral Alk-2 inhibitors for the treatment of fibrodysplasia ossificans progressive (“FOP”). RAPIVAB® (peramivir injection), a viral neuraminidase inhibitor for the treatment of influenza, is BioCryst's first approved product and has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan, Korea and the European Union. Post-marketing commitments for RAPIVAB are ongoing. For more information, please visit the Company's website at www.BioCryst.com.
This press release contains forward-looking statements, including statements regarding future results, performance or achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause BioCryst’s actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: that developing any HAE product candidate may take longer or may be more expensive than planned; that ongoing and future preclinical and clinical development of HAE drug candidates (including ZENITH-1, APeX-2 and APeX-S) may not have positive results; that BioCryst may not be able to enroll the required number of subjects in planned clinical trials of product candidates; that the Company may not advance human clinical trials with product candidates as expected; that the FDA, EMA or other applicable regulatory agency may require additional studies beyond the studies planned for product candidates, or may not provide regulatory clearances which may result in delay of planned clinical trials, or may impose a clinical hold with respect to such product candidate, or withhold market approval for product candidates. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, all of which identify important factors that could cause the actual results to differ materially from those contained in BioCryst’s projections and forward-looking statements.
CONTACT: Thomas Staab, BioCryst Pharmaceuticals, +1-919-859-7910
Shares of Common Stock Will Begin Trading on Split-Adjusted Basis on May 25, 2018
EXTON, Pa., May 24, 2018 (GLOBE NEWSWIRE) -- Fibrocell Science, Inc. (NASDAQ:FCSC), a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, today announced that it will effect a one-for-five reverse stock split of its common stock at 5:00 pm ET today. Beginning with the opening of trading on May 25, 2018, the Company’s common stock will trade on The Nasdaq Capital Market on a split-adjusted basis under a new CUSIP number 315721407.
Fibrocell’s stockholders approved an amendment authorizing the reverse stock split at its Annual Meeting of Stockholders on May 23, 2018. The specific one-for-five ratio was subsequently approved by the Company's Board of Directors and the reverse stock split was effected by filing a Certificate of Amendment to the Company's Restated Certificate of Incorporation with the Secretary of State of the State of Delaware.
The reverse stock split will be effected simultaneously for all outstanding shares of common stock and the ratio determined by the Board will be the same for all outstanding shares of common stock. The reverse stock split will affect all holders of shares of Fibrocell common stock uniformly and each stockholder will hold the same percentage of Fibrocell common stock outstanding immediately following the reverse stock split as that stockholder held immediately prior to the reverse stock split, except for adjustments that may result from the treatment of fractional shares as described below. The reverse stock split will not reduce the number of authorized shares of common stock, or preferred stock, or change the par values of Fibrocell common stock (which will remain at $0.001 per share) or preferred stock (which will remain at $0.001 per share). No fractional shares will be issued in connection with the reverse stock split. Any fractional share of common stock that would otherwise have resulted from the reverse stock split will be rounded up to the nearest whole share.
The reverse stock split is intended to increase the per share trading price of the Company’s common stock to permit the Company to regain compliance with the continued listing requirements of The Nasdaq Capital Market.
Information for Stockholders
Fibrocell’s transfer agent, Broadridge Corporate Issuer Solutions (BCIS), will act as exchange agent for the reverse stock split, and will provide stockholders of record holding certificates representing pre-split shares of the Company's common stock as of the effective date with a letter of transmittal providing instructions for the exchange of stock certificates for post-split shares. Stockholders owning shares via a broker or other nominee will have their positions automatically adjusted to reflect the reverse stock split, subject to the broker's or nominee's particular procedures for processing the reverse stock split, and will not be required to take any action in connection with the reverse stock split. BCIS may be reached by telephone at 1-877-830-4936.
Additional information about the reverse stock split can be found in Fibrocell’s definitive proxy statement, filed April 2, 2018, and Form 8-K filed today with the Securities and Exchange Commission, copies of which are available at www.sec.gov and at www.fibrocell.com under the SEC Filings tab located on the Investors page.
Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin and connective tissue. Fibrocell’s most advanced product candidate, FCX-007, is the subject of a Phase 1/2 clinical trial for the treatment of recessive dystrophic epidermolysis bullosa. Fibrocell is also developing FCX-013, the Company’s product candidate for the treatment of moderate to severe localized scleroderma. Fibrocell’s gene therapy portfolio is being developed in collaboration with Precigen, Inc., a wholly owned subsidiary of Intrexon Corporation (NYSE: XON), a leader in synthetic biology. For more information, visit http://www.fibrocell.com or follow Fibrocell on Twitter at @Fibrocell.
Fibrocell, the Fibrocell logo and Fibrocell Science are trademarks of Fibrocell Science, Inc. and/or its affiliates. All other names may be trademarks of their respective owners.
This press release contains, and Fibrocell’s officers and representatives may from time to time make, statements that are “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. All statements that are not historical facts are hereby identified as forward-looking statements for this purpose and include, among others, statements relating to Fibrocell’s ability to achieve the desired effect of increasing the stock price above the $1.00 closing bid price per share minimum for the requisite period so as to regain compliance with the Nasdaq continued listing requirement with respect to the trading price of its common stock.
Forward-looking statements are based upon management’s current expectations and assumptions and are subject to a number of risks, uncertainties and other factors that could cause actual results and events to differ materially and adversely from those indicated herein including, among others: the risk that the reverse stock split does not increase Fibrocell’s per share trading price above the minimum amount for the requisite period of time, risks and uncertainties inherent in Fibrocell’s business, and the risks, uncertainties and other factors discussed under the caption “Item 1A. Risk Factors” in Fibrocell’s most recent Form 10-K filing and Form 10-Q filings. As a result, you are cautioned not to place undue reliance on any forward-looking statements. While Fibrocell may update certain forward-looking statements from time to time, Fibrocell specifically disclaims any obligation to do so, whether as a result of new information, future developments or otherwise.
Investor & Media Relations Contact:
-- 1st global Phase 3 trial focused on synovial sarcoma patients
SEATTLE and SOUTH SAN FRANCISCO, Calif., May 24, 2018 (GLOBE NEWSWIRE) -- Immune Design (Nasdaq:IMDZ), an immunotherapy company focused on novel therapies in oncology, today announced the launch of the patient and healthcare provider websites for the SYNOVATE study – a pivotal trial to evaluate CMB305 immunotherapy in synovial sarcoma patients. Clinical sites will soon be open for enrollment initially in the US, with information available at www.synovatestudy.com.
“Patients with advanced synovial sarcoma often have few systemic treatment options immediately after they complete first line chemotherapy. Clinically, we often give them a break from therapy, following closely with the hope that they will not progress in the relatively short time period that is unfortunately common in this disease,” said William D. Tap, M.D., Medical Oncology and Chief, Sarcoma Medical Oncology Service, Memorial Sloan Kettering Cancer Center. “It is our hope that CMB305 may offer a new option for patients to receive a potentially beneficial therapy that will defer the need for subsequent therapy.”
SYNOVATE is a randomized, global Phase 3 clinical trial that will evaluate CMB305 monotherapy versus placebo in 248 patients 12 years of age and older with NY-ESO-1 positive, unresectable, locally-advanced or metastatic synovial sarcoma. Patients who are responding to a first-line therapy can become eligible for SYNOVATE following completion of their chemotherapy. SYNOVATE will be opened and recruiting patients in cancer centers throughout the United States, Canada, Europe, and the Asia Pacific region.
“We are very pleased to be working with high quality clinical groups around the country and internationally to launch the SYNOVATE study to explore CMB305 immunotherapy in patients with synovial sarcoma,” said Sergey Yurasov, M.D., Ph.D., Chief Medical Officer, Immune Design. “SYNOVATE is the first randomized, global Phase 3 trial of an immunotherapy focused in synovial sarcoma, and we hope it results in a new, approved therapy for these patients.”
Patient and Health Care Provider Resouces for SYNOVATE Study
About Synovial Sarcoma
Soft tissue sarcomas are malignancies that arise from the soft tissues of the body, such as tissues that connect, support and surround other body structures including muscle, fat, blood vessels, nerves, tendons and the lining of joints. Synovial sarcoma is a sub type of soft tissue sarcoma where 70% of diagnoses occur in patients under 40 years old, is associated with a high risk of recurrence, and has been shown to have high expression of the NY-ESO-1 tumor antigen. The primary treatment for patients with locally advanced, unresectable or metastatic synovial sarcoma typically consists of an anthracycline-based chemotherapy regimen administered alone or in combination with other agents. Following disease progression after first line systemic therapy, treatment options are limited and median overall survival rates have been reported to be approximately 12 months. In connection with the planned Phase 3 study for CMB305 monotherapy, the FDA has agreed with Immune Design that synovial sarcoma patients constitute an unmet medical need.
CMB305 is a prime-boost cancer vaccine targeting NY-ESO-1-expressing tumors. NY-ESO-1 is a cellular protein that is typically found only in certain cancer cells and is often frequently expressed in synovial sarcomas. CMB305 is administered to participants by injection and is designed to provide clinical benefit by generating an anti-NY-ESO-1 immune response by activating a participant’s antigen-presenting dendritic cells.
About Immune Design
Immune Design is a late-stage immunotherapy company employing next-generation in vivo approaches to enable the body's immune system to fight disease. The company's technologies are engineered to activate the immune system's natural ability to generate and/or expand antigen-specific cytotoxic immune cells to fight cancer and other chronic diseases. CMB305 and G100, the leading product candidates with broad potential in oncology, are based on the company’s two technology platforms that are potent stimulators of the immune system – ZVex® and GLAAS® - the fundamental technologies of which were licensed from the California Institute of Technology and the Infectious Disease Research Institute (IDRI), respectively. Both ZVex and GLAAS also have potential applications in infectious disease and allergy indications, which are being developed through ongoing pharmaceutical collaborations. Immune Design has offices in Seattle and South San Francisco. For more information, please visit www.immunedesign.com.
Cautionary Note on Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “may,” “will,” “expect,” “plan,” “anticipate,” “target,” “estimate,” “intend” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Immune Design’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause Immune Design’s clinical development programs, future results or performance to differ significantly from those expressed or implied by the forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, scope and results of clinical trials and the timing and likelihood of obtaining regulatory approval of Immune Design’s product candidates. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, the uncertainties and timing of the regulatory approval process, and unexpected litigation or other disputes. Other factors that may cause Immune Design’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Immune Design’s filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” sections contained therein. Except as required by law, Immune Design assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
RESEARCH TRIANGLE PARK, N.C. and EXTON, Pa., May 24, 2018 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (“BioCryst”) (NASDAQ:BCRX), and Idera Pharmaceuticals, Inc. (“Idera”) (NASDAQ:IDRA), today announced that they will be presenting at the Jefferies 2018 Global Healthcare Conference on Thursday, June 7, 2018 at 8:30 A.M. E.T.
Links to a live audio webcast and replay of this presentation may be accessed in the Investors section of BioCryst’s website at http://www.biocryst.com and in the Investors and Media section of Idera’s website at http://www.iderapharma.com.
This press release contains forward-looking statements, including statements regarding future results and achievements. These statements involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially different from any future results, performances or achievements expressed or implied by the forward-looking statements. Please refer to the documents periodically filed by both BioCryst and Idera with the Securities and Exchange Commission.
Ligandal’s Bioinspired Nanomaterials Deliver CRISPR into Nucleus of a T-Cell
SAN FRANCISCO, May 23, 2018 (GLOBE NEWSWIRE) -- For the first time, a ligand-targeted nanoparticle has been successfully used to deliver CRISPR into the nucleus of a T-cell.
“From the start, CRISPR and other genetic therapies have suffered from the classic ‘last-mile’ problem,” said Andre Watson, founder, chairman and CEO of Ligandal. “For all their power to heal, genetic therapies are ineffective at best if they cannot be delivered to the precise cellular subtypes and locations inside cells.
“Now they can be,” Watson said.
Ligandal’s bioinspired nanomaterials utilize peptide targeting ligands, which bind to precise cellular surface profiles of targeted cell populations, to target CRISPR and other genetic materials into T-cells or other cells. The ligand targeting capacity is an industry first for these gene therapy modalities: Ligandal’s technology can selectively target T-cells with manifold greater efficiency than other blood cell populations.
“In part, the flexibility and ease of this platform is enabled by state-of-the-art peptide synthesis and fluid handling robotics, driven by a back-end machine-learning approach to rapid ligand and nanomaterials discovery,” Watson said. “Of course, none of these robotics or machine learning approaches would be meaningful without our breakthroughs in bioinspired nanomaterials design and synthesis.”
In addition to Ligandal’s breakthroughs in high-throughout biological nanomaterials screening, the company has demonstrated that its first-in-class ligand-targeted particles are efficiently performing gene edits.
In a representative image, Ligandal documented the delivery of CRISPR to a T-cell nucleus via 3D super-resolution microscopy. Such an image has never been shown for a nanoparticle delivering a gene editing tool, and Ligandal is the first to demonstrate ligand-targeting of CRISPR via a non-viral construct. Furthermore, these particles are serum stable, forming the basis of a breakthrough approach to in vivo gene therapy.
“This is just the start,” Watson said. “Our novel imaging pipeline coupled to our leading genomics, peptide synthesis, gene editing and biological nanomaterials expertise will ramp up production by giving us real-time quantification of nanoparticle uptake and gene-editing efficiency for thousands of nanoparticle variants per week. Most importantly, this infrastructure is forming the basis of the first diagnostically-responsive nanomaterials lab the world has ever seen. We are pleased to have brought on leading scientists in the field to drive us forward on our mission of creating curative, scalable, cost-effective genetic therapies for humanity at large.”
Ligandal is now working to program T-cells to target markers of viruses and cancer cells, as well as developing a novel set of nano-immunotherapies with no industry precedent. Current data shows that Ligandal’s approach can differentially target human primary T-cells within mixed blood cell populations, delivering CRISPR and other genetic tools with high efficiency and performing gene edits. The company has also generated compelling data in bone marrow and additional blood models.
Watson gave thanks to Shuailiang Lin, PhD, for his novel genome engineering work; Ryan Spencer, PhD, for his peptide engineering breakthroughs; Matthew Dobbin, PhD’s for his novel computational imaging pipeline; and Kola Awe for his computational robotics and nanomaterials design pipeline. The company also thanks additional team members involved in the project.
Overall, Ligandal is developing a complete platform to unlock the human genome to tackle multiple complex diseases to free humanity from the pain and suffering caused by each disease.
Contact: Vinh Vuong
February 13, 2018 (Investorideas.com Newswire) Medical malpractice affects thousands of individuals around the world. If you think you have been injured, or wrongfully treated by a medical professional, you may be entitled to compensation.