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Opioids are very effective analgesics, but they are also highly addictive. Methadone is used to treat opioid dependence (OD), acting as a selective agonist at the μ-opioid receptor encoded by the gene OPRM1. Determining the optimal methadone maintenance dose is time consuming; currently, no biomarkers are available to guide treatment. In methadone-treated OD subjects drawn from a case and control sample, we conducted a genome-wide association study of usual daily methadone dose. In African-American (AA) OD subjects (n=383), we identified a genome-wide significant association between therapeutic methadone dose (mean=68.0 mg, s.d.=30.1 mg) and rs73568641 (P=2.8 × 10-8), the nearest gene (306 kilobases) being OPRM1. Each minor (C) allele corresponded to an additional ~20 mg day-1 of oral methadone, an effect specific to AAs. In European-Americans (EAs) (n=1027), no genome-wide significant associations with methadone dose (mean=77.8 mg, s.d.=33.9 mg) were observed. In an independent set of opioid-naive AA children being treated for surgical pain, rs73568641-C was associated with a higher required dose of morphine (n=241, P=3.9 × 10-2). Similarly, independent genomic loci previously shown to associate with higher opioid analgesic dose were associated with higher methadone dose in the OD sample (AA and EA: n=1410, genetic score P=1.3 × 10-3). The present results in AAs indicate that genetic variants influencing opioid sensitivity across different clinical settings could contribute to precision pharmacotherapy for pain and addiction.Molecular Psychiatry advance online publication, 24 January 2017; doi:10.1038/mp.2016.257. © 2017 Macmillan Publishers Limited, part of Springer Nature.

Aevi Genomic Medicine, Inc. ( : GNMX) (the Company) announced today recent highlights and reported financial results for the quarter ended March 31, 2017. "Following additional analysis of the SAGA trial, we remain positive about the genetic subset of patients identified that demonstrate a clear and statistically significant response to AEVI-001," said Mike Cola, CEO of Aevi Genomic Medicine. "This discovery will allow us to move forward with the development of AEVI-001 in ADHD, and potentially other neurodevelopmental disorders in the future, including Autism Spectrum Disorder. We look forward to initiating the Phase 2 trial of AEVI-001 in patients with the identified genetic subset during the second half of 2017 and anticipate announcing top-line data in mid-2018." Cash and cash equivalents as of March 31, 2017 were $29.20 million, compared to $39.84 million as of December 31, 2016. The decrease in cash was primarily related to the advancement of our AEVI-001 program. The Company expects its reported cash balance to fund operations through the end of the second quarter 2018. Research and development expenses for the three months ended March 31, 2017 were $7.95 million, increasing from $6.95 million for the same period in 2016 mainly due to increased cost associated with the clinical advancement of AEVI-001 and AEVI-002. General and administrative expenses for the three months ended March 31, 2017 were $2.99 million, decreasing from $4.19 million for the same period in 2016 primarily due to severance benefits recorded in 2016 related to the termination of an officer of the Company. For the quarter ended March 31, 2017 the Company reported a net loss of $10.92 million or $0.29 per share, compared with a net loss of $11.14 million or $0.34 per share for the comparative quarter in 2016. Conference Call and Webcast Information Aevi Genomic Medicine will hold a conference call today at 8:30 am ET to discuss these results. To access the conference call by phone, please dial (888) 481-2845 (domestic) or (719) 325-2327 (international). The conference passcode is 9118660. The live webcast can be accessed under "Events" in the Investors section of the Company's website at or via the following link. Following the live webcast a replay of the call will be available May 10, 2017 through June 15, 2017. To access the replay, please dial (888) 203-1112 (domestic) or (719) 457-0820 (international) and reference the reply passcode 9118660. The archived webcast will be available for 30 days in the Investor section of Aevi Genomic Medicine website at Aevi Genomic Medicine, Inc. is dedicated to unlocking the potential of genomic medicine to translate genetic discoveries into novel therapies. Driven by a commitment to patients with pediatric onset life-altering diseases, the company's research and development efforts leverages an internal genomics platform and an ongoing collaboration with the Center for Applied Genomics (CAG) at The Children's Hospital of Philadelphia (CHOP). This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and as that term is defined in the Private Securities Litigation Reform Act of 1995, which include all statements other than statements of historical fact, including (without limitation) those regarding the Company's financial position, its development and business strategy, its product candidates and the plans and objectives of management for future operations. The Company intends that such forward-looking statements be subject to the safe harbors created by such laws. Forward-looking statements are sometimes identified by their use of the terms and phrases such as "estimate," "project," "intend," "forecast," "anticipate," "plan," "planning, "expect," "believe," "will," "will likely," "should," "could," "would," "may" or the negative of such terms and other comparable terminology. All such forward-looking statements are based on current expectations and are subject to risks and uncertainties. Should any of these risks or uncertainties materialize, or should any of the Company's assumptions prove incorrect, actual results may differ materially from those included within these forward-looking statements. Accordingly, no undue reliance should be placed on these forward-looking statements, which speak only as of the date made. The Company expressly disclaims any obligation or undertaking to disseminate any updates or revisions to any forward-looking statements contained herein to reflect any change in the Company's expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based. As a result of these factors, the events described in the forward-looking statements contained in this release may not occur.

LOS ALTOS, Calif. & DURHAM, N.C.--(BUSINESS WIRE)--PMWC International, the preeminent precision medicine conference leader that since 2009 has attracted recognized leaders, top global researchers and medical professionals, and innovators across healthcare and biotechnology sectors, today announced that Duke Health and Duke University will co-host the first-ever East Coast Precision Medicine World Conference in North Carolina on May 24-25, 2017. Hundreds of healthcare leaders, representing a variety of companies, technologies, researchers and medical centers with leadership roles in precision medicine are expected to attend. A total of 100 speakers (including N.C. Governor Roy Cooper) from healthcare and biotechnology sectors will be featured in more than 35 sessions; topics include: The conference will recognize five individuals who have played and continue to play a significant role in transforming today’s healthcare by advancing precision medicine into the clinic. This includes Dr. Francis S. Collins, director of the National Institutes of Health, who played a vital role in leading the human genome sequencing project as well as major government initiatives such as the 1-million-person All of Us cohort program. Additional honorees include Dr. Elaine Mardis, Nationwide Children’s Hospital; Kathy Giusti, Multiple Myeloma Research Foundation; Dr. Keith Yamamoto, UCSF; and Mark Levin, Third Rock Ventures. “Duke established itself as a founder and recognized leader in precision medicine, embracing it both scientifically and clinically. For us, it is a logical choice to bring the PMWC to Duke and to allow us to highlight what is happening on our campus, in our region and on the East Coast,” said Dr. Ralph Snyderman, chancellor emeritus of Duke University, who is chairing the program together with Dr. Geoff Ginsburg, the director of the Duke Center for Applied Genomics & Precision Medicine. “PMWC’s expertise in showcasing practical content and bringing together recognized and established leaders across the field helps close the knowledge gap between different sectors and catalyzes cross-functional dialog and collaboration, which can benefit all conference attendees,” said Tal Behar, co-founder and president of PMWC International. Since 2009, recognized as a vital cornerstone for all constituents of the healthcare and biotechnology community, PMWC ( provides an exceptional forum for the exchange of information about the latest advances in technology (e.g., DNA sequencing technology), in clinical implementation (e.g., cancer and beyond), research, and in all aspects related to the regulatory and reimbursement sectors.

News Article | May 24, 2017

DURHAM, N.C.--(BUSINESS WIRE)--Panaceutics Inc., a personalized medicine and clinical nutrition company, today announced that Chief Executive Officer Edison Hudson will present at the Precision World Medicine Conference, PMWC-2017, on Thursday, May 25th at 2:15 pm at Duke University’s Fuqua School of Business. Recognized as the original and leading annual forum on precision medicine, this event attracts over 11,000 attendees to listen to presentations and panels made by the world’s leading authorities and experts across the health care and bioinformatics sectors. These experts are delivering the most current, up-to-date content across all facets of personalized and precision medicine. “As a team, we are bringing cross-functional stakeholders of this exciting field together to exchange ideas and the learnings gathered so far that can be translated into new winning strategies for the future of precision medicine as cutting edge development in healthcare,” said PMWC-2017 program co-chair Geoff Ginsberg, M.D., Ph.D., Director of the Duke Center for Applied Genomics and Precision Medicine. The event will be launched on Wednesday, May 24th by North Carolina Governor Roy Cooper and Mary E. Klotman, Chair of the Duke University School of Medicine. “I am honored to be part of this esteemed conference and among the professionals who are blazing the path toward commercializing precision medicine. My talk will focus on how personalized medicine could be a game changer for patient adherence, actionable while reducing prescription costs,” said Hudson. Hudson is no stranger to inventing new technologies that automate complex tasks. Before co-founding Panaceutics to lead the team to design a novel automated personalized medicine fulfillment system, he was a leader in several technology and robotics companies, including iRobot, Adept Technology, and Redzone Robotics, spending over a decade in the semiconductor industry in Silicon Valley. Founder of four companies, he has more than 17 patents in machine design, control systems, machine vision, algorithms, and semiconductor processes. A Morehead Scholar at the University of North Carolina, he also studied artificial intelligence at Oxford University and received an MBA from Duke University. Panaceutics delivers personalized monthly subscriptions of “pill free” suspensions combining up to 20 active macro and micro nutrient ingredients in tasty puree filled pouches that are a convenient way to take individualized formulas of clinical nutrition and pharmacy grade dietary supplements. In several market trials, this delivery form has been found to be favored by a large majority over taking multiple pills. Cloud-based software is used to choose the right ingredients and dosage for an individual, and the formula is then sent to Panaceutics’ proprietary robotic system that builds a 30-day supply in a few minutes under cGMP quality rules. Panaceutics, with the addition of recently acquired Triangle Compounding Pharmacy, gains expertise in pharmaceutical delivery and a 503B outsourcing pharmacy facility. Dr. Michael Jay, Chairman of the Division of Molecular Pharmaceutics at the UNC Eshelman School of Pharmacy heads the Company’s Advisory Board and along with several other distinguished medical professionals are advising co-founder Lloyd Staton Noel, III, a 20 year veteran of drug discovery at GSK, in the development and testing of the company’s patented and proprietary technologies applied to precision combination pharmaceutical therapies. In a joint development signed with a large hospital system’s cardiology team, the company will develop cardiovascular “poly therapies” including approved generic pharmaceutical ingredients individually formulated and combined in a form designed to dramatically improve adherence. Panaceutics’ formulation software and robotic small batch manufacturing technologies will enable the principles of precision medicine and health to be economically applied to large markets.

Diskin S.J.,Applied Genomics
Journal of the National Cancer Institute | Year: 2014

TP53 is the most frequently mutated gene in human malignancies; however, de novo somatic mutations in childhood embryonal cancers such as neuroblastoma are rare. We report on the analysis of three independent case-control cohorts comprising 10290 individuals and demonstrate that rs78378222 and rs35850753, rare germline variants in linkage disequilibrium that map to the 3' untranslated region (UTR) of TP53 and 5' UTR of the Δ133 isoform of TP53, respectively, are robustly associated with neuroblastoma (rs35850753: odds ratio [OR] = 2.7, 95% confidence interval [CI] = 2.0 to 3.6, P combined = 3.43×10(-12); rs78378222: OR = 2.3, 95% CI = 1.8 to 2.9, P combined = 2.03×10(-11)). All statistical tests were two-sided. These findings add neuroblastoma to the complex repertoire of human cancers influenced by the rs78378222 hypomorphic allele, which impairs proper termination and polyadenylation of TP53 transcripts. Future studies using whole-genome sequencing data are likely to reveal additional rare variants with large effect sizes contributing to neuroblastoma tumorigenesis.

Wong J.T.-F.,Applied Genomics
Frontiers in Bioscience - Landmark | Year: 2014

This study tracks the rise, evolution and postevolution of the genetic information system through emergence of life. The major stages traversed include prebiotic synthesis, functional RNA selection by metabolite, RNA World, peptidated RNA world, co-evolution of genetic code and amino acid biosynthesis, last universal common ancestor, Darwinian evolution and synthetic life.

A composition and method for generating reagents and the composition of these reagents for the stabilization and preservation of viability of cancer tissue which has been surgically excised and the suspension and/or termination of apoptosis (cell death) by significant modulation of cell metabolism by low molar concentrations of synergistic chemistries and hormonal growth enhancers while maintaining normal gene expression patterns of the surgically excised tissue.

A composition and method for generating reagents and the composition of these reagents for the stabilization and preservation of viability of cancer tissue which has been surgically excised and the suspension and/or termination of apoptosis (cell death) by significant modulation of cell metabolism by low molar concentrations of synergistic chemistries and hormonal growth enhancers while maintaining normal gene expression patterns of the surgically excised tissue.

Applied Genomics | Date: 2014-01-08

The present invention relates to the regulation of the natural defense system of plants through the introduction of foreign/native genes into plant cells, preferably into their genomes. More specifically, the methods relate to increasing citrus plant disease resistance by over-expressing genes involved in the innate plant defense system.

A composition and method for generating reagents and the composition of these reagents for the stabilization and preservation of viability of cancer tissue which has been surgically excised and the suspension and/or termination of apoptosis (cell death) by significant modulation of cell metabolism by low molar concentrations of synergistic chemistries and hormonal growth enhancers while maintaining normal gene expression patterns of the surgically excised tissue.

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