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First U.S. patient treated with innovative gene therapy at Bascom Palmer Eye Institute

A Puerto Rican patient with X-linked retinitis pigmentosa (XLRP) is hoping to save his vision after an innovative gene therapy procedure at Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine.

19 Sep 2018

Study reveals new therapeutic target for pediatric tumor-associated intractable epilepsy

Pediatric brain tumors are characterized by frequent complications due to intractable epilepsy compared to adult brain tumors. However, the genetic cause of refractory epilepsy in pediatric brain tumors has not been elucidated yet, and it is difficult to treat patients because the tumors do not respond to existing antiepileptic drugs and debilitate children's development.

19 Sep 2018

DermTech completes enrollment in clinical study to assess DNA damage and reversal

DermTech, Inc., the global leader in non-invasive molecular dermatology, announced today it has completed enrollment in a clinical study to assess DNA damage induced by an excimer laser and evaluate the potential of topical agents T4 endonuclease and photolyase to reduce DNA damage induced as measured through gene expression analysis.

19 Sep 2018

AmpliPhi receives positive feedback from FDA for its AB-PA01 product targeting P.aeruginosa infections

AmpliPhi Biosciences Corporation, a clinical-stage biotechnology company focused on precisely targeted bacteriophage therapeutics for antibiotic-resistant infections, today announced that the company has received positive feedback, via written response, from the U.S. Food and Drug Administration regarding its development plans for AB-PA01, without the need for a Type B Pre-IND meeting.

19 Sep 2018

UTILITY receives FDA approval for Investigational New Drug applications

UTILITY therapeutics Ltd, a pharmaceutical company focused on developing and commercializing antibiotics in the US, today announces that the US Food and Drug Administration has approved the Investigational New Drug applications for mecillinam and pivmecillinam.

19 Sep 2018

Novel clinical trial to examine cannabis as potential treatment for essential tremor

Researchers at University of California School of Medicine are preparing to launch a novel clinical trial to examine the safety, efficacy and pharmacological properties of cannabis as a potential treatment for adults with essential tremor.

18 Sep 2018

Researchers use CRISPR screen to reveal new targets in squamous cell carcinomas

A little p63 goes a long way in embryonic development - and flaws in p63 can result in birth defects like cleft palette, fused fingers or even missing limbs.

18 Sep 2018

Research finds physical connection between the brain's fluid reservoirs and meningeal lymphatics

PureTech Health plc, an advanced biopharmaceutical company developing novel medicines for dysfunctions of the Brain-Immune-Gut Axis, today notes the publication of research in Nature Neuroscience that identifies the physical connection between the brain's fluid reservoirs and the meningeal lymphatics, through which immune cells traffic out of the central nervous system.

18 Sep 2018

LEO Pharma and MorphoSys announce expansion of strategic alliance to develop peptide-derived drugs

LEO Pharma A/S, a global leader in medical dermatology, and MorphoSys AG today announced an expansion of their existing strategic alliance to include peptide-derived therapeutics.

18 Sep 2018

KAIST researchers develop heart-targeting drug delivery technology using tannin acid

Typical methods of drug delivery to the heart require surgical procedures involving incisions in the chest wall and bones.

18 Sep 2018

Brigham Genomic Medicine program unravels 30 medical mysteries

A table in a recently published paper tells the story of 30 families who have, sometimes after years of searching, finally received an answer about the condition that has plagued one or more family members.

18 Sep 2018

Children's National and NIAID launch pediatric clinical research partnership

The National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, and Children's National Health System, a pediatric academic medical center in Washington, D.C., have launched a clinical research partnership devoted to treating and preventing allergic, immunologic and infectious diseases in children.

17 Sep 2018

Rare antibodies could be key ingredients in therapeutics capable of neutralizing many Ebola types

Two new studies by scientists at Scripps Research are bringing Ebola virus's weaknesses into the spotlight, showing for the first time exactly how human and mouse antibodies can bind to the virus and stop infection--not only for Ebola virus, but for other closely related pathogens as well.

13 Sep 2018

Alivio Therapeutics receives DoD grant to advance development of new therapy for IC/BPS

Alivio Therapeutics, an affiliate of PureTech Health plc developing therapies to treat inflammatory disorders through targeted disease immunomodulation, today announced a $3.3 million U.S. Department of Defense Technology/Therapeutic Development Award to advance Alivio's product candidate, ALV-107, for the treatment of interstitial cystitis/bladder pain syndrome with Hunner's lesions.

12 Sep 2018

Penn receives NIH grant to create Center for Sub-Cellular Genomics

The five-year, $10.5 million grant from the NIH is the first CEGS awarded to Penn and funds the creation of the Center for Sub-Cellular Genomics.

12 Sep 2018

Researchers uncover secret of immortality mechanism in aggressive glioblastoma

UC San Francisco researchers have discovered how a mutation in a gene regulator called the TERT promoter -- the third most common mutation among all human cancers and the most common mutation in the deadly brain cancer glioblastoma -- confers "immortality" on tumor cells, enabling the unchecked cell division that powers their aggressive growth.

10 Sep 2018

BioIVT announces research into mechanisms involved in cholestatic drug-induced liver injury

BioIVT, a leading provider of research models and services for drug development, today announced its research into the mechanisms involved in cholestatic drug-induced liver injury has been published in Applied In Vitro Toxicology.

10 Sep 2018

Mimicking Human Brain Development Using Organoids

Dr. Paul Tesar from the Tesar Laboratory at Case Western University, Ohio, discusses the importance of organoids in biological research and the development of organoids which are capable of simulating the early stages of human myelin.

7 Sep 2018

Researchers test experimental cancer vaccine on mice with aggressive melanoma

An experimental cancer vaccine that boosts the immune system's ability to fight cancers could work in tandem with other cancer therapies to fight aggressive tumors, scientists reported recently in the Proceedings of the National Academy of Sciences.

7 Sep 2018

UNC and Cell Microsystems receive NIH Phase I SBIR contract to develop HIV diagnostic assay

The University of North Carolina at Chapel Hill HIV Cure Center and Cell Microsystems, a provider of innovative tools and scalable solutions for the sorting and isolation of single cells, announced they have received an approximately $283,000, 12-month Phase I Small Business Innovation and Research contract from the National Institutes of Health to develop an automated platform to quantify the latent HIV reservoir.

7 Sep 2018

Therapeutics News and Research

First U.S. patient treated with innovative gene therapy at Bascom Palmer Eye Institute

Study reveals new therapeutic target for pediatric tumor-associated intractable epilepsy

DermTech completes enrollment in clinical study to assess DNA damage and reversal

AmpliPhi receives positive feedback from FDA for its AB-PA01 product targeting P.aeruginosa infections

UTILITY receives FDA approval for Investigational New Drug applications

Novel clinical trial to examine cannabis as potential treatment for essential tremor

Researchers use CRISPR screen to reveal new targets in squamous cell carcinomas

Research finds physical connection between the brain's fluid reservoirs and meningeal lymphatics

LEO Pharma and MorphoSys announce expansion of strategic alliance to develop peptide-derived drugs

KAIST researchers develop heart-targeting drug delivery technology using tannin acid

Brigham Genomic Medicine program unravels 30 medical mysteries

Children's National and NIAID launch pediatric clinical research partnership

Rare antibodies could be key ingredients in therapeutics capable of neutralizing many Ebola types

Alivio Therapeutics receives DoD grant to advance development of new therapy for IC/BPS

Penn receives NIH grant to create Center for Sub-Cellular Genomics

Researchers uncover secret of immortality mechanism in aggressive glioblastoma

BioIVT announces research into mechanisms involved in cholestatic drug-induced liver injury

Mimicking Human Brain Development Using Organoids

Researchers test experimental cancer vaccine on mice with aggressive melanoma

UNC and Cell Microsystems receive NIH Phase I SBIR contract to develop HIV diagnostic assay

Understanding the DNA Damage Response in Innate Immunity eBook

Bringing real-time analytics to downstream bioprocess monitoring

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

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