Cell and Gene Therapy: Taking a New Facility from Construction to...
This webinar will focus on the common pitfalls encountered during the startup phase of cell and gene therapy facilities, with special emphasis on lessons learned in the CMO space.(PRWeb February 21, 2020)Read the full story at https://www.prweb.com/releases/cell_and_gene_therapy_taking_a_new_facility_from_construction_to_production_upcoming_webinar_hosted_by_xtalks/prweb16926002.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - February 21, 2020 Category: Pharmaceuticals Source Type: news

BIA Separations and University of Zagreb Sign License Agreement to Commercialize Novel Elution Method for Virus and Viral Vector purification
Agreement gives BIA Separations access to proprietary technology to better preserve integrity, infectivity and potency of immunoaffinity-purified viral vectors for gene therapy (Source: The Scientist)
Source: The Scientist - February 20, 2020 Category: Science Tags: The Scientist The Marketplace Source Type: news

Plant-based relatives of cholesterol could give boost to gene therapy
(Oregon State University) Gene-infused nanoparticles used for combating disease work better when they include plant-based relatives of cholesterol because their shape and structure help the genes get where they need to be inside cells. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 20, 2020 Category: International Medicine & Public Health Source Type: news

Why this company picked NC for a $110 million investment
Gene therapy company Audentes Therapeutics has tapped Sanford as the next location for its second large-scale manufacturing operation. Here's why. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - February 19, 2020 Category: Biotechnology Authors: Seth Thomas Gulledge Source Type: news

Gene therapy company picks North Carolina for 200-job manufacturing facility
Gene therapy company Audentes Therapeutics, which last month completed a $3 billion buyout by Astellas Pharma, picked a North Carolina site over competing locations in California, Massachusetts and Colorado for a 209-job manufacturing facility, the company said Tuesday. The expected $109.4 million investment at the site in Sanford, North Carolina, comes in exchange for a "job development investment grant" worth up to $3.7 million that was approved by North Carolina state officials. Additionally,… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - February 18, 2020 Category: Biotechnology Authors: Lauren Ohnesorge Source Type: news

Gene therapy company picks North Carolina for 200-job manufacturing facility
Gene therapy company Audentes Therapeutics, which last month completed a $3 billion buyout by Astellas Pharma, picked a North Carolina site over competing locations in California, Massachusetts and Colorado for a 209-job manufacturing facility, the company said Tuesday. The expected $109.4 million investment at the site in Sanford, North Carolina, comes in exchange for a "job development investment grant" worth up to $3.7 million that was approved by North Carolina state officials. Additionally,… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - February 18, 2020 Category: Pharmaceuticals Authors: Lauren Ohnesorge Source Type: news

Gene therapy treatment for blindness underway
First patients begin promising gene therapy Related items fromOnMedica Gene therapy hopes for AMD Baby born with three-person DNA Myopia could be linked to longer periods spent in education Mexican fish offer clues on heart repair Online resource aims to help GPs spot early signs of degenerative muscle disease (Source: OnMedica Latest News)
Source: OnMedica Latest News - February 18, 2020 Category: UK Health Source Type: news

First patients begin gene therapy treatment for blindness
The first patients have received a revolutionary new gene therapy that can restore eyesight. (Source: NHS Networks)
Source: NHS Networks - February 18, 2020 Category: UK Health Source Type: news

Gene Therapy for Mesothelioma Has ‘Real Potential’
Gene therapy is moving closer to becoming part of standard-of-care treatment for pleural mesothelioma, according to the latest multicenter clinical trial. The phase III trial, known as the INFINITE clinical research study, is designed to evaluate the intrapleural delivery of an investigational drug — a type of gene therapy — in combination with celecoxib and gemcitabine, anti-inflammatory and chemotherapy drugs, respectively. Researchers hope to stop, or at least slow, the growth of mesothelioma tumor cells with the combination therapy. “This is a very interesting concept,” oncologist Dr. Bernardo G...
Source: Asbestos and Mesothelioma News - February 17, 2020 Category: Environmental Health Authors: Matt Mauney Source Type: news

Mesothelioma Gene Therapy Trial Shows ‘Real Potential’
Gene therapy is moving closer to becoming part of standard-of-care treatment for pleural mesothelioma, according to the latest multicenter clinical trial. The phase III trial, known as the INFINITE clinical research study, is designed to evaluate the intrapleural delivery of an investigational drug — a type of gene therapy — in combination with celecoxib and gemcitabine, anti-inflammatory and chemotherapy drugs, respectively. Researchers hope to stop, or at least slow, the growth of mesothelioma tumor cells with the combination therapy. “This is a very interesting concept,” oncologist Dr. Bernardo G...
Source: Asbestos and Mesothelioma News - February 17, 2020 Category: Environmental Health Authors: Matt Mauney Source Type: news

Gene therapy that gives cricket-mad Jake, 23, a chance to hit blindness for six
Jake Ternent, 22, from Ludworth, Co Durham had resigned himself to going completely blind due to an inherited degenerative eye condition. However, pioneering gene therapy may reverse the damage. (Source: the Mail online | Health)
Source: the Mail online | Health - February 15, 2020 Category: Consumer Health News Source Type: news

How S.F. plans to go on a $700 million drug-buying spree
The contracts allow for big increases in some drugs and the potential multimillion-dollar cost for each dose of cutting-edge gene therapies. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - February 13, 2020 Category: Biotechnology Authors: Ron Leuty Source Type: news

Gene therapy prevents disorders with alcohol exposure in ALDH2 deficiency
(Mary Ann Liebert, Inc./Genetic Engineering News) A new study has shown that gene therapy to treat one of the most common hereditary disorders, aldehyde dehydrogenase type 2 (ALDH2) deficiency, may prevent increased risk for esophageal cancer and osteoporosis associated with chronic alcohol exposure. (Source: EurekAlert! - Social and Behavioral Science)
Source: EurekAlert! - Social and Behavioral Science - February 12, 2020 Category: International Medicine & Public Health Source Type: news

Catalent Appoints Jim Walter as Vice President of Operations for Oral...
Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today...(PRWeb February 12, 2020)Read the full story at https://www.prweb.com/releases/catalent_appoints_jim_walter_as_vice_president_of_operations_for_oral_and_specialty_delivery/prweb16906402.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - February 12, 2020 Category: Pharmaceuticals Source Type: news

Encoded Therapeutics Expands Gene Therapy Leadership with Key Appointment and Promotion
Salvador Rico, M.D., Ph.D., named Chief Medical Officer Martin Moorhead, Ph.D., promoted to Chief Technology Officer SOUTH SAN FRANCISCO, Calif., Feb. 11, 2020 -- (Healthcare Sales & Marketing Network) -- Encoded Therapeutics, Inc. (Encoded), a prec... Biopharmaceuticals, Personnel Encoded Therapeutics, gene therapy, Dravet Syndrome (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - February 11, 2020 Category: Pharmaceuticals Source Type: news

Catalent and Zumutor Biologics Collaborate to Manufacture...
Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, today...(PRWeb February 11, 2020)Read the full story at https://www.prweb.com/releases/catalent_and_zumutor_biologics_collaborate_to_manufacture_first_in_class_mab_to_treat_solid_tumors/prweb16902380.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - February 11, 2020 Category: Pharmaceuticals Source Type: news

Novel techniques for mining patented gene therapies offer promising treatment options
(Purdue University) A team of scientists from Purdue University and other research institutions around the world have come together to better understand the growing number of worldwide patented innovations available for gene therapy treatment. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - February 7, 2020 Category: Cancer & Oncology Source Type: news

St. Jude's scientist receives ACGT grant for sarcoma gene therapy
(Alliance for Cancer Gene Therapy) Stephen Gottschalk, MD, of St. Jude Children's Research Hospital receives $500k grant to use genetically engineered immune cells to attack pediatric sarcoma. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - February 7, 2020 Category: Cancer & Oncology Source Type: news

Rejuvenate Bio launches to help dogs live longer, healthier lives
(Wyss Institute for Biologically Inspired Engineering at Harvard) Gene therapy isn't just for humans anymore: Rejuvenate Bio has secured a license to commercialize a Wyss-developed technology that can treat multiple age-related conditions in dogs, helping them live longer, healthier lives. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - February 6, 2020 Category: Biology Source Type: news

Philadelphia gene therapy startup plans $125M IPO
The company's founders include Penn Medicine gene therapy pioneer Dr. James Wilson. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - February 3, 2020 Category: Biotechnology Authors: John George Source Type: news

Philadelphia gene therapy startup plans $125M IPO
The company's founders include Penn Medicine gene therapy pioneer Dr. James Wilson. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - February 3, 2020 Category: Pharmaceuticals Authors: John George Source Type: news

Here's how many commercialization deals Nationwide Children's made in 2019, its busiest year yet
Gene therapy does the "heavy lifting" in income to Nationwide Children's Hospital from licensing its inventions, but record activity is building a diverse portfolio of products. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 31, 2020 Category: Pharmaceuticals Authors: Carrie Ghose Source Type: news

'I trust you': How a Lancaster family helped lay the building blocks for gene-therapy breakthroughs at Nationwide Children ’s
Andrew is the “And” in Andelyn Biosciences. He was 3 in 2001 when he was diagnosed with Duchenne muscular dystrophy, the most common and among the most aggressive forms of the inherited condition. Patients usually don’t reach 20. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 30, 2020 Category: Biotechnology Authors: Carrie Ghose Source Type: news

UPenn scientists receive ACGT grant to accelerate CAR T-Cell clinical trial
(Alliance for Cancer Gene Therapy) Alliance for Cancer Gene Therapy awards grant to University of Pennsylvania scientists to use CAR T-cell gene therapy to treat advanced metastatic prostate cancer. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 30, 2020 Category: International Medicine & Public Health Source Type: news

FDA continues strong support of innovation in development of gene therapy products
This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient's cells. (Source: World Pharma News)
Source: World Pharma News - January 29, 2020 Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news

Novartis sales rise 9% as new medicines provide a boost
Novel gene therapy a boon to Swiss drugmaker (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - January 29, 2020 Category: Pharmaceuticals Source Type: news

Catalent Appoints Ricci Whitlow to Lead Clinical Supply Services...
Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, gene therapies, and consumer health products, today announced...(PRWeb January 29, 2020)Read the full story at https://www.prweb.com/releases/catalent_appoints_ricci_whitlow_to_lead_clinical_supply_services_business/prweb16870617.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - January 29, 2020 Category: Pharmaceuticals Source Type: news

Six patients with rare blood disease are doing well after gene therapy clinical trial
(University of California - Los Angeles Health Sciences) UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD - which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan -- had to rely on bone marrow donations for a chance at remission. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 28, 2020 Category: International Medicine & Public Health Source Type: news

Gene therapy success in chronic septic granulomatosis
(AFM-T é l é thon) American and British teams published yesterday in Nature Medicine the conclusive results of a gene therapy trial conducted in the United States and Great Britain in 9 patients with X-linked Chronic Septic Granulomatosis (X-CGD), a rare and severe immune dysfunction. Genethon, which contributed to the research that led to these trials and sponsored initial clinical studies, is pleased with these results. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 28, 2020 Category: International Medicine & Public Health Source Type: news

Six patients with rare blood disease are doing well after gene therapy clinical trial
UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD – which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan – had to rely on bone marrow donations for a chance at remission.“With this gene therapy, you can use a patient’s own stem cells instead of donor cells for a transplant,&rdqu...
Source: UCLA Newsroom: Health Sciences - January 28, 2020 Category: Universities & Medical Training Source Type: news

Gene scissors against incurable muscular disease
(Technical University of Munich (TUM)) Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy. Researchers at Technical University of Munich (TUM), Ludwig Maximilian University of Munich (LMU) and the German Research Center for Environmental Health (Helmholtz Zentrum M ü nchen) have developed a gene therapy that may provide permanent relief for those suffering from DMD. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 27, 2020 Category: International Medicine & Public Health Source Type: news

Researchers uncover mechanism for how common gene therapy vectors enter cells
(Massachusetts Eye and Ear Infirmary) Researchers have identified a novel cellular entry factor for adeno-associated virus vector (AAV) types -- the most commonly used viral vectors for in vivo gene therapy. The researchers identified that GPR108, a G protein-coupled receptor, served as a molecular 'lock' to the cell. The discovery could one day enable scientists to better direct AAV gene transfers to specific tissues. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - January 23, 2020 Category: Biology Source Type: news

Catalent Biologics Supports DiaMedica ’s New Treatment for Chronic...
Catalent, the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, gene therapies, and consumer health products, today welcomed the...(PRWeb January 23, 2020)Read the full story at https://www.prweb.com/releases/catalent_biologics_supports_diamedicas_new_treatment_for_chronic_kidney_disease/prweb16857264.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - January 23, 2020 Category: Pharmaceuticals Source Type: news

Krystal Biotech appoints new chief commercial officer
Krystal Biotech announced Tuesday that it had named Jennifer Chien to the newly created position of chief commercial officer, effective Monday. Chien joins the Pittsburgh-based gene therapy company after serving as vice president, head of genetic diseases at Sanofi Genzyme and has more than 20 years of experience in the biopharmaceutical industry. “Jennifer’s leadership and broad experience launching new medicines will be critical as we continue to build the commercial capabilities needed… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 21, 2020 Category: Pharmaceuticals Authors: Luke Torrance Source Type: news

Gene therapies test Europe’s willingness to pay
Two $2m treatments launch this year but a reluctance to fork out could dim investor enthusiasm (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - January 16, 2020 Category: Pharmaceuticals Source Type: news

Gene therapy company eyes adding 100 jobs at new East Bay plant
The company already has 80-90 employees in Menlo Park who will move to the new facility. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 15, 2020 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

Into pharma's roaring twenties
“I drained the last of my cocktail, gazing up at the ceiling. It was one of those moments that curls the hairs on your neck. At once, the grand scale of this labyrinth of cathedrals became clear, the desert wind blowing through the clever hieroglyphics carved into every available surface. I turned my head back down to ground level just in time to see the man draw back his fist in anger, and then.And then.With a piercing shriek, he lunged straight at my jaw –”Do you ever wake from a dream, marvelling at the inventiveness and detail of your subconscious mind? That disorienting moment where you lie blinking ...
Source: EyeForPharma - January 14, 2020 Category: Pharmaceuticals Authors: Paul Simms Source Type: news

Roche aims to 'underwhelm' on SMA drug price to challenge rivals
Swiss drugmaker Roche plans to price its oral spinal muscular atrophy drug (SMA) risdiplam aggressively to challenge two of the world's most expensive medicines, Biogen's Spinraza and Novartis's gene therapy Zolgensma. (Source: Reuters: Health)
Source: Reuters: Health - January 14, 2020 Category: Consumer Health News Tags: healthNews Source Type: news

Gene Therapy Finds a Fertile Home in Ohio
The midwestern state has quietly laid the groundwork for a biotech hub. (Source: The Scientist)
Source: The Scientist - January 13, 2020 Category: Science Tags: Bio Business Magazine Issue Source Type: news

At 16, She ’s a Pioneer in the Fight to Cure Sickle Cell Disease
Helen Obando is the youngest person ever to get a gene therapy that scientists hope will cure the disease, which afflicts 100,000 Americans. (Source: NYT Health)
Source: NYT Health - January 12, 2020 Category: Consumer Health News Authors: Gina Kolata Tags: Sickle Cell Anemia Genetics and Heredity Genetic Engineering Stem Cells Clinical Trials Boston Children ' s Hospital Bluebird Bio Source Type: news

Cambridge startup raises $110M for re-doseable gene therapies
One of Cambridge ’s emerging gene therapy startups has raised $110 million to support the company as it heads toward in-human trials.  Generation Bio closed the Series C round this week, bringing in new investors T. Rowe Price, Farallon and Wellington Management, as well as previous backers Atlas Venture, Fideli ty and others. The Series C round brings the total money raised by Generation Bio to more than $235 million. It’s also the first financing round led by former Vertex Pharmaceuticals… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 10, 2020 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

A Teenager ’s Breakthrough Gene Therapy for Sickle Cell Disease
Doctors reset Helen Obando ’s DNA in an effort to cure her of a painful genetic blood disorder. She’s the youngest person to receive the treatment. (Source: NYT Health)
Source: NYT Health - January 10, 2020 Category: Consumer Health News Tags: Sickle Cell Anemia DNA (Deoxyribonucleic Acid) Genetics and Heredity Boston Children ' s Hospital Clinical Trials FX (TV Network) Hulu.com The Weekly (TV Program) Source Type: news

enGene Announces Additions to Senior Management Team
BOSTON and MONTRÉAL, Jan. 9, 2020 -- (Healthcare Sales & Marketing Network) - enGene Inc., the biotechnology company developing rationally designed gene therapies targeted to mucosal tissues through its proprietary non-viral vector DDX® platfor... Biopharmaceuticals, Drug Delivery, Personnel enGene, DDX, Gene Pill (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - January 9, 2020 Category: Pharmaceuticals Source Type: news

How safe is a popular gene therapy vector?
(Source: ScienceNOW)
Source: ScienceNOW - January 9, 2020 Category: Science Authors: Kaiser, J. Tags: Cell Biology, Medicine, Diseases In Depth Source Type: news

Nanoparticles deliver 'suicide gene' therapy to pediatric brain tumors growing in mice
(Johns Hopkins Medicine) Johns Hopkins researchers report that a type of biodegradable, lab-engineered nanoparticle they fashioned can successfully deliver a " suicide gene " to pediatric brain tumor cells implanted in the brains of mice. The poly(beta-amino ester) nanoparticles, known as PBAEs, were part of a treatment that also used a drug to kill the cells and prolong the test animals' survival. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 8, 2020 Category: International Medicine & Public Health Source Type: news

Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community. (Source: The Scientist)
Source: The Scientist - January 7, 2020 Category: Science Tags: News & Opinion Source Type: news

NC university is leading the country in gene editing research
As the Triangle's gene therapy industry continues to represent the nation's leading edge, Duke University is now tied for the most federal grants supporting the research behind it. In 2018, the National Institutes of Health (NIH) launched the Somatic Cell Genome Editing program (SCGE) to fund research behind genome editing techniques for use in the cells and tissues of the body. Those regions are directly responsible for thousands of disorders caused by DNA mutations inc luding Duchenne muscular… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 7, 2020 Category: Biotechnology Authors: Seth Thomas Gulledge Source Type: news

Mayo Clinic researchers pursue single-dose gene therapy to treat cocaine addiction
(Mary Ann Liebert, Inc./Genetic Engineering News) In a radical new approach to treat cocaine addition, researchers at the Mayo Clinic are seeking approval for first-in-human studies of a single-dose gene therapy. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 7, 2020 Category: International Medicine & Public Health Source Type: news

Sight loss research needs urgent investment | Letter
Leading ophthalmologists call on the new government to develop a national plan to fight blindness and address the critical lack of fundingAs leading ophthalmologists and researchers we are joining the eye research charity Fight for Sight to call for urgent action on blindness in 2020 to address the research funding gap.We know that serious sight loss doesn ’t discriminate – it can affect anyone at any time and it is on the increase. Science can already do so much and with the advent of new gene therapies and stem cell treatments we are so close to outcomes that were not possible a decade ago. Yet so much more n...
Source: Guardian Unlimited Science - January 6, 2020 Category: Science Authors: Letters Tags: Blindness and visual impairment Medical research Disability Health Society Science Conservatives Source Type: news

Dog Study Revives Concerns About Virus Used for Gene Therapy
Canines treated with an adeno-associated viral (AAV) vector showed evidence that the therapeutic DNA held within the virus can integrate into the host genome, risking the activation of oncogenes. (Source: The Scientist)
Source: The Scientist - January 6, 2020 Category: Science Tags: News & Opinion Source Type: news