StemCell Therapy

PHILADELPHIA, Oct. 06, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced that management will participate in the virtual 2020 Cell & Gene Meeting on the Mesa, October 12-16, 2020.

Passage Bios Chief Operating Officer, Jill M. Quigley, J.D., will deliver an overview of the company as part of the 2020 company presentations segment of the conference. Passage Bios company presentation will be available to view on-demand throughout the entirety of the conference.

Additionally, Alex Fotopoulos, Chief Technical Officer of Passage Bio, will participate in the panel discussion titled, Accelerating Biotherapeutics Development and Production for Increased Market Impact of Life Saving Therapeutics, moderated by Susan DCosta, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific. The session will cover key topics such as integrated vs. modular manufacturing approaches, ensuring security of supply, proactive approaches to regulatory compliance, and ultimately, the panels vision for the future. The panel session will be available on-demand to attendees as of Monday, October 12, 2020.

Organized by the Alliance for Regenerative Medicine, the Cell & Gene Meeting on the Mesa is a five-day virtual conference featuring more than 120 dedicated company presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering, and broader regenerative medicine technologies. The meeting also includes over 100 panelists and featured speakers taking part in 20 in-depth sessions covering all aspects of cell and gene therapy commercialization.

Please visit http://www.meetingonthemesa.com for full information including registration. Complimentary attendance at this event is available for credentialed investors and members of the media only. Investors should contact Laura Stringham at lstringham@alliancerm.org and interested media should contact Kaitlyn Dupont at kdupont@alliancerm.org.

About Passage BioPassage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP). The GTP conducts discovery and IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy and commercialization activities under the agreement. The company has a development portfolio of six product candidates, with the option to license eleven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia and Krabbe disease.

For further information, please contact:

Investors:Sarah McCabe and Zofia MitaStern Investor Relations, Inc.sarah.mccabe@sternir.com zofia.mita@sternir.com

Media:Gwen FisherPassage Bio215.407.1548gfisher@passagebio.com

View post:
Passage Bio to Participate in 2020 Virtual Cell & Gene Meeting on the Mesa - GlobeNewswire

ALISO VIEJO, Calif., Oct. 1, 2020 /PRNewswire/ --Ambry Genetics(Ambry), a leading clinical genetic testing lab, is launching its CARE for COVID Program with Western Springs School District 101 and The Green Alliance International. The Ambry Genetics Comprehensive, Assessment, Risk, and Education (CARE) for COVID Program is designed to help identify and test individuals in need of coronavirus testing. The program provides Western Springs School District 101 with the system, tools, and support needed to screen and test their faculty and other employees as they return to their offices and classrooms this fall. The Western Springs School District, located in a suburb of Chicago, Illinois, serves students from kindergarten to eighth grade. The Green Alliance International will be using the CARE for COVID Program's screening and exposure questionnaire as part of their Gateway Entry Systems program. The Gateway Entry Systems program provides school systems, sports venues, and businesses across the U.S. with the tools needed to safely reopen, including disinfecting technology, wristband body temperature screening, and the CARE for COVID program's symptom and exposure digital questionnaire for remote monitoring.

The CARE for COVID program includes viral testing by RT-PCR for individuals who are exhibiting symptoms or have known exposure, with results returnedwithin 24-48hours of receipt of the sample. Ambry's RT-PCR test uses saliva collection and creates a simpler and more convenient experience than the nasopharyngeal swabs commonly used by other labs.

The CARE for COVID Program also includes:

The robust, one-stop nature of the CARE for COVID Program ensures that individuals are not missed through multiple engagement points. The CARE for COVID Program incorporates the latest guidance from federal agencies, including the CDC. Ambry worked directly with noted experts in academia, industry, and government to develop the program.

"Over the last six months or so, our team has worked tirelessly to transition our lab and the CARE Program into a comprehensive, end-to-end solution to support workforce and community testing efforts," Ambry Genetics CEO Aaron Elliott said. "We look forward to helping more businesses and schools to safely reopen."

Ambry is expanding the CARE for COVID Program to other school districts and businesses this fall. The Program can be tailored depending on the organization's needs, and employers can choose the frequency for asking individuals to complete the screening questionnaire, whether daily or less often. Ambry is contracted with health plans representing over 90% of insured individuals, making it convenient for the employers to bill insurance for the testing performed.

"With the development of our CARE for COVID program, we learned that organizations want an end-to-end solution," Ambry Genetics Chief Commercial Officer Tom Schoenherr said. "If a critical component is missing in the solution, it will not work. The CARE for COVID program includes education, evaluation, assessment, counseling, testing, post-test counseling, workplace exposure tracing, and reporting."

The Ambry CARE program is a population-health, precision-medicine tool that can address employee health and wellness across a range of medical issues. Already used to help people learn whether they are at risk for hereditary cancers including, breastand colon cancers, the program can also be adapted to help identify and manage diabetes, cardiovascular, and other diseases. To learn more about the CARE program and hereditary cancer risk, please visitambrygen.com/care.

ABOUT AMBRY GENETICS

Ambry Genetics, as part of Konica Minolta Precision Medicine, excels at translating scientific research into clinically actionable test results based upon a deep understanding of the human genome and the biology behind genetic disease. Our unparalleled track record of discoveries over 20 years, and growing database that continues to expand in collaboration with academic, corporate and pharmaceutical partners, means we are first to market with innovative products and comprehensive analysis that enable clinicians to confidently inform patient health decisions. We care about what happens to real people, their families, and the people they love, and remain dedicated to providing them and their clinicians with deeper knowledge and fresh insights, so together they can make informed, potentially life-altering healthcare decisions. For more information, please visitambrygen.com.

For more information on risk factors for hereditary cancer, please visit cancer.gov's fact sheet on hereditary cancer and genetic testing.

Press Contact:Liz Squirepress@ambrygen.com(202) 617-4662

View original content to download multimedia:http://www.prnewswire.com/news-releases/konica-minolta-precision-medicine-kmpm-company-ambry-genetics-announces-comprehensive-covid-19-screening-management-and-testing-program-to-help-schools-and-businesses-to-safely-reopen-301143750.html

SOURCE Ambry Genetics

Read the original here:
Konica Minolta Precision Medicine (KMPM) Company Ambry Genetics Announces Comprehensive COVID-19 Screening, Management, and Testing Program To Help...

Philadelphia, October 5, 2020Through genetic sequencing and targeted treatment, researchers from Childrens Hospital of Philadelphia (CHOP) have resolved a severe lymphatic disorder in a young woman with kaposiform lymphangiomatosis (KLA), a complex and rare disorder that causes lymphatic vessels around the heart and lung to leak fluid, causing breathing difficulties, infections, and often death. The treatment, which the research team has used successfully in other patients with lymphatic disorders, led to a complete resolution of the patients symptoms and fully remodeled her lymphatic system within a matter of months.

The findings were recently published in EMBO Molecular Medicine.

The resolution of lung disease with lymphatic remodeling is remarkable and potentially should change how we evaluate and treat lung disease in this patient population, said first author Jessica B. Foster, MD, an attending physician in CHOPs Division of Oncology. These results offer hope to other patients with lymphatic-induced lung disease and warrant further investigation.

Brenna, the patient described in the paper, first developed symptoms related to KLA at age 6 and was diagnosed with the condition when she was 10. For nearly a decade, she was treated with a variety of therapies, including rapamycin, an immunosuppressant; prednisone, a steroid; and vincristine, a chemotherapy drug. Despite aggressive treatment, her debilitating symptoms persisted, and so her clinical team at CHOP decided to explore other therapies.

Many patients with KLA have a mutation in the NRAS gene, and the team had previously used a mitogen-activated protein kinase (MEK) inhibitor called trametinib in another patient with a severe lymphatic disorder and NRASmutation. The drug resolved his symptoms and completely remodeled his lymphatic system. Although that patient did not have KLA, the researchers felt the success of trametinib in treating his severe lymphatic symptoms, combined with Brennas rapidly deteriorating condition, warranted the request for expanded access to treat Brenna with the drug.

As Brenna began taking trametinib, a drug historically used to treat metastatic melanoma, the researchers sent samples of her lymph fluid for genetic sequencing. The researchers learned that Brenna did not harbor an NRAS mutation and instead had a mutation in a different gene: CBL, a gene that operates along the Ras pathway, the same genetic pathway implicated in other lymphatic disorders, including the one for which they had used trametinib with great success. Mutations along the Ras pathway result in an overproduction of MEK, which leads to the uncontrolled proliferation of lymphatic vessels. A MEK inhibitor like trametinib brings the production of MEK under control, putting the brakes on a system in overdrive.

Brenna was the first patient with KLA to take trametinib for a lymphatic issue, and within four weeks of starting a low dose, her symptoms improved. Her shortness of breath, coughing, and difficulty breathing while lying flat disappeared. She was able to exercise again, and during the second cycle of her therapy, she began training for a 5K race.

The relatively low dose required for dramatic improvement in this case highlights that a small amount of trametinib may be sufficient to treat lymphatic disorders, which will likely limit the side effect profile compared to the higher doses used for oncologic cases, said senior author Yoav Dori, MD, PhD, Director of the Jill and Mark Fishman Center for Lymphatic Disorders at CHOP. We are now preparing ongoing prospective studies to evaluate Ras pathway inhibition in clinical trials of large cohorts of patients.

Follow up tests have shown significant improvement in Brennas restrictive lung disease, and the fluid in her lungs has disappeared. Her overabundant lymph vessels have remodeled themselves and are now behaving normally. Now 20 years old, she continues to take a daily dose of trametinib, which has kept her symptoms at bay.

Genomic evaluation of vascular anomalies such as KLA have only just begun in recent years, said Denise Adams, MD, Director of the Comprehensive Vascular Anomalies Program (CVAP), a CHOP Frontier Program. This case study demonstrates the power of collaborative, cutting-edge research that reaches across disciplines, from genetics to oncology to cardiology, to bring breakthrough treatments to patients.

Foster et al. Kaposiform lymphangiomatosis effectively treated with MEK inhibition, EMBO Molecular Medicine, online September 7, 2020, DOI: 10.15252/emmm.202012324

#

About Childrens Hospital of Philadelphia: Childrens Hospital of Philadelphia was founded in 1855 as the nations first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, Childrens Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. In addition, its unique family-centered care and public service programs have brought the 564-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu

More here:
CHOP Researchers Use Precision Medicine to Reverse Severe Lymphatic Disorder in a Patient with KLA - Newswise

CAMBRIDGE, Mass., Oct. 01, 2020 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that management will participate in the following upcoming investor conference:

Chardan Virtual 4th Annual Genetic Medicines ConferenceFireside ChatDate: Monday, October 5, 2020Time: 10:30 a.m. ET

The event will be webcast live and may be accessed on the Editas Medicine website in the Investors and Media section. Archived recordings will be available for approximately 30 days following the event.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a (also known as Cpf1) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Investor ContactMark Mullikin(617) 401-9083mark.mullikin@editasmed.com

Media ContactCristi Barnett(617) 401-0113cristi.barnett@editasmed.com

Read more here:
Editas Medicine to Participate in Upcoming Investor Conference - GlobeNewswire

Oct. 7, 2020 11:00 UTC

BEDFORD, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc., (Nasdaq: STOK), a clinical-stage biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced plans to move forward with dosing of STK-001 in children and adolescents in its ongoing Phase 1/2a MONARCH study for Dravet syndrome. Following recent interactions with the U.S. Food and Drug Administration (FDA) related to the partial clinical hold on higher dose levels in the MONARCH study, the FDA will allow the Company to add an additional higher dose level to the single ascending dose (SAD) portion of the study (previously Part A). A total of three dose levels will now be evaluated in this portion of the study: 10 mg, 20 mg and 30 mg. Dosing above 30 mg in this study remains on FDA partial clinical hold.

In addition, subject to FDA review, the Company is preparing to add a multiple ascending dose (MAD) portion to the MONARCH study, replacing Part B. This decision is based on new preclinical repeat-dose toxicology data, which were reviewed by the FDA as part of ongoing discussions with the Company. There were no adverse effects observed in the non-human primate (NHP) repeat dose study. The Company plans to submit a protocol amendment to the FDA, which will reflect these changes to the SAD and MAD portions of the study.

There is an urgent need for more effective medicines for people who are living with Dravet syndrome, so we are pleased to be moving ahead quickly with our plans to continue dosing children and adolescents in this important Phase 1/2a study of STK-001, said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. We appreciate the FDAs timely review of our additional data and look forward to evaluating a total of three individual dose levels in the single ascending dose portion of the study. In addition, we are encouraged by preclinical data that demonstrated the ability to safely achieve greater exposure levels with multiple doses of STK-001. Based on these data, we plan to also evaluate multiple ascending doses of up to 30 mg in this ongoing study. Our team is working diligently to submit a revised protocol to the FDA in the coming days.

In March 2020, the Company announced the FDA had placed a partial clinical hold on higher doses of STK-001 in the MONARCH study, pending additional preclinical testing to determine the safety profile of doses higher than the current no observed adverse effect level (NOAEL). When intrathecal doses above the NOAEL were administered to NHPs, adverse hind limb paresis was observed. This finding is known to occur following intrathecal delivery of antisense oligonucleotides (ASOs) to NHPs and is not known to translate to the human experience. When extremely high dose levels were administered, acute convulsions were observed immediately following STK-001 administration. The dose levels were well above the range of corresponding human doses that would ever be administered in the clinic, and were delivered in a formulation that was at a higher concentration than would be administered in the clinic. There is no apparent correlation of these acute adverse events with the mechanism of action of STK-001.

Enrollment and dosing in MONARCH is ongoing. Preliminary safety and pharmacokinetic data are still anticipated in 2021.

About STK-001

STK-001 is an investigational new medicine for the treatment of Dravet syndrome. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-001. STK-001 has been granted orphan drug designation by the FDA as a potential new treatment for Dravet syndrome.

About Phase 1/2a Clinical Study (MONARCH)

The MONARCH study is a Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome and have evidence of a pathogenic genetic mutation in the SCN1A gene. The primary objectives for the study will be to assess the safety and tolerability of STK-001, as well as to characterize human pharmacokinetics. A secondary objective will be to assess the efficacy as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency over a 12-week treatment period. Stoke also intends to measure non-seizure aspects of the disease, such as quality of life, as secondary endpoints. Stoke plans to enroll approximately 48 patients across 20 sites in the United States.

About Dravet Syndrome

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures, beginning within the first year of life. Dravet syndrome is difficult to treat and has a poor long-term prognosis. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. The effects of the disease go beyond seizures and often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, chronic infections, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome affects approximately 35,000 people in the United States, Canada, Japan, Germany, France and the United Kingdom, and it is not concentrated in a particular geographic area or ethnic group.

About Stoke Therapeutics

Stoke Therapeutics (Nasdaq: STOK), is a clinical-stage biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company on Twitter at @StokeTx.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectation about the dosing, timing and execution of our Phase 1/2a MONARCH study of STK-001, including our ability to include a multiple ascending dose portion in the study and the partial clinical hold on Part B of the study; the expansion of our pipeline and the use of the TANGO platform to treat other genetic diseases; our ability to use study data to advance the development of STK-001; the ability of STK-001 to treat the underlying causes of Dravet syndrome; and the ability of TANGO to design medicines to increase protein production and the expected benefits thereof. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop, obtain regulatory approval for and commercialize STK-001 and future product candidates; the timing and results of preclinical studies and clinical trials; the risk that positive results in a clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; failure to protect and enforce our intellectual property, and other proprietary rights; failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; environmental risks; risks relating to the use of social media for our business; and the other risks and uncertainties that are described in the Risk Factors section of our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201007005223/en/

More here:
Stoke Therapeutics Announces Plans to Move Forward With Dosing of STK-001 in Children and Adolescents in its Ongoing Phase 1/2a MONARCH Study for...

THE zebrafish a popular addition to many fish tanks may hold the key to curing blindness.

Scientists found the fish can regrow body parts, including retinas.

2

2

Damage to cells in the retina, light sensitive tissue at the back of the eye, is one of the most common causdes of blindness.

The fish can repair the optic nerve carrying vital information to the brain in just a week.

Zebrafish share around 70 per cent of their genes with humans.

And because their flesh is nearly transparent during development, researchers can observe their internal organs.

SURFACE ISSUECovid does NOT spread through surfaces like door handles, scientists claim

LIFE THREATENING Covid-linked Kawasaki-like disease 'can be fatal in adults & kids'

HOT SPOTSCoronavirus cases soar in Manchester and 6 other parts of UK after testing glitch

TRACK & DISGRACE'50k' Brits not told to isolate after 'Excel spreadsheet file got too big'

Warning

HORROR SHOWThe Exorcyst doc squeezes 10-year-old fatty lump size of a ball from man's back

TOTALLY DENTALThe dangerous dentistry hacks flooding TikTok - from nail files to BLEACH

US research showed a reduction in a chemical in the fishs brain triggered cells into action.

Prof James Patton said: These cells then migrate to damaged retina and differentiate into whatever is needed for repair.

It is hoped one day, under the care of their doctor, patients will simply be able to grow new retinas - just like the zebrafish.

GOT a story? RING The Sun on 0207 782 4104 or WHATSAPP on 07423720250 or EMAILexclusive@the-sun.co.uk

Original post:
Fish tank favourite zebrafish could hold the key to curing blindness in humans - The Sun

A LANDMARK will be turned yellow to mark Blindness Awareness Day in support of little Elsie Steinbach.

The two-year-old, who regularly visits the Clacton Pier with her family, was diagnosed with an underdeveloped optic nerve when just four and a half months old.

Optic Nerve Hypoplasia (ONH) is a very rare condition and was formed in early pregnancy.

It means Elsie has a severe visual impairment and she has her own Facebook page to raise awareness and combat blindness with her latest fundraiser topping 10,000.

Elsie, who lives in Basildon with parents, Hayley and Zak, and the family has a caravan in Harwich.

Along with her Auntie Kerrylea Collins and her family, they go to Clacton Pier about once a month.

It was Kerrylea who contacted the pier to make a request for the lighting to be turned yellow on Thursday, October 8.

It has been very difficult for my sister and brother-in-law but along with Elsie they have done amazingly well as a family, she said.

Now they are trying hard to raise awareness and money for research.

Elsie loves the pier and feels safe there with her cousins.

"We will all be going to see it lit up yellow as this is a shade she can recognise.

Fundraising has been particularly hard this year due to Covid so the pier doing this for Elsie and us all as a family is really a lovely thing to help raise awareness.

Elsie goes to pre-school in Basildon where she is doing well with one-to-one help.

Pier director Elliot Ball said it is a pleasure to be able to support the family.

We love to hear from our customers, and they are all important to us, he added.

Little Elsie is battling to cope with her condition, and we are delighted to be able to back her by turning our lights yellow.

"They will remain that colour until October 11.

Read more from the original source:
Family raise awareness of rare condition in support of Elsie, 2 - Gazette

Electrogas director and shareholder Paul Apap Bologna was cautioned several times by the Board of Inquiry looking into journalist Daphne Caruana Galizias assassination as he told the judges he was unaware of a number of factors tied to Electrogas, some of which had been in the public domain for several months if not years.

Apap Bologna said he did not know why prime murder suspect Yorgen Fenech had resigned from Electrogas in November 2019 and that he was unaware of the reason for Azerbaijani-British national Turab Musayevs resignation in December 2019 both happened at a time when protests were being held daily in Malta following revelations of links between Caruana Galizias death and the Electrogas project.

He also said he did not know that Electrogas had gone into default, despite it being mentioned in the Auditor Generals report. Apap Bologna said that he did not read the Auditor Generals report in its entirety. The Board of Inquiry was incredulous, with Judge Abigail Lofaro warning him repeatedly that he was under oath.

How do you not know the conclusions of the report? You have a right to disagree with it, but do not tell me that you do not know! Judge Emeritus Joseph Said Pullicino said in disbelief.

You dont care, it doesnt bother you. Thats a logical conclusion, Judge Lofaro added.

Asked about whether Electrogas had confronted Fenech after the media reported that he was the owner of 17 Black, Apap Bologna said that they had asked Fenech if 17 Black was his and he did not answer.

A director is involved in a transaction and youre asking about his involvement and he isnt answering you! Said Pullicino stressed.

This is incredible, Im sorry, Judge Lofaro said.

They were media accusations, Apap Bologna said.

He also told the Board that he was not aware of the contents of a statement issued by Gasan saying that they are seeking to exit the project. He said that they had not discussed the statement.

You are business partners, you are family, and he doesnt tell you, said Lofaro, who grew increasingly frustrated throughout the sitting. Apap Bolognas wife is a member of the Gasan group.

On several occasions, the Electrogas director told the Board that he will need to check and get back on basic questions related to operations and financials.

He repeatedly denied having presented the project to the Labour Party prior to the Party winning the elections in 2013 and the energy project being one of the main electoral promises.

He told the Board that he had presented a research paper, drawn up with the help of a friend in the electricity generation sector overseas, in 2007 to the then-Nationalist administration, including to John Dalli. Yet the project did not move forward with the administration and it was eventually shelved, Apap Bologna said.

It is not the first time that Dallis name has cropped up in connection to the Electrogas project. Dalli had been one of the faces published by Caruana Galizia in her first mention of 17 Black. Dallis sister, Anna Fenech, is also a shareholder in the company.

Asked whether Fenechs company New Energy Supply Ltd, which holds 8.17% of the project over and above the other families shares was a vehicle for kickbacks, Apap Bologna replied that Fenech had requested that share as compensation for dedicating his time to the project.

Apap Bologna denied contributing money to the Labour Party prior to the election and said that the first time they presented the project to the Labour Party was following statements on the matter by disgraced former minister Konrad Mizzi in January 2013.

It was then that he spoke to Fenech about it since the Fenech family has the team, the experience with project management as well as the financial means.

Yet it was pointed out to him by the lawyers representing the Caruana Galizia family that the financials Mizzi presented were very specific and matched the proposal he had presented in previous years. The fact that the project was put together in six weeks including tech giant Siemens and Gasol was also noted.

Apap Bologna told the Board that it was Gasol that got SOCAR and Siemens on board. He did not have any qualms about the Azerbaijani interest since they were traders in oil and were introduced by Gasol, whom he trusted.

Did Apap Bologna know of Gasols financial problems? Again, no.

In a post following the sitting, former PN General Secretary Paul Borg Olivier retorted that it was in 2009 that Apap Bologna had approached him and that at that time research he had done on Gasol showed them to be very shady, to say the least.

Borg Olivier said Apap Bologna had mentioned a different year in a bid to distance the meeting to a time before Joseph Muscat became Leader of the Opposition.

Apap Bologna insisted that he was not aware of Fenechs relationship with former Chief of Staff Keith Schembri and Muscat.

That sounds naive, but anyway, be that as it may, said Lofaro.

Apap Bologna said he got to know about the relationship between Fenech and Schembri/Muscat well after the bid.

Did you not question it? asked Comodini Cachia.

Asked what action was taken, Apap Bologna said they spoke to Fenech and after allegations started an audit, which was also encouraged by their auditors PWC.

Following the Panama Papers, Apap Bologna said they were assured by the directors that there was no wrongdoing.

The former prime minister, his chief of staff and the minister who was the face of the Electrogas project were forced to resign.

Continue reading here:
A tale of wilful blindness - The Shift News

WALTHAM, Mass., Oct. 03, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced the results of a post hoc analysis of the Phase 2 FILLY study investigating intravitreal pegcetacoplan (APL-2) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The post hoc analysis found that monthly treatment with pegcetacoplan significantly reduced the rate of progression from nascent GA to GA in areas of the retina outside of existing GA lesions. The data were presented today in a late-breaking oral session at the European Society of Retina Specialists 2020 Virtual (EURETINA).

Pegcetacoplan is the only targeted C3 therapy in Phase 3 clinical trials for GA, a complement-driven eye disease1,2 that causes blindness, affects approximately five million people globally3,4 and has no approved treatment.

Our findings from this post-hoc analysis demonstrate that intravitreal pegcetacoplan, a targeted C3 therapy, significantly lowers the rate of progression from nascent GA to GA in patients when compared to sham controls, said SriniVas Sadda, M.D., President & Chief Scientific Officer of the Doheny Eye Institute and lead investigator. This study provides exciting evidence to support further exploration of the potential of pegcetacoplan for earlier intervention in the course of GA.

The post hoc analysis, a collaboration with the Doheny Image Reading Research Lab, included FILLY patients from the monthly pegcetacoplan-treated group (n=42) and sham-treated group (n=69) who completed the Month 12 study visit and who did not develop exudative or neovascular AMD. The objective of the analysis was to investigate the effects of pegcetacoplan on complement-driven progression of AMD outside of GA lesions.

Patients with geographic atrophy experience changes in the retina that progress from the earlier stages of AMD to the beginning of atrophy and ultimately irreversible vision loss, said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. This post hoc analysis of the FILLY study demonstrates that pegcetacoplan slows early disease progression and may have the potential to delay the onset of GA and vision loss in patients.

Earlier this year, Apellis announced the completion of patient enrollment in the ongoing Phase 3 DERBY and OAKS studies investigating pegcetacoplan in patients with GA secondary to AMD. Top-line results from these pivotal trials are expected in Q3 2021.

About Pegcetacoplan (APL-2)Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.

About Geographic Atrophy (GA)GA is an advanced form of age-related macular degeneration (AMD), a leading cause of blindness. Excessive complement activation drives irreversible lesion growth in GA5, and C3 is the only target to precisely control complement overactivation. Pegcetacoplan, studied in early and late-stage trials comprising a total of approximately 1,500 patients, is the only targeted C3 inhibitor being evaluated in late-stage trials to control lesion growth in GA.6

GA lesions affect the central portion of the retina, known as the macula, which is responsible for central vision. GA is progressive and irreversible, leading to central visual impairment and permanent loss of vision. Based on published studies, approximately one million people have GA in the United States and 5 million people have GA globally.1,2 There are currently no approved treatments for GA.

About FILLYThe FILLY study was a 246-patient, Phase 2, multicenter, randomized, single-masked, sham-controlled clinical trial evaluating pegcetacoplan in patients with GA secondary to AMD conducted at over 40 clinical sites in the United States, Australia and New Zealand. Pegcetacoplan was administered as an intravitreal injection monthly or every other month (EOM) for 12 months, followed by six months of monitoring after the end of treatment. The primary efficacy endpoint was the change in GA lesion area from baseline to Month 12 in pegcetacoplan-treated patients compared to sham.

About DERBY and OAKSDERBY (621 patients enrolled) and OAKS (638 patients enrolled) are Phase 3, multicenter, randomized, double-masked, sham-controlled studies to compare the efficacy and safety of intravitreal pegcetacoplan with sham injections in patients with GA secondary to AMD. The primary objective of the studies is to evaluate the efficacy of pegcetacoplan compared to sham injection in patients with GA secondary to AMD assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence (FAF).

About ApellisApellis Pharmaceuticals, Inc. is a global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. Leaders in targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.

Apellis Forward-Looking StatementStatements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the implications of preliminary clinical data. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether the companys clinical trials will be fully enrolled and completed when anticipated; whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; whether results obtained in preclinical studies and clinical trials will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the companys clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G or any other indication when expected or at all; whether, if Apellis products receive approval, they will be successfully distributed and marketed; and other factors discussed in the Risk Factors section of Apellis Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on July 30, 2020 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Media Contact:Mark Dolemedia@apellis.com617.997.3484

Investor Contact: Sam Martin / Maghan MeyersArgot Partnerssam@argotpartners.com / maghan@argotpartners.com212.600.1902__________________________________

1 Weber, BHF, Issa, PC, et al. The Role of the Complement System in Age-Related Macular Degeneration. Dtsch Arztebl Int 2014; 111(8): 1338.

2 Heesterbeek, TJ, Lechanteur YTE, et al. Complement activation levels are related to disease stage in AMD. Invest Ophthalmol Vis Sci. 2020;61(3):18.

3 Rudnicka AR, Jarrar Z, Wormald R, et al. Age and gender variations in age-related macular degeneration prevalence in populations of European ancestry: a meta-analysis. Ophthalmology 2012;119:571580.

4 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106116.

5 Seddon, JM, Rosner, B. Validated prediction models for macular degeneration progression and predictors of visual acuity loss identify high-risk individuals. Am J Ophthalmol 2019;198:223261.

6 Yates, JRW, Sepp T, et al. Complement C3 Variant and the Risk of Age-Related Macular Degeneration. N Engl J Med 2007;357.

Visit link:
Apellis Announces New Analysis Demonstrating Targeted C3 Therapy Pegcetacoplan Slows Progression of Early Disease in Patients with Geographic Atrophy...

Glaucoma is the leading cause of irreversible blindness in India

Maxivision super speciality eye hospital, Madinaguda/Chandanagar, Hyderabad.

It is an annual day of awareness held on the second Thursday of October, to focus on global blindness and vision impairment. It falls on 8 th October 2020 .it is coordinated by International Agency for prevention of blindness (IAPB) under WHO VISION 2020 , The Right to Sight , Global initiative.

This year the theme is Hope in Sight

More than 75% of visual impairment is avoidable. After Cataract, Refractions Errors and Diabetic eye disease comes Glaucoma. The blindness due to these major problems can be prevented.

Glaucoma is the leading cause of irreversible blindness in India and at least 12 million people affected and nearly 1.2 million people blind from the disease. More than 90% of glaucoma cases remain undiagnosed.

Early diagnosis and treatment of Glaucoma is the main goal to prevent blindness.

This world sight day, the main focus is on the awareness of importance for regular eye check up in people above 40 years ,

The public are requested to take note of these above points and have a complete eye examination by an ophthalmologist for early diagnosis of Glaucoma and prevent their eyes from going blind.

A slit lamp examination, applanation tonometry, visual field testing and dilated fundus examination are the key tests to be undergone periodically for prevention of blindness.

Glaucoma can be prevented if diagnosed early and further progress can be stopped by local eye drops medications, LASER treatment to the eyes or surgical interventions.

Therefore, the public are requested to take this initiative of Hope in Sight on this world sight day by IAPB and get their eyes examined for Glaucoma and other eye disease for early detection and prevention of the problem. Maxivision eye hospitals have all the latest equipment and technology for an early detection of the diseases.

An appeal to the public in this Covid times we at Maxivision super speciality eye hospital are taking all precautions for a safe eye examination or surgery to the patient and all our staff and Doctors are following the covid protocols sincerely.

BY:Dr K.UMA,Senior consultant, Glaucoma & Cataract services

Continued here:
World sight day 2020(WSD) on 8th October: This year theme 'Hope in Sight ' - NewsPatrolling

Professor Mark Gillies with a patient.

Co-author and ophthalmologistProfessor Mark Gilliesfrom the University of SydneyFaculty of Medicine and HealthandSave Sight Institutesaid the findings reinforced the need for more education.

Ninety-eight percent of serious vision loss from diabetes can be prevented with regular eye examinations and early treatment, said Professor Gillies.

I encourage people to use services like KeepSight to keep on top of their appointments. Its also important they understand the kind of eye check required, as only eye checks that include dilation of the pupil with eye drops (fundus dilation) is appropriate to detect changes in the eye-related to diabetes.

Diabetes AustraliasKeepSight program, which commenced just over 12 months ago, is helping to ensure that the proportion of people with diabetes accessing eye checks increases in coming years and ultimately, every person with diabetes get the necessary eye checks and this helps prevent vision loss and blindness.

KeepSight is an online eye check reminder program easily accessed from a mobile phone.

Diabetes Australia CEO Professor Greg Johnson said KeepSight has enrolled 100,000 people since it started last year.

Having 100,000 Australians with diabetes registered with KeepSight is an important milestone for the program but there are currently over 1.36 million Australians living with diabetes so we are encouraging every person with diabetes, and all health professionals, to register with KeepSight, said Professor Johnson.

Keep Sight provides electronic alerts and reminders to help people with diabetes remember their diabetes eye checks. When its time for a diabetes eye check you get a reminder. Its that simple. KeepSight can also help you find an optometrist if you dont know one.

The KeepSight program, which is run by Diabetes Australia in partnership with Vision 2020 Australia, Centre for Eye Research Australia and Oculo, has been co-funded by the Australian Government, Specsavers, Bayer, Novartis and Mylan. The program has widespread support from leading diabetes and eye health groups including the Royal Australian and New Zealand College of Ophthalmologists, Orthoptics Australia, Optometry Australia, the Australian Diabetes Society and the Australian Diabetes Educators Association.

The University of Sydney-led research is part of a series of population-based record linkage projects using the NSW 45and Up study to evaluate the uptake and long-termhealth impact of government-funded services and programs implemented to support care and reduce complications in people with diabetes.

Here is the original post:
Living with diabetes Get your eyes checked - News - The University of Sydney

DORVAL, QC, Oct. 5, 2020 /CNW Telbec/ - Novartis Pharmaceuticals Canada Inc. (Novartis) is pleased to announce that Beovu (brolucizumab injection) is now available in Canada for the treatment of neovascular (wet) age-related macular degeneration (AMD). The next-generation anti-VEGF therapy for wet AMD, offers the option for eligible patients to start on three-month dosing intervals after the loading phase2,3. Beovu has been reviewed by the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) and has received a positive recommendation for reimbursement by participating public health plans1.

With wet AMD, which can be aggressive in its progression, abnormal blood vessels form and grow under the central retina (macula) and cause it to swell up, distorting central vision. These abnormal blood vessels may leak fluid or blood in the eye, interfering with the macula's function and causing increasingly severe central vision loss.2,4

"Novartis has a deep history in ophthalmology and an ongoing commitment to eliminating preventable blindness by bringing innovative treatments to patients with serious eye diseases. We are dedicated to continuing to invest in research and as well as programs that improve the lives of people with wet AMD and are very proud to bring this latest advancement to Canadians," said Andrea Marazzi, General Manager, Novartis Pharmaceuticals Canada Inc. "We are pleased that the clinical value and benefit of Beovu have been recognized by CADTH and look forward to collaborating with decision makers to ensure access to Canadians in a timely manner."

AMD is the leading cause of vision loss in people over the age of 50, affecting more than a million Canadians. Symptoms of AMD involve loss of central vision, which is needed for recognizing faces, reading, driving and other daily activities. AMD comes in both dry and wet forms, with wet AMD being rarer and much more severe.4

"It's so easy to attribute a change in eyesight to getting older. We have been working hand in hand with Canadian vision organizations to drive the message across that any changes in vision should be immediately addressed. We know just how profound the impact of untreated wet AMD can be on a person's quality of life. Keeping up with injections may be challenging on older adults who are less inclined to want to visit clinics and hospitals. This is truer today than ever before," said Louise Gillis, President, Canadian Council of the Blind. "We are pleased that Beovu is available in Canada. It's a new option that offers the potential for Canadians diagnosed with the leading cause of blindness to maintain their vision and not have to worry about frequent visits for their injections. Missing out on treatment could potentially be catastrophic for someone with wet AMD."

Anti-vascular endothelial growth factor (anti-VEGF) therapy is widely regarded as the standard of care for patients with wet AMD1. Approved by Health Canada in March 2020, Beovu is the latest available anti-VEGF therapeutic advance, addressing the strong need for a treatment that reduces the need for frequent follow up.

Beovu (brolucizumab injection) Important Safety InformationThe full prescribing information for Beovu can be found at: http://www.novartis.ca.

About Novartis in ophthalmologyAt Novartis, our mission is to discover new ways to improve and extend people's lives. In ophthalmology, we develop and deliver life-changing medicines and therapies for diseases and conditions from front to back of the eye, enabled by data and transformative technologies. Our ophthalmic solutions reach more than 150 million people per year, from premature infants to the elderly.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2019, the company invested $51.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,500 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consultwww.novartis.ca.

About NovartisNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 105,000 people of more than 140 nationalities work at Novartis around the world. Find out more at http://www.novartis.com.

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Lori Bogdanis, Novartis Pharmaceuticals Communications, +1 514 708 4801, E-mail: [emailprotected]

http://www.novartis.ca

Read the original:
Next-generation wet AMD therapy Beovu, now available in Canada, receives positive recommendation from CADTH Drug Expert Committee - Canada NewsWire

When Jim OConnell joined the University of Florida as the assistant vice president of technology commercialization in 2016, UF Innovate | Ventures was part of his overall plan. He secured the money for the venture fund, recruited a seasoned director, and made Ventures a reality last September. The goal: quality UF startups.

Startups play a significant role in economic growth creating jobs, introducing new ideas and solutions to the marketplace, said OConnell, but without proper leadership, direction, and funding, the odds are against them. Ventures seeks to support UF startups in those areas.

Toward this aim, Ventures hired four Entrepreneurs in Residence, selected an investment committee and advisory council, invested in four different startups, created a network of investors on speed-dial, and helped new startups form and strengthen.

The EIRs have provided valuable insight and connections to investors and entrepreneurs across the state of Florida, said Jackson Streeter, MD, director of Ventures. Before joining UF Innovate, Streeter was the head of the Florida Technology Seed Capital Fund. He also served as CEO for Banyan Biomarkers, a UF startup.

Meet the entrepreneurs in residence

The four Entrepreneurs in Residence turn early business concepts into fundable companies, help companies find capital and strategic resources. One of the four said his biggest contribution to Ventures is offering warm introductions to his ties to companies, investor groups, successful entrepreneurs, and other talent that might provide value to the program.

Its been a pleasure working with the other Entrepreneurs in Residence from throughout the state of Florida to assist Jim OConnell and Dr. Jackson Streeter and the entire UF family, said Dr. Richard Munassi, an Entrepreneur in Residence located in the Tampa/St. Petersburg area. Being able to support the university, researchers, and students has been incredibly rewarding.

Munassi is a seasoned entrepreneur who has been working with tech startups for more than a decade; his primary focus is on healthcare technology, medical devices, and health IT. Entrepreneur in Residence Chris Carter of Ponte Vedra Beach is an angel investor and has served in a variety of mentorship, advisory, consulting, and venture development roles in support of entrepreneurs.

Robert Mino, a triple Gator living in Palm Beach County, is a corporate and patent attorney with broad commercial and research experience beyond the practice of law. Kelly Murray of Orlando centers her work on bridging health innovation and affordable housing with private investment and collaboration in both the public and private sectors.

Though our backgrounds have similarities at some point, we also augment each other by providing value in different ways with regard to the entire life sciences, health tech, and biotech industries, said Munassi. I think its a really strong group top to bottom. Jim and Jackson did a great job of choosing the Entrepreneurs in Residence, and I feel lucky to be a part of that.

Meet the investment committee and advisory council members

In addition to the four Entrepreneurs in Residence, Ventures enlisted the help of Investment Committee and Advisory Council members who independently review and approve investments made by UF Innovate | Ventures and provide strategic advice to the fund and the portfolio companies.

It is very exciting that UF has for the first time created a venture fund, said Streeter. Most leading universities have associated venture funds, and now UF has as well.

Two members of the Investment Committee and Advisory Council, Bill Gurley and Rodney Rogers, are UF graduates. Gurley has spent 20 years at Benchmark as a venture capitalist working with such companies as GrubHub, OpenTable, Stitch Fix, Uber, and Zillow. Rogers has more than 30 years in the tech services industry and is a noted serial entrepreneur and expert technologist known for his leadership contributions to startups that achieved billion-dollar valuations.

Bob Crutchfield is the managing director of Hotspot Ventures. He has also served on the boards for companies such as Shipt, Wellcentive, nContact, and Novamin. Randy Scott is a partner and co-founder of HealthQuest Capital, a venture capital firm investing in companies that transform the value of healthcare. Chris Walker is a private equity expert with HarbourVest Partners who has focused most of his efforts on U.S. venture capital and buyout fund investments.

Companies funded in FY20

The group selected four companies to benefit from the $1 million venture fund Ventures can invest each fiscal year. FY20s recipients included:

An example as to how Ventures works

In addition to investing in those existing UF startups, Ventures also helps form new startups by evaluation of market opportunities, strategic planning, recruiting management and board members, and finding investors. This year, Ventures helped Agricultural Intelligence,Rain Neuromorphics, Adjuvolt, and others.

Adjuvolts story shows how Ventures works. It began in the UF vet school, which encounters numerous instances of osteosarcoma, bone cancer. Osteosarcoma is the most common form of bone cancer. It is highly metastatic. If it is located in a dogs leg, it requires amputation and chemotherapy, which is only moderately effective.

In interactions with the vet school, Ventures leader Streeter heard about a technology developed byRowan Milner, a cancer vaccine to treat osteosarcoma in dogs. This isnt a vaccine given to prevent a dog from developing cancer; its a vaccine given to a dog to treat cancer once a tumor has developed.

Developing effective cancer vaccines has proven challenging due to the difficulty of eliciting a robust and consistent immune response against self antigens. Dr. Milner has found a way to formulate a vaccine that targets an antigen specific to osteosarcomas. Hes used it hundreds of times in dogs with success.

Humans and dogs have shared histopathologic and genetic features. In humans, osteosarcoma is often found in children. Could this technology extend to human care?

Ventures explores the technology, decides it has value and potential. It then identifies management for a potential startup: Richard Currie, Ph.D., BVM&S. Currie is a veterinarian and an entrepreneur, has experience in the multinational pharmaceutical and diagnostic sectors.

A company is born. Ventures will continue to help guide Adjuvolt, connect it to investors. The hope is that Adjuvolt can take a vaccine created for dogs in a lab at UFs vet school and, eventually, create vaccines for humans. The sequence might save the leg of a small human suffering from osteosarcoma. And many more.

Sara Dagen October 5, 2020

Go here to see the original:
UF Innovate | Ventures celebrates first year successes - University of Florida

TEL AVIV, Israel, Oct. 5, 2020 /PRNewswire/ --Tarsius Pharma, a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat patients with blinding ocular diseases, announced today that the GADOT 20/20 trial of its novel TRS01 drug for treating Active Anterior Non-Infectious Uveitis, demonstrated significant improvement in critical measures such as ACC (Anterior Chamber Cells), pain reduction and increased visual acuity.

The GADOT 20/20 trial was a dose-ranging, randomized, double-masked, controlled phase I/II clinical trial that evaluated the safety and efficacy of TRS01 in 16 subjects with active non-infectious anterior uveitis in the U.S. The study compared two different doses of TRS01.

Results showed a statistically significant improvement in the signs and symptoms of the disease, with the high-dose usage being superior to low-dose in a few important measures. Firstly, a consistent clearing of the anterior chamber cells (ACC) from baseline was achieved. Secondly, prompt resolution of pain was present, alongside increase of visual acuity. Also, compared to rescued subjects that received steroids, a reduction of IOP (intraocular pressure) was demonstrated in patients of both treatment arms.

"The results of this clinical trial with TRS01 are truly impressive, and suggest meaningful progress towards the development of a novel and clinically important class of ocular therapeutics topical anti-inflammatory agents with efficacy that is at least equal to corticosteroids, but with none of the known corticosteroid-associated side-effects," stated Dr. Ron Neumann, Tarsius Pharma's CMO.

Dr. Daphne Haim-Langford, Tarsius' CEO, said: "The results of this study bring us one big step forward in our journey to save the eye-sight of patients with uveitic glaucoma, who have no approved treatment available today for this devastating condition. Completing this trial during the COVID-19 pandemic, proves once again the emergency nature of uveitis flare-ups. On behalf of our entire Tarsius team, I thank the investigators, volunteers, and all study team members for contributing to the professional execution and evaluation of the clinical process, and to the understanding of TRS01 in humans."

About Ocular Inflammatory Diseases

Ocular inflammatory diseases impose a significant medical and economic burden on society, affecting hundreds of million people worldwide and posing severe risks of vision loss and blindness.

In more than 50 years, corticosteroids are the most common method used to treat ocular non-infectious inflammation of any kind. Rather than treating the disease itself, however, steroids merely relieve its symptoms, and long-term use can result in life-changing consequences.

The TRS Platform Technology has the potential to effectively treat a broad array of autoimmune and inflammatory ocular diseases. After a successful PoC which demonstrated TRS01's safety and efficacy, Tarsius Pharma is well-positioned to advance its novel platform technology in other sight threatening ocular diseases.

About Tarsius Pharma

Tarsius Pharma was established in 2016and is focused on developing TRS, a breakthrough, bio-inspired platform technology for the treatment of blinding ocular diseases.

This project has received funding from the European Union's Horizon 2020 research and innovation program under grant agreement No. 879598.

Contact:info@tarsiuspharma.comTel: +1-914-3392556

Photo - https://mma.prnewswire.com/media/1307023/Tarsius_Pharma.jpg

View original content:http://www.prnewswire.com/news-releases/tarsius-pharma-achieves-positive-results-in-proof-of-concept-study-for-trs01-targeting-uveitis-301145540.html

SOURCE Tarsius Pharma

Visit link:
Tarsius Pharma Achieves Positive Results in Proof of Concept Study for TRS01 Targeting Uveitis - BioSpace

Ministry of Health is close to declaring Wadelai (Pakwach District) and West Nile (Koboko and Yumbe) foci as River Blindness Free

Ministry of Health is close to declaring Wadelai (Pakwach District) and West Nile (Koboko and Yumbe) foci as River Blindness Free

Uganda has stopped interventions in 2019 among 608,219 people in Nyagak Bondo focus previously treated for river blindness (Onchocerciasis), based on the current World Health Organization (WHO) guidelines, bringing the country closer to achieving its goal of eliminating river blindness nationwide. River blindness has been known as a public health problem affecting a significant population of Uganda since early 1950's.

The Government of Uganda declared a nationwide elimination policy in 2007. River blindness is a vector borne disease that is transmitted by the black flies which breed in fast flowing rivers. It causes serious eye and skin disease that may result in blindness. The disease was originally endemic in 40 districts in Uganda with about 4.6 million people at risk, excluding districts in the Victoria Nile focus that eliminated the disease in the early 1970's. CLICK HERE FOR MORE ON THIS NOTICE

Go here to read the rest:
Notice From Ministry Of Health - New Vision

The global intraocular lens market size was estimated at USD 3.95 billion in 2019 and projected to reach USD 6.40 billion at a CAGR of 5.5% over the forecast period. Different informative opinions and markets on the global intraocular lens market from different global C-level professionals and industry leaders have been embedded in the report. Different top-level key players across the global regions have been profiled to get better insights into the global intraocular market. Additionally, it offers a detailed elaboration on various aspects such as global market policies, rules, and regulations imposed by governments to set standards for global businesses. Furthermore, different global competitors and new entrants are also presented in the report for understanding the degree of competition globally. The global market research report has been presented in a clear and professional chapter-wise format for understanding the subject matter.

Get Sample Copy: https://www.alchemymarketresearch.com/healthcare/intraocular-lens-market/#req

The following factors are encouraging the global intraocular lens market growth over the forecast period:

Alchemy Market Research Profiling the Leading Companies Functioning In the Intraocular Lens Market:

Driving Factors:

Get Sample Copy: https://www.alchemymarketresearch.com/healthcare/intraocular-lens-market/#req

The global market intelligence report has been prepared with the help of the following pointers:

Key Questions Answered Through This Global Market Research Report:

Get Sample Copy: https://www.alchemymarketresearch.com/healthcare/intraocular-lens-market/#req

Here is the original post:
Intraocular Lens Market is projected to reach USD 6.40 billion at a CAGR of 5.5% - Credence Turbine

In this day and age, corporations are really able to do virtually anything and everything they could want. If they wish to open a new office or launch a new division, they are often able to do so. Why then, are so many companies turning to philanthropic measures? Let's take a look.

Public Pressure

Nowadays, brand loyalty can often mean nothing to customers. While you once would have picked one company and stayed with them for your entire life, this is not the case any longer. If a brand does something that is not approved of by many of their customers, there is a chance that they will leave and go to a competitor.

As a result, many corporations have turned to philanthropic pursuits. This is a great way for them to respond to the concerns of their customers while also acknowledging some of the major events and issues currently facing us worldwide. For example, many corporations are making a commitment to going carbon-neutral within their business dealings. While this will require an awful lot of work on their part, and might also involve some serious redirection within the business, it is a fantastic move that will satisfy old clients and potentially attract new ones too.

Caring for Causes

Of course, it is not just the public who have control over these philanthropic pursuits. Company directors and CEOs are also directing their corporations towards issues and areas that they personally care about.

Two great examples here are Tej Kohli and Bill Gates. Tej Kohli is the founder of the philanthropic Tej Kohli Foundation which is engaged in several missions around the world. In particular, they are committed to trying to eliminate corneal blindness, one of the most common types of blindness in the world. Bill Gates and his wife Melinda set up the Bill and Melinda Gates Foundation. They have been engaged in healthcare philanthropy to help improve the living conditions for many thousands of people all across the world.

Uplifting Others

As there continues to be a growing wealth gap between the top percentiles of our society, corporations are beginning to look into ways they can offer help to some of those lower down. This could be in the form of a scholarship to attend higher education, or it could be through the form of work experience within the company.

It could even be as simple as constructing a local community centre for everyone to use. There are many small ways that a corporation can provide better opportunities to those in poorer areas. We never know what someone can achieve when given the right tools for success.

This is a trend that is most definitely not going away. With more and more corporations turning to philanthropic efforts, we are likely to see a lot of good come out of this world in the future. Take a look at some of your favourite companies to see what they are doing, or even look at some of the paths that you could adopt as a business owner.

Read the rest here:
Why Corporations Are Turning To Philanthropy - International Business Times UK

The National Research & Training Center on Blindness & Low Vision (NRTC) is proud to announce our receipt of another competitive national grant. This grant, Independent Living Services for Older Individuals Who Are Blind Training and Technical Assistance, is sponsored by the Rehabilitation Services Administration under the U.S. Department of Education. This 5-year grant, which provides approximately $3 million in funding, begins October 1, 2020.

Sylvia Stinson-Perez is the project director for this grant known as the OIB-TAC. She is excited to take the strong foundation from the last five years of the NRTCs work on this grant and add new and innovative projects. There are three major products related to this grant:

Michele McDonnall, NRTC Director, is happy that the funding will allow the NRTC to continue to improve the provision of services to older individuals who are blind.

Read more from the original source:
The NRTC Awarded Second Major National Grant | The National Research and Training Center on Blindness and Low Vision - Mississippi State Newsroom

Avrobio has licensed a Hunter syndrome lentiviral gene therapy from the University of Manchester in the U.K. The deal positions Avrobio to join Regenxbio and Sangamo Therapeutics in the race to develop gene therapies for use in patients with the rare disorder.

Hunter syndrome, also known as mucopolysaccharidosis Type II, is driven by a mutation that limits a patients ability to break down sugar molecules generated as a byproduct of cell activity. The buildup of the molecules causes progressive damage to organs such as the brain, affecting the development of children with the lysosomal disorder.

Takedas approved drug Elaprase gives Hunter syndrome patients the enzyme they need to break the molecules down, but its inability to cross the blood-brain barrier renders it ineffective against the CNS manifestations of the disease.

Inside the COVID-SeroIndex Assay Design and Performance

Join a panel of experts as they discuss the rigor involved in optimizing and scaling a reproducible serological assay for COVID-19.

Avrobio has become the latest company to identify gene therapy as a potentially better way to treat Hunter syndrome. The biotech, which is active in other lysosomal disorders, has agreed to pay the University of Manchester $8 million upfront for the global rights to a stem cell gene therapy that is set to enter the clinic in the second half of next year.

Brian Bigger, Ph.D., a professor of cell and gene therapy at the U.K. university, developed the candidate and co-authored a paper about it in 2018. The paper describes work on a braintargeted hematopoietic stem cell gene therapy designed to give patients the lysosomal enzyme iduronate-2-sulfatase (IDS).

Avrobio will make the therapy, now called AVR-RD-05, by modifying a patients own hematopoietic stem cells with a transgene for IDS expression and a protein tag intended to improve stability of the enzyme. If AVR-RD-05 works as hoped, the cells will engraft in the bone marrow and make copies of themselves that also carry the transgene.

The differentiation of the cells into components of the immune system could enable AVR-RD-05 to cause a lasting, body-wide increase in the presence of the enzyme at the root of Hunter syndrome. Avrobio will fund a 9.1 million ($11.8 millon) investigator-sponsored phase 1/2 trial to assess whether the gene therapy performs as hoped. The biotech is also on the hook for up to $80 million in milestones.

Avrobio will face competition if it gets AVR-RD-05 all the way to market. Takeda is the incumbent, Regenxbio and Sangamo have gene therapies in the clinicalthough the latter has underwhelmed so farand Denali Therapeutics is developing an IDS enzyme designed to cross the blood-brain barrier.

Visit link:
Avrobio bags gene therapy to join Regenxbio and Sangamo in race - FierceBiotech

Professor Robert MacLaren on the exciting potential of CRISPR gene editing and patients seeing stars for the first-time following treatment

Professor Robert MacLaren, of Oxford University, is leading work to develop gene therapies for inherited retinal conditions through Nightstar Therapeutics. The company was acquired in 2019 by US biotechnology company, Biogen, for $US 877 million.

One night I was at my computer and a patient emailed me to say he could see a night star. This was a positive thing. I said, If you are still there can you go and check which eye it was. He came back and said, It is definitely the eye that I had the gene therapy in.

Subsequently, when we developed the gene therapy company, the name NightStar was an obvious one because since then several of our patients have described seeing night stars after gene therapy. It symbolizes looking up at the heavens and hope for the future, but there is also that practical element of achieving better vision at night.

Although I am an ophthalmologist treating patients, when we are looking at the mechanisms of the disease, we get right down to the molecular level, looking at how the DNA is assembled and how it is processed.

It is very exciting as a physician in any specialty to be able to treat disease at the genetic level. Ultimately if the disease occurs at the genetic level and you treat it there, you are treating the origin of the disease itself. If you are just giving drugs to try and correct the genetic disease or compensate for it, it may help but that is not a real treatment. It provides insight into the mechanisms of life itself seeing how DNA is processed and how the genes evolve.

You can treat potentially any disease with that, whereas gene replacement therapy is limited to small genes. This is still good but ideally if you correct the mutation it is a better treatment than replacing the whole gene.

See the original post here:
Gene therapy: It provides insight into the mechanisms of life itself - AOP