Record number of stem cell donors apply after Ex On The Beach star plea – expressandstar.com

Ashley Cain with his girlfriend Safiyya and daughter Azaylia Diamond

A blood cancer charity has seen record numbers of people applying to become stem cell donors following a plea from former footballer and Ex On The Beach star Ashley Cain.

Mr Cains 12-week-old daughter Azaylia Diamond Cain was diagnosed with an aggressive form of leukaemia when she was two months old.

Doctors recently told the reality star and his girlfriend Safiyya that, while their daughter had responded well to her chemotherapy and treatment, she would require a stem cell transplant from a stranger to be cured.

Anthony Nolan, a charity which recruits people aged between 16 and 30 to join the stem cell register, is now searching for a donor for Azaylia.

Mr Cain appealed for potential donors to sign up on Friday and the charity has since seen 41,000 people apply within 48 hours, compared to the usual 100 per day.

Mr Cain said: You know your own child and we knew she wasnt well.

She has a rare and aggressive form leukaemia thats made even rarer by her being only eight-weeks-old when she was diagnosed.

She has tumours on her lungs, stomach and kidneys.

Finding out that your baby has a serious and life-threatening illness is something no parent in the world should have to go through.

It was the single most upsetting, terrifying and heartbreaking experience we have ever been through.

Something that is not only incomprehensible, but it also has a devastating impact on our physical and mental health.

Azaylia has Caribbean, Indian and northern European ethnicity, which means she has a 20% chance of finding an unrelated stem cell donor match, compared to a 69% chance for people with white European heritage.

Mr Cain added: Its out of Azaylias hands now, shes almost climbed this mountain, but she now needs someone elses hand to help her get to the top.

She cant do this on her own, she needs a donor.

As well as appealing for more donors to apply, Anthony Nolan is appealing for financial support as it costs 40 for each donor to be registered.

Henny Braund, chief executive at the charity, said: We are extremely grateful to Ashley for using his platform to raise awareness of the need for more stem cell donors.

Its incredible that so many people have been inspired by Azaylias story to join the register.

Every single one of the new potential donors has the potential to give hope to little girls, like Azaylia, who are in desperate need of an urgent stem cell transplant.

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Record number of stem cell donors apply after Ex On The Beach star plea - expressandstar.com

Bisbee: A commitment to health care initiatives The Journal Record – Journal Record

Julie Bisbee

This month marks the 20th anniversary of voters creation of the Oklahoma Tobacco Settlement Endowment Trust, a grant-making state agency devoted to preventing cancer and cardiovascular disease, Oklahomas leading causes of death. Past accomplishments and new initiatives give Oklahomans reason to be proud of TSET.

In 1998, 46 states including Oklahoma reached the Master Settlement Agreement with Big Tobacco, resolving a lawsuit over the tobacco industrys practice of lying to the public about smoking dangers. In 2000, voters approved a constitutional amendment that safeguarded the bulk of the MSA payments in an endowment. Endowment investment earnings fund grants and programs to improve health.

On Nov. 3, voters affirmed the wisdom of TSETs structure, with each of Oklahomas 77 counties rejecting a state question that sought to reduce the contribution to the TSET endowment. This vote upholds the voter-created structure that funds tobacco use cessation and prevention, cancer research, community-based programs and other initiatives that improve quality of life.

Since 2000, TSETs funding of cancer research has given Oklahomans access to cutting-edge cancer treatment. TSETs funding of Phase 1 clinical trials helped the Stephenson Cancer Center achieve the prestigious National Cancer Institute designation.

To date, TSET has funded not only the Stephenson Cancer Center but also the TSET Health Promotion Research Center and the Oklahoma Center for Adult Stem Cell Research. For every dollar TSET has invested, research institutions have attracted an additional $3 in outside funding.

TSET also addresses physician shortages. This year, TSETs partnership with the Physician Manpower Training Commission filled all 42 slots in its loan repayment program, which helps pay off medical school loans when doctors agree to practice in rural and underserved areas.

TSETs Healthy Living Program is another success. Now in its second five-year grant cycle, the program works at the local level to improve health and make it easy for people to make healthy choices.

Policies help shape the environment. Tobacco-free policies protect the public from toxic secondhand smoke and encourage tobacco users to quit. Thanks to work of TSET grantees at the local level, more than 80% of school districts had adopted a tobacco-free policy before state law was enacted to protect all Oklahoma children from tobacco and e-cigarette use.

TSET remains committed to preventing cancer and cardiovascular disease. Visit tset.ok.gov.

Julie Bisbee is executive director of the Tobacco Settlement Endowment Trust, a voter-created grant-making trust devoted to preventing cancer and cardiovascular disease, Oklahomas leading causes of death.

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Bisbee: A commitment to health care initiatives The Journal Record - Journal Record

California election results: Where state propositions stand as of Thursday at 5 p.m. – Desert Sun

Associated Press Published 2:01 p.m. PT Nov. 4, 2020 | Updated 11:53 p.m. PT Nov. 5, 2020

Uber and Lyft decals are shown on a vehicle at Palm Springs International Airport on Wednesday, Jan. 22, 2020 in Palm Springs, Calif.(Photo: Vickie Connor/The Desert Sun)

Access to Uber and Lyft rides proved to be important to California voters Tuesday, but raising property taxes on commercial properties in the statewas narrowly defeated in one of the most hotly contested of 12 state propositions.

Voters also gave a huge defeat to an attempt to require certified doctors at kidney dialysis clinics and also rejected anattempt to re-establish affirmative action in hiring in California.

Following are results as of Thursday at 5 p.m. as reported by the California Secretary of State.

Uber, Lyft and other app-based ride-hailing and delivery services spent $200 million in a winning bet on Proposition 22 to circumvent California lawmakers and the courts to preserve their business model by keeping drivers from becoming employees eligible for benefits and job protections.

The titans of the so-called gig economy bankrolled the most expensive ballot measure in state history, which was decided Tuesday with 58.5% of voters choosing to keep drivers classified as independent contractors able to set their own hours.

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More: Photos: Election Day voting across Coachella Valley, Riverside County

More: Raul Ruiz claims re-election in 36th Congressional District; Cruz not conceding with votes uncounted

The outcome was a defeat for labor unions that had pushed for a state law aimed directly at Uber and Lyft, mandating they provide drivers with protections like minimum wage, overtime, health insurance and reimbursement for expenses.

Supporters of Proposition 22 said the outcome showed voters wanted to preserve the flexibility of the current system. Opponents said the companies had bought their own law and vowed to continue fighting for drivers' rights.

A ballot measure to partially dismantle Californias longtime system of tying property taxes to the last sales price appears to have lost byjust over 400,000 among the 11.5 million ballots cast. No votes on Proposition 15 garnered 51.8%of the votes.

The measure would have reassessed commercial and industrial properties every three years. Residential property would have remained under current rules. Since a 1978 ballot measure Proposition 13 sparked a national outcry for tax cuts and helped pave Gov. Ronald Reagans path to the White House, California has limited tax increases to 2% a year for inflation until a property is sold.

Supporters saidasplit-roll system would go a long way toward fixing inequities that shield wealthy corporations, depriving property tax proceeds for schools and local governments.

Opponents called it a massive tax increase that will cripple businesses in a pandemic-wracked economy. Their advertising portrayedit as a step toward completely dismantling the system established under 1978's Proposition 13, even though supporters disavowed plans to change how residential property is assessed.

A ballot measure to reinstate affirmative action in California government saw a 56.1% No vote Tuesday. Public polling had indicated that Proposition 16 was struggling, suggesting that voters were not inclinedto repeal a quarter-century-old ban on affirmative action in the state.

A national awakening on race drove a well-funded campaign to reinstate preferential treatment based on race and gender in public hiring, contracting and college admissions. Supporters said such programs are critical to undoing generations of systemic racism and sexism. Opponents said merit alone should determine whether someone gets a job or gets accepted into college.

Democratic vice presidential nominee Kamala Harris backedthe effort. Opponents saidthe government should treat every person equally, and never use race, ethnicity or gender to promote or discriminate against an individual.

The biggest margin of victory on the night belonged to No votes on Proposition 23, the second ballot measure on kidney dialysis in the state in as many elections. Voters rejected the ballot measure to require a doctor or highly trained nurse at each of Californias 600 dialysis clinics, with "no" votes taking64%.

The measure drew more than $110 million in spending. Supporters said a doctor is needed at clinics whenever the states 80,000 dialysis patients are being treated to make sure they get quality care. Opponents, financed by dialysis clinic companies, said the measure would have created a financial burden that could lead some clinics to close.

Proposition 23 was the second attempt by the unions to increase regulation of dialysis clinics in California, where DaVita Inc. and Fresenius Medical Care two of the countrys largest for-profit dialysis providers operate about three-quarters of the states dialysis market.

California voters also voted to maintain the status quo on criminal justice. They rejected an attempt to eliminate cash bail, with Proposition 25 losing with 55.5% of thevotes cast as No.

On Proposition 20, which would have scaled back two earlier ballot measures approved by voters in 2014 and 2016, about 62% of voters were opposed. The proposition would again bar those convicted of certain serious offenses from earlier release and increase penalties for repeated retail thefts.

By rejecting Proposition 25, voters in the most populous state overturned a 2018 law that stalled when the bail industry challenged it at the ballot box through the proposition.Supporters of the change had said the traditional bail system punishes the poor often racial minorities because they lack the money to buy their freedom or can least afford to pay a bail bondsman.Opponents included some prominent civil rights groups who said the alternatives risk assessment tools also are racially and socioeconomically biased.

A proposition that would keep alive Californias first-of-its-kind stem cell research program appears to have won in a tight race. Proposition 14 saw voters give it 51% Yes votes Tuesday. The measure would authorize a $5.5 billion bond sale to bail out the California Institute for Regenerative Medicine, which was created by a similar $3 billion bond measure in 2014 but is now nearly broke. With dozens of stem cell research trials underway, supporters say the money is desperately needed. Opponents saidthat in a pandemic-induced economic crisis, California simply cant afford it.

Voters rejected a measure that would have allowed cities to expand rent control. Proposition 21 would have let cities limit rent hikes on properties that are more than 15 years old. No votes led with 59.8% of the vote. Opponents argued that the measure would have discouraged new home construction at a time when its sorely needed. Proponents said the measure was an urgent attempt to slow spiraling rent increases. A recent report said more than half of Californias renters spend over 30% of their incomes on rent.

A measure to expand California's digital privacy law easily won with 56.1% of the voters approving Proposition 24. The measure would update a law approved two years ago that gave Californians the right to know what personal information companies collect about them online. The proposition also would triple the fines for companies that violate kids privacy.

Proponents saidthe measure will strengthen Californias privacy law and help hold big business accountable. Opponents argued that the 52-page initiative was too complicated for voters and its too soon to rewrite a law that just took effect.

California voters appeared to give narrow approval to Proposition 19, which would give Californians 55 or older a big property tax break when buying a new home. To fund that new tax break, the measure would curtail a separate tax break Californians may receive on homes inherited from parents and grandparents.

Yes on Prop. 19 was leading with 51.4% of the vote.

In one of two measures involving voting rights, voters decisively passed a ballot measure restoring the right to vote for felons on parole. Proposition 17 will change the state Constitution to allow an estimated 50,000 felons to vote, with 59% ofvoters approving the measure.

Another voting rights measure, Proposition 18, which would have allowed 17-year-olds to vote in primaries if they turn 18 before the general election, was rejected by55.% of voters.

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Homology Medicines Announces Upcoming Oral Presentation on pheNIX Gene Therapy Clinical Trial for Adults with PKU – GlobeNewswire

November 05, 2020 09:15 ET | Source: Homology Medicines, Inc.

Homology Webcast / Conference Call Scheduled for Friday, November 6 at 4:30 p.m. ET

BEDFORD, Mass., Nov. 05, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the upcoming oral presentation of data from the dose-escalation portion of the Companys ongoing Phase 1/2 pheNIX gene therapy clinical trial with HMI-102 for adults with phenylketonuria (PKU), the worlds first PKU gene therapy clinical trial. The data will be presented by pheNIX principal investigator Olaf Bodamer, M.D., Ph.D., FACMG, FAAP, Park Gerald Chair in Genetics & Genomics and Associate Chief of Genetics & Genomics at Boston Childrens Hospital, a premier center for metabolic disorders and the first site to enroll a patient in the pheNIX trial. The pheNIX trial results will be featured at the virtual New England Consortium of Metabolic Programs (NECMP) annual meeting, which is focused on new research in metabolic disorders, including PKU, on Friday, November 6. NECMP includes metabolic clinics, healthcare providers, patient organizations and others dedicated to increasing knowledge of metabolic disorders and improving delivery of healthcare to patients.

We are pleased that additional data from our pheNIX trial, the first-ever gene therapy trial for PKU, will be shared for the first time by a respected leader in the field of metabolic disorders and directly with the PKU community as part of a featured, peer-reviewed scientific session, stated Gabe Cohn, M.D., Chief Medical Officer of Homology Medicines. The oral presentation by Dr. Bodamer will include data from three dose cohorts in the dose-escalation phase of our pheNIX trial, the results of which are informing the upcoming expansion phase of our study.

Webcast/Conference Call The NECMP annual meeting is for members of the Consortium. Homology will host a conference call and webcast following the meeting on Friday, November 6 at 4:30 p.m. ET. The webcast will be accessible on Homologys website in the Investors section, and the webcast replay will be available on the website for 90 days following the presentation. To access using the conference call line, dial (866) 244-8091 (U.S./Canada toll-free) or (602) 563-8623, with Conference ID 7394503.

About HMI-102 HMI-102 is an investigational gene therapy in clinical development for the treatment of phenylketonuria (PKU) in adults. HMI-102 is designed to encode the PAH gene, which is mutated in people with PKU, delivered via the liver-tropic AAVHSC15 vector. Homology has received Fast Track Designation and orphan drug designation for HMI-102 from the U.S. Food and Drug Administration (FDA), and orphan drug designation from the European Medicines Agency (EMA).

About Phenylketonuria (PKU) PKU is a rare inborn error of metabolism caused by a mutation in thePAHgene. PKU results in a loss of function of the enzyme phenylalanine hydroxylase, which is responsible for the metabolism of phenylalanine (Phe), an amino acid obtained exclusively from the diet. If left untreated, toxic levels of Phe can accumulate in the blood and result in progressive and severe neurological impairment. Currently, there are no treatment options for PKU that target the underlying genetic cause of the disease. According to the National PKU Alliance, PKU affects nearly 16,500 people in the U.S. with approximately 350 newborns diagnosed each year. The worldwide prevalence of PKU is estimated to be 50,000 people.

About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations surrounding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; plans and timing for the release of clinical data from the Phase 1/2 pheNIX trial; our position as a leader in the development of genetic medicines; and our participation in upcoming presentations and conferences. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our business and operations, including our preclinical studies and clinical trials, and on general economic conditions; we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities of our manufacturing facility; risks relating to the regulatory approval process; interim, topline and preliminary data may change as more patient data become available, and are subject to audit and verification procedures that could result in material changes in the final data; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property and significant costs as a result of operating as a public company. These and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarterly period endedJune 30, 2020and our other filings with theSECcould cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

Company Contacts Theresa McNeely Chief Communications Officer and Patient Advocate tmcneely@homologymedicines.com 781-301-7277

Media Contact: Cara Mayfield Senior Director, Patient Advocacy and Corporate Communications cmayfield@homologymedicines.com 781-691-3510

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Homology Medicines Announces Upcoming Oral Presentation on pheNIX Gene Therapy Clinical Trial for Adults with PKU - GlobeNewswire

Homology Medicines Announces Presentation of Positive Data from the Dose-Escalation Phase of the pheNIX Gene Therapy Trial for Adults with PKU | DNA…

Details Category: DNA RNA and Cells Published on Friday, 06 November 2020 17:53 Hits: 882

- Marked Reductions in Phe Observed at Two Doses -

- Achieved Goal with Plans to Advance to Randomized, Concurrently Controlled Expansion Phase of Trial -

BEDFORD, MA, USA I November 06, 2020 I Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of positive data from the dose-escalation portion of the Phase 1/2 gene therapy pheNIX clinical trial for adults with phenylketonuria (PKU). The results showed that product candidate HMI-102 was generally well-tolerated, and resulted in marked reductions in phenylalanine (Phe) and the Phe-to-tyrosine (Tyr) ratio (Phe/Tyr ratio) at two doses. Phe is a registrable endpoint in PKU, and the Phe/Tyr ratio is a clinically relevant diagnostic measurement for PKU. With these positive results, Homology is progressing to the randomized, concurrently controlled expansion phase of the trial, which has the potential to be converted to a registrational trial.

The data were presented today in an oral presentation by Olaf Bodamer, M.D., Ph.D., FACMG, FAAP, Park Gerald Chair in Genetics & Genomics and Associate Chief of Genetics & Genomics at Boston Childrens Hospital, and principal investigator of the pheNIX trial, during the New England Consortium of Metabolic Programs (NECMP) annual meeting, which is focused on new research in metabolic disorders. NECMP includes metabolic clinics, healthcare providers, patient organizations and others dedicated to increasing knowledge of metabolic disorders and improving delivery of healthcare to patients.

This is the first-ever PKU gene therapy clinical trial, and I am excited to share these data with the PKU community as I believe they demonstrate the potential of HMI-102 to treat the underlying genetic cause and reduce the therapeutic burden for patients and their families, stated Dr. Bodamer. PKU is a challenging condition, and a treatment that establishes normal metabolism could change the prognosis for patients with this rare genetic disorder. We look forward to participating in the next phase of the study.

We are pleased to have met the goals of the dose-escalation portion of the trial, which were evaluation of safety and efficacy of a single I.V. administration of HMI-102 and dose determination for the expansion phase of the trial, stated Gabe Cohn, M.D., Chief Medical Officer of Homology Medicines. Even as many patients self-liberalized their diets, there were patients at the mid- and high-doses with plasma Phe values below 360 mol/L and/or 600 mol/L, and one of these patients achieved a Phe level within the normal range. This is the first time a genetic medicines approach has achieved these results in patients with PKU. We have learned a tremendous amount in the dose-escalation phase and are applying these learnings to the expansion phase of the trial, which we anticipate initiating in early 2021.

Dr. Cohn continued, We greatly appreciate the PKU community of patients, clinicians and caregivers who have participated in this first phase of the pheNIX trial, and we look forward to working together during the next phase.

As of the data cutoff date of October 19, 2020, six patients in the dose-escalation phase of the pheNIX trial had received gene therapy product candidate HMI-102 across three dose cohorts (low-dose Cohort 1, n=2; mid-dose Cohort 2, n=2; high-dose Cohort 3, n=2). Cohorts included males and females, with an age range of 21-49 and time in study ranging from 13 weeks to 52 weeks (end of study).

Safety Observations HMI-102 was generally well-tolerated, and there were no treatment-related serious adverse events (SAEs). There were no clinically significant changes in ECG or vital signs and no clinical signs of complement activation. The Grade 1 and 3* alanine aminotransferases (ALTs) observed in Cohorts 2 and 3, which is common in AAV-based gene therapy, were managed with increased steroids when necessary. The patients who experienced Grade 3 ALTs had pre-existing underlying immune conditions. An independent data monitoring committee, which provided guidance throughout the pheNIX trial, concluded that there were no safety concerns related to bilirubin, and that ALT elevations may be associated with reduced efficacy.

Updates to the expansion phase of the pheNIX trial, including key learnings related to patient selection, monitoring and steroid regimen, are being incorporated.

Efficacy Observations

Cohort 1 (Low-Dose) Through 52 weeks, patients in Cohort 1 continued to show no meaningful reductions in Phe.

Cohorts 2 and 3 (Mid- and High-Dose) The mean percent change from baseline in Phe observed in patients in Cohorts 2 and 3 were significant, compared to Cohort 1**. These Phe reductions occurred while patients self-liberalized their diets.

Through 48 weeks, one patient in Cohort 2 had Phe levels of <360 mol/L and/or <600 mol/L*** at multiple timepoints and had reached a minimum Phe level of 42 mol/L, compared with a baseline level of 1,010 mol/L. Through 13 weeks, one patient in Cohort 3 had a Phe level <360 mol/L and several Phe levels <600 mol/L at multiple timepoints and had reached a minimum Phe level of 303 mol/L, compared with a baseline level of 1,060 mol/L.

In Cohorts 2 and 3, Phe reductions were greater among patients with Grade 1 ALTs compared to patients with Grade 3 ALTs****; ALT elevations were managed with increased steroids when necessary. It appears higher ALT elevations may limit therapeutic activity, but can be managed with a modified steroid regimen, which is being incorporated into the expansion phase.

Expansion Phase Based on the safety and efficacy results observed in the dose-escalation phase, Homology is advancing to the randomized, concurrently controlled, dose expansion phase of the pheNIX trial, which has the potential to be converted to a registrational trial.

All cohorts in the dose-escalation phase showed an acceptable safety profile and certain patients in Cohorts 2 and 3 showed marked Phe reductions. Based on these collective data, Homology has selected two doses for the expansion phase: the mid-dose from Cohort 2 and a dose between the doses in Cohorts 2 and 3. The Company believes the latter dose has the potential to improve Phe reductions while reducing steroid exposure that was required at the high-dose. The Company believes that advancing two doses in parallel provides the potential to convert to a registrational trial quickly with the optimal dose as the expansion phase does not include staggered dosing between patients.

Webcast/Conference Call Homology management and Dr. Bodamer will host a conference call and webcast today, Friday, November 6 at 4:30 p.m. ET. The webcast will be accessible on Homologys website in the Investors section, and the webcast replay will be available on the website for 90 days following the presentation. To access using the conference call line, dial (866) 244-8091 (U.S./Canada toll-free) or (602) 563-8623, with Conference ID 7394503.

About HMI-102 HMI-102 is an investigational gene therapy in clinical development for the treatment of phenylketonuria (PKU) in adults. HMI-102 is designed to encode the PAH gene, which is mutated in people with PKU, delivered via the liver-tropic AAVHSC15 vector. Homology has received Fast Track Designation and orphan drug designation for HMI-102 from the U.S. Food and Drug Administration (FDA), and orphan drug designation from the European Medicines Agency (EMA).

About Phenylketonuria (PKU) PKU is a rare inborn error of metabolism caused by a mutation in thePAHgene. PKU results in a loss of function of the enzyme phenylalanine hydroxylase, which is responsible for the metabolism of phenylalanine (Phe), an amino acid obtained exclusively from the diet. If left untreated, toxic levels of Phe can accumulate in the blood and result in progressive and severe neurological impairment. Currently, there are no treatment options for PKU that target the underlying genetic cause of the disease. According to the National PKU Alliance, PKU affects nearly 16,500 people in the U.S. with approximately 350 newborns diagnosed each year. The worldwide prevalence of PKU is estimated to be 50,000 people.

About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

*ALT Grades based on Common Terminology Criteria for Adverse Events (CTCAE) Version 5 **P<0.004; Post-hoc comparison of Cohort 1 vs Cohorts 2&3 using repeated measures MANOVA/regression analysis ***U.S. and EU PKU treatment guidelines described in: Vockley J et al. Phenylalanine hydroxylase deficiency: diagnosis and management guideline. Genetics in Medicine 2014;16: 188-200. van Spronsen FJ et al. Key European guidelines for the diagnosis and management of patients with phenylketonuria. Lancet Diabetes Endocrinol 2017; 5: 74356. ****P<0.05; Post-hoc comparison of Patients 3&6 vs Patients 4&5 using repeated measures MANOVA/regression analysis

SOURCE: Homology Medicines

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Homology Medicines Announces Presentation of Positive Data from the Dose-Escalation Phase of the pheNIX Gene Therapy Trial for Adults with PKU | DNA...

COVID vaccine to be rolled out within months: Hunt – Daily Mercury

Health Minister Greg Hunt has announced Pfizer's groundbreaking COVID vaccine will be rolled out in Australia from March.

"I'm delighted to announce that the government has secured as part of its agreement with Pfizer, full cold chain logistics, distribution for the Pfizer vaccine.

It's what you call an mRNA vaccine, we have 10m units of that, part of a 134.8m unit, four-vaccine strategy We have secured that for Australia, well ahead of expectations and on schedule, to deliver vaccines to Australians, commencing in March 2021," he said.

"That is I think extremely important news. While, again, the advice is today that we may well have another zero community transmission case day for Australia, they are still waiting on two jurisdictions, we nevertheless have to be aware that we will not be out of this until we have a nation which has had a full vaccination program."

The Therapeutic Goods Administration has given the AstraZeneca and Pfizer vaccines the green light after "very promising" data from early clinical trial phases.

This puts both developers on track for approval in January and for Australians to be vaccinated against COVID-19 from March 2021.

"It essentially expedites the process and brings critical medicines, or vaccines, at a faster rate then would otherwise be the case but with an absolute premium on safety," Mr Hunt said.

The Therapeutic Goods Administration is expected to receive more clinical information needed to approve the vaccines around December.

The news comes as the Morrison government confirmed it would be able to distribute Pfizer's world-first messenger ribonucleic acid (mRNA) type vaccine, which needs to be kept at minus 70C.

Very "sophisticated eskies", which require dry ice that lasts for 14 days, would be used to hold and transport the vaccines, Therapeutic Goods Administration deputy secretary John Skerritt said.

"(The eskies) can be refilled twice without the need to connect to electricity," Professor Skerritt said.

"These eskies with the two refills gives you a month-and-a-half of cold chain protection."

The distribution process that goes to the National Cabinet on Friday includes giving vaccines to hospitals, respiratory clinics and general practices.

Professor Skerritt said the eskies would speed up the rollout of the vaccines because they allowed for multiple distribution points.

However, he said the jabs would not be approved until experts were confident they met the requirements of efficacy and safety.

WHO WILL GET THE COVID VACCINE:

The first batch of Pfizer's successful COVID-19 vaccine will reach just a fifth of the Australian population, starting with frontline health workers and the elderly.

These five million Australians will receive two doses of the drug - using all ten million rations the government has secured - and we can't manufacture more doses here.

The roll out of the vaccine, in the first three to six months of 2021, will also be difficult because it has to be kept at an extremely low temperature - minus 70 degrees - to remain stable.

There should be just enough doses for the 609,000 practising frontline health workers including doctors, nurses, dentists, pharmacists, 3.8 million people aged over 65 and 250,000 aged care workers.

Prime Minister Scott Morrison said the Pfizer trial results were "very promising and I'm optimistic and hopeful next year about the rollout of those vaccine programs".

Like much of the world, Australia's sharemarket responded positively to the vaccine news.

The key S&P/ASX 200 index closed 42 points higher at 6341, a 0.7 per cent rise for the day, after surging 2.2 per cent in morning trade.

Health Minister Greg Hunt told News Corp Pfizer had committed to providing the cold chain delivery equipment to distribute the vaccine in Australia.

Experts warn the vaccine won't mean the end of social distancing, handwashing and mask wearing until almost every Australian has received it.

CSIRO's Director of biosecurity Dr Rob Grenfell said while the vaccine would prevent people getting sick but it would not "prevent you getting colonised by the virus".

To eliminate the virus entirely, vaccines would have to be developed to stop the virus in the nasal cavity. The Pfizer vaccine does not do this.

Successful trials by Australia's Doherty Institute of delivering the Oxford AstraZeneca vaccine into the noses of ferrets have led to a human trial of this method in the UK.

"If that develops an immunity at the surface of the nasal cells and it's demonstrated to have nasal sterility, which is really the endpoint you're looking for, that would then go a long way towards decreasing the spread of the virus," Dr Grenfell said.

Conventional vaccines use a weakened form of the virus to prompt an immune response but mRNA vaccines like the one made by Pfizer use the virus's genetic code to make a person's own cells produce vaccine antigens and generate immunity.

This new age vaccine technology has never been used in humans before.

Nucleus Network's Paul Griffin has trialled a number of mRNA flu vaccines in humans with no serious side effects.

"It's not gene therapy, it is not able to be incorporated into the host genome so there's absolutely no prospect of altering human DNA using the vaccine," he said.

While Australia's vaccine manufacturer CSL is producing two other COVID-19 vaccine candidates it does not have the equipment needed to make Pfizer's mRNA vaccine and we will be dependent on receiving our supply of the vaccine from overseas manufacturers.

Mr Hunt told News Corp the government was considering over the longer term setting up an advanced pharmaceutical manufacturing plant in Australia that could produce this new type of vaccine.

While pharmaceutical giant Pfizer and BioNTech announced clinical trials of their COVID-19 vaccine were 90 per cent effective, the study was yet to be published or peer reviewed.

Pfizer said it would produce 50 million doses of its vaccine by the end of the year and 1.3 billion doses in 2021 but this would be enough to vaccinate less than one per cent of the world's population.

There are over 200 vaccines in development and more than 40 are in clinical trials with several key trials due to report this month.

The Australian Government has deals to buy a total of 134 million vaccine doses made by Oxford AstraZeneca, University of Queensland, Novavax and Pfizer.

The Australian sharemarket rise was more subdued than offshore markets where key share indices in the US, Britain and Europe soared between 3 and 7 per cent.

Among local stocks were some big movers, with Corporate Travel Management climbing 16 per cent and Flight Centre adding more than 8 per cent.

Investment platform eToro's market analyst, Adam Vettese, said the vaccine news had "injected optimism into travel stocks in particular".

"However, while this is obviously a positive step forward there is still a way to go," he said.

JBS Financial Strategists CEO Jenny Brown said markets were forward-looking and buyers were betting on a return to international travel.

"There's a lot of positivity but it has pulled back a bit," she said.

MORE NEWS

Pfizer COVID-19 vaccine explained: How it will affect you

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Originally published as The Aussies who will get Pfizer's COVID-19 vaccine

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COVID vaccine to be rolled out within months: Hunt - Daily Mercury

Autologous Stem Cell Based Therapies Market 2020 Emerging Trend and Advancement – News by aeresearch

The recent study on the Autologous Stem Cell Based Therapies market offers a competitive advantage to organizations operating in this industry vertical through a comprehensive assessment of the present and future growth prospects.

The report explicates important facets such as primary growth catalysts, and opportunities that will ensure the revenue flow in the coming years. Further, it lists the challenges and limitations along with solutions to overcome them. Insights germane to the market share and growth rate estimates of the industry segments are also provided as well.

Apart from this, the study delves into the business scenario across the various regional markets and profiles the companies that have reigned in these geographies. Further, it highlights the prevalent strategies adopted by leading companies while simultaneously suggesting changes and new tactics for adapting to the uncertainties brought in by the Covid-19 pandemic.

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Key pointers from the TOC of the Autologous Stem Cell Based Therapies market report:

Product gamut

Application scope

Regional outlook

Competitive landscape

In conclusion, the study systematically investigates the Autologous Stem Cell Based Therapies market through various segments to provide a broad view of this business sphere. In addition, it expounds the supply chain in terms of distributors, downstream consumers, and upstream material and equipment traders in this industry.

Reasons to access this Report:

The key questions answered in this report:

Significant Point Mentioned in theResearch report:

Table of Contents for market shares by application, research objectives, market sections by type and forecast years considered:

Autologous Stem Cell Based Therapies Market Share by Key Players: Here, capital, revenue, and price analysis by the business are included along with other sections such as development plans, areas served, products offered by key players, alliance and acquisition and headquarters distribution.

Global Growth Trends: Industry trends, the growth rate of major producers, and production analysis are the segments included in this chapter.

Market Size by Application: This segment includes Autologous Stem Cell Based Therapies market consumption analysis by application.

Autologous Stem Cell Based Therapies market Size by Type: It includes analysis of value, product utility, market percentage, and production market share by type.

Profiles of Manufacturers: Here, commanding players of the global Autologous Stem Cell Based Therapies market are studied based on sales area, key products, gross margin, revenue, price, and production.

Autologous Stem Cell Based Therapies Market Value Chain and Sales Channel Analysis: It includes customer, distributor, market value chain, and sales channel analysis.

Market Forecast: This section is focused on production and production value forecast, key producers forecast by type, application, and regions

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Autologous Stem Cell Based Therapies Market 2020 Emerging Trend and Advancement - News by aeresearch

Platelet Rich Plasma in Patients with Partial Thickness Rotator Cuff Tears or Tendinopathy Leads to Significantly Improved Short-Term Pain Relief and…

Purpose: The purpose of this study was to perform a randomized controlled trial, comparing platelet-rich plasma (PRP) to standard corticosteroid (CS) in providing pain relief and improved function in patients with rotator cuff tendinopathy and partial thickness rotator cuff tears (PTRCT).

Methods:This double-blind randomized-controlled trial enrolled patients with ultrasound or MRI proven PTRCT to either an ultrasound-guided PRP or CS injection. Patients completed patient reported outcomes at baseline, 6 weeks, 3 and 12 months post-injection. The primary outcome was improvement in visual analog scale for pain (VAS). Secondary outcomes included change in American Shoulder Elbow Surgeons (ASES) and Western Ontario Rotator Cuff index (WORC) scores. Failure of treatment was defined by subsequent injection, consent to surgery or operative intervention.

Results:99 patients (47 in the PRP and 52 in the CS groups) were followed to 12 months post-injection. There were no differences in baseline patient demographics including age, sex or duration of symptoms. Despite randomization, patients in the PRP group had worse baseline VAS (46.0 vs. 34.7, p=0.01), ASES (53.9 vs. 61.8, p=0.02) and WORC (42.2 vs. 49.5, p=0.03) scores. At 3 months post-injection, the PRP group had superior improvement in VAS (-13.6 vs. 0.4, p=0.03), ASES (13.0 vs. 2.9, p=0.02) and WORC (16.8 vs. 5.8, p=0.03) scores. There were no differences in patient reported outcomes at 6 weeks or 12 months. There was no difference in failure rates (p=0.31) or conversion to surgery (p=0.83) between groups.

Conclusions:Patients with PTRCTs or tendinopathy experienced clinical improvement in pain and patient-reported outcome scores after both ultrasound-guided CS and PRP injections. Patients who received PRP obtained superior improvement in pain and function at short-term follow up (3 months). There was no sustained benefit of PRP over CS at longer-term follow-up (12 months).

Level of evidence:Level 1, Randomized controlled trial.

Continued here:
Platelet Rich Plasma in Patients with Partial Thickness Rotator Cuff Tears or Tendinopathy Leads to Significantly Improved Short-Term Pain Relief and...

Platelet Rich Plasma Market Outlook 2020 Scope and Business Growth Overview by Regions, Latest Trends, and Opportunities Forecast to 2024 – The Daily…

Octapharma AG

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Key Market Trends:

Pure PRP Segment by Type is Expected to Hold the Largest Market Share

The pure PRP segment of the global platelet rich plasma market is believed to have the largest market share.

The prime factor responsible for the growth of this segment is the significance of this type of platelet plasma for the person. Pure PRP has an edge over traditional PRP, as it requires a two-step concentration process that helps in eliminating unwanted red blood cells (RBCs) and neutrophils. RBCs (that have no therapeutic effects for regeneration) can create a more viscous solution that can be more painful when injected. Neutrophils, a type of white blood cell, have inflammatory components that may increase pain and inflammation post-treatment.

Pure PRP helps the stem cells and regenerative cells in the repair and in rebuilding the damaged tissue. This ultimately speeds up the healing process and reduces pain. In addition, it promotes increased strength and improves the overall function. Therefore, owing to the contribution of pure PRP in the healing process and the rising use of it as a blood product, the segment is expected to dominate the market in the coming future.

North America Dominates the Market and is Expected to Continue the Same Trend for Next Few Years

North America currently dominates the platelet-rich plasma market and is expected to continue its stronghold for a few more years. The United States is a major market, and this is mainly due to the US governments initiatives to develop blood products. In addition, the emergence and adoption of novel technologies are going to help the market in a positive manner.

Competitive Landscape:

Most of the companies present in the market are efficient at the technological front, but require significant support for enhancing their services and expanding their businesses. Thus, mergers and acquisitions offer significant opportunities to gain the attention of a large number of providers across developed regions.

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Platelet Rich Plasma Market Covers Following Points in TOC:

1 INTRODUCTION 1.1 Study Deliverables 1.2 Study Assumptions 1.3 Scope of the Study

2 RESEARCH METHODOLOGY

3 EXECUTIVE SUMMARY

4 MARKET DYNAMICS 4.1 Market Overview 4.2 Market Drivers 4.2.1 Rising Incidences of Sports Injuries 4.2.2 Increasing Number of Androgenic Alopecia Patients 4.2.3 Growing Use of Platelet-rich Plasma in Various Therapeutic Areas 4.2.4 Rising Prevalence of Arthritis 4.3 Market Restraints 4.3.1 Stringent Regulatory Policies 4.3.2 High Prices of Plasma Therapy 4.4 Porters Five Forces Analysis 4.4.1 Threat of New Entrants 4.4.2 Bargaining Power of Buyers/Consumers 4.4.3 Bargaining Power of Suppliers 4.4.4 Threat of Substitute Products 4.4.5 Intensity of Competitive Rivalry

5 MARKET SEGMENTATION 5.1 By Type 5.1.1 Pure PRP 5.1.2 Leukocyte-rich PRP 5.1.3 Pure Platelet-rich Fibrin 5.1.4 Leukocyte-rich Fibrin 5.2 By Source 5.2.1 Autologous 5.2.2 Allogenic 5.3 By Application 5.3.1 Orthopedic 5.3.1.1 Arthritis 5.3.1.2 Chronic Tendinitis 5.3.1.3 Bone Repair and Regeneration 5.3.2 Dermatology 5.3.2.1 Androgenic Alopecia 5.3.2.2 Plastic Surgery 5.3.2.3 Cardiac Muscle Injury 5.3.2.4 Dental 5.3.2.5 Nerve Injury 5.3.2.6 Other Applications 5.3.3 By End User 5.3.3.1 Hospitals and Clinics 5.3.3.2 Research Institutes 5.3.3.3 Other End Users 5.4 Geography 5.4.1 North America 5.4.1.1 United States 5.4.1.2 Canada 5.4.1.3 Mexico 5.4.2 Europe 5.4.2.1 Germany 5.4.2.2 United Kingdom 5.4.2.3 France 5.4.2.4 Italy 5.4.2.5 Spain 5.4.2.6 Rest of Europe 5.4.3 Asia-Pacific 5.4.3.1 China 5.4.3.2 Japan 5.4.3.3 India 5.4.3.4 Australia 5.4.3.5 South Korea 5.4.3.6 Rest of Asia-Pacific 5.4.4 Middle East & Africa 5.4.4.1 GCC 5.4.4.2 South Africa 5.4.4.3 Rest of Middle East & Africa 5.4.5 South America 5.4.5.1 Brazil 5.4.5.2 Argentina 5.4.5.3 Rest of South America

6 COMPETITIVE LANDSCAPE 6.1 Company Profiles 6.1.1 Bio Product Laboratory Ltd (BPL) 6.1.2 Biolife Plasma Services 6.1.3 Biotest AG 6.1.4 Cambryn Biologics LLC 6.1.5 China Biologic Products Inc. 6.1.6 CSL Ltd 6.1.7 Grifols International SA 6.1.8 Kedrion SpA 6.1.9 LFB SA 6.1.10 Octapharma AG

7 MARKET OPPORTUNITIES AND FUTURE TRENDS

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Platelet Rich Plasma Market Outlook 2020 Scope and Business Growth Overview by Regions, Latest Trends, and Opportunities Forecast to 2024 - The Daily...

Platelet-rich Plasma Market To Witness the Highest Growth Globally in Coming Years 2020-2025 – The Daily Philadelphian

The Platelet-rich Plasma Market Research Report 2020 to 2025 presents an in-depth assessment of the Station Platelet-rich Plasma It provides the industry overview with market growth analysis with a historical & futuristic perspective considering the following parameters; cost, revenue, demands, and supply data (as applicable). The bottom-up approach was used to estimate the global market size of Station Platelet-rich Plasma based on end-use applications in the various regions, in terms of USD million. The report covers the historic growth of the market, the impact of the COVID-19, and also forecasts its recovery post-COVID-19. The report also presents forecasts for Platelet-rich Plasma investments from 2020 to 2025.

Top Companies in the Global Platelet-rich Plasma Market:Arthrex, Inc., Cambryn Biologics LLC, China Biologic Products Inc., DePuy Synthes, Dr. PRP USA LLC, EmCyte Corporation, Glofinn Oy, T-Biotechnology, Terumo BCT, Inc., and Zimmer Biomet

The platelet-rich plasma (PRP) market is expected to register a CAGR of approximately 14.2%, over the forecast period.

Market OverviewThe key factors that are augmenting the growth of the market include rising incidences of sports injuries, an increasing number of cosmetic and surgical procedures, and growing use of platelet-rich plasma in various therapeutic areas. PRP contains at least seven growth factors, and it can be used for various treatments, such as chronic tendon injuries, acute ligament, and muscle injuries, surgery, knee arthritis, fractures, and pain. In orthopedics, the use of PRP is expected to increase over the period. PRP is increasingly being used in the treatment of acute sports injuries, such as muscle injuries. The popularity of PRP is on the rise, but its true effectiveness is yet to be fully established. The PRP is thus widely used in surgery to repair torn knee ligaments, such as the anterior cruciate ligament (ACL). The PRP facilitates the fast healing of the tissue after surgical procedures. Several research studies are currently being conducted to evaluate the effectiveness of PRP in other treatment areas, such as in the case of herniated disc and hair loss. The use of PRP has grown widely in different therapeutic areas, owing to the increasing availability and ability to enable faster healing.

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Market InsightsThe Orthopedics Segment by Application is Expected to Hold the Largest Market Share According to the US Arthritis Foundation, arthritis is the leading cause of disability among adults in the country. By conservative estimates, about 54 million adults were diagnosed with arthritis and almost 300,000 children were suffering from arthritis or rheumatic condition (2017-2018). Despite not being considered the standard practice, a growing number of people are opting for PRP injections, to treat an expanding list of orthopedic conditions, including osteoarthritis. It is most commonly used for knee osteoarthritis but may be used on other joints as well. While treating osteoarthritis with platelet-rich plasma, a doctor injects PRP directly into the affected joint, in order to reduce pain, improve joint function, and slow, halt, or even repair damage to the cartilage. In such cases, platelet-rich plasma is derived from a sample of the patients own blood. Platelet-rich plasma therapy is thus witnessing a significant rise in its popularity and acceptance, for treating arthritis, owing to the fact that it inhibits inflammation, reduces the rate of progression of osteoarthritis, stimulates the formation of new cartilage, and increases the production of natural lubricating joint fluid, thus, easing painful joint friction, and it also contains proteins that alter a patients pain receptors and reduces pain sensation. Hence, the aforementioned factors are expected to fuel the growth of the arthritis segment.

North America Dominates the Market and is Expected to Depict Similar Trend Over the Forecast Period The market growth in the country can be attributed to several factors, including the rise in the prevalence of several disorders, such as arthritis, well-established healthcare infrastructure, better regulatory framework, and government support. As per a survey conducted by the Centers for Disease Control and Prevention (CDC), in the United States, in 2018, about 54.4 million adults (22.7%) were diagnosed with common arthritis, rheumatoid arthritis, and gout. Moreover, in the country, due to the growing aging population, the prevalence of doctor-diagnosed arthritis is expected to rise significantly, over the forecast period. Osteoarthritis is the most prevalent type of arthritis, and it affects about 31 million Americans every year.

Regions Are covered By Platelet-rich Plasma Market Report 2020 To 2025.

North America, Europe, China, Japan, Southeast Asia, India. North America (USA, Canada, and Mexico). Europe (Germany, France, UK, Russia, and Italy). Asia-Pacific (China, Japan, Korea, India, and Southeast Asia).

The report has 150 tables and figures browse the report description and TOChttps://www.marketinsightsreports.com/reports/10192354530/platelet-rich-plasma-market-growth-trends-and-forecasts-2020-2025?Mode=46

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-Detailed overview of Platelet-rich Plasma Market -Changing the Platelet-rich Plasma market dynamics of the industry -In-depth market segmentation by Type, Application, etc. -Historical, current and projected Platelet-rich Plasma market size in terms of volume and value Recent industry trends and developments -Competitive landscape of Platelet-rich Plasma Market -Strategies of key players and product offerings -Potential and niche segments/regions exhibiting promising growth.

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Platelet-rich Plasma Market To Witness the Highest Growth Globally in Coming Years 2020-2025 - The Daily Philadelphian