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WATCH: Gov. Ron DeSantis to give State of the State Address – WMBB – mypanhandle.com

Posted: March 5, 2021 at 5:01 am

Gov. Ron DeSantis gave his State of the State address Tuesday to formally start the 2021 legislative session. Here are his remarks, as prepared for delivery:

Mr. Speaker, Mr. President, members of the House and Senate and fellow citizens:

I see, in many parts of our country, a sad state of affairs: schools closed, businesses shuttered and lives destroyed.

This calamitous reality is just the beginning of what will likely be long-term damage to children, families and society.

Sow the wind, reap the whirlwind.

While so many other states kept locking people down, Florida lifted people up.

Floridas schools are open and we are one of only a handful of states in which every parent has a right to send a child to school in-person.

All Floridians have a right to earn a living and our citizens are employed at higher rates than those in the nation as a whole.

Every job is essential.

If you are working hard to earn a living, we got your back in the state of Florida.

Every business in Florida has a right to operate. We have stood up for small, family-owned businesses and have saved thousands of them from ruin.

Because of our actions, Florida is leading the nation in the number of people submitting business formation applications and we are one of the top destinations for business relocation.

Friends, legislators, Floridians, lend me your ears: We will not let anybody close your schools, we will not let anybody take your jobs and we will not let anybody close your businesses!

One year ago, COVID-19 had not yet been declared a global pandemic. We had scant knowledge of the virus, little ability to test for it, and no approved treatments.

Today, we have three safe and effective vaccines, great treatments like monoclonal antibodies, and the ability to conduct rapid diagnostic tests.

A lot has happened over the past year.

We are saddened by the thousands of Floridians and hundreds of thousands of Americans who have died with COVID. And we sympathize with the family members who in many instances were not even permitted to see their loved ones in person, at a nursing home or in the hospital.

To honor those who have died with COVID and to recognize the toll the virus has taken on family members, the state of Florida will be lowering the flags to half-staff on Wednesday.

We thank the health care professionals throughout Florida who cared for those who became ill due to COVID. This includes front-line doctors and nurses, who provided top-notch care to hospitalized patients, as well as staff at long-term care facilities who worked tirelessly to protect our most vulnerable seniors.

Their efforts helped to save thousands of lives and are a major reason why Florida, with perhaps the most vulnerable population to COVID, has per capita COVID mortality that is below the national average.

From the outset, Florida has been steadfast in focusing efforts on the protection of the elderly. We rejected the policy of sending contagious COVID patients back into nursing homes; indeed, we prohibited the practice. Florida also established COVID-only nursing facilities so that infections in long-term care facilities could be more effectively contained.

And perhaps most importantly, we are prioritizing our senior citizens for vaccinations. Florida is putting seniors first because it is the best strategy to save lives and is the best way to honor our elders from whom we draw inspiration. We have vaccinated millions of seniors throughout Florida parents, grandparents, veterans of the second world war, survivors of the Holocaust.

We have made vaccinations available all across the state: in retail pharmacies including Publix, Walmart and CVS, at hundreds of hospitals throughout Florida, at drive-thru sites in places like The Villages retirement community, at houses of worship from the First Baptist Church Piney Grove in Lauderdale Lakes to the St. Paul AME in Jacksonville, at senior communities such as Century Village and Kings Point in South Florida, and in underserved areas such as Pahokee.

We even have delivered thousands of shots to homebound seniors in the comfort of their own homes.

Our efforts saved lives. In fact, 40 states have suffered higher COVID mortality for seniors aged 65-plus on a per-capita basis than Florida.

And the cases and hospitalizations for seniors in Florida have plummeted as vaccinations have increased since Jan. 30, the number of seniors hospitalized for COVID has declined by 80% and cases among seniors have declined by 71%.

Florida was right to prioritize the elderly. Seniors first works.

As we worked to protect seniors, we also worked to give opportunities to our kids. Florida has led the way in providing all parents the right to send their kids to school for in-person instruction. Florida is one of only four states and the only large state to offer in-person instruction to 100% of its students.

Across the nation, millions of students have been locked out of the classroom for nearly a year and for many there is no end in sight. These students have fallen behind on academics, have been denied the opportunity to participate in activities such as athletics, and have seen their social development stunted.

The consequences of shutting kids out of school for a year, year-and-a-half and maybe even, in some places, two years, will be catastrophic and long-lasting.

The failure of so many places outside of Florida to open schools at the beginning of the school year will go down as one of the biggest policy blunders of our time.

Florida did not make that mistake. We followed the data and stood by our parents and students. We ignored the political posturing and fear-mongering and did what was right for Floridians.

Florida has succeeded where so many other states have failed in providing opportunities for its students in large measure because of the tireless efforts of school superintendents, administrators, teachers and coaches. They knew keeping kids out of school would be a disaster and were not going to let that happen on their watch.

On behalf of a grateful state and millions of grateful parents, thank you.

Given the unique circumstances we faced, you may never have an opportunity to play such a crucial role in ensuring opportunities for students as well as in preventing long-term damage to society.

Open schools have been a godsend to parents throughout Florida, especially for single moms responsible for putting food on the table.

Economic lockdowns are a luxury of the largely affluent Zoom class; many Floridians cannot do their jobs over a computer; they need to show up.

Over the past many months, Florida has led the way in protecting the jobs and livelihoods of its hard-working citizens from construction workers to bartenders, from servers to hair stylists, everyone has a right to earn a living.

Floridas efforts have made a lasting impact on the lives of millions of people. But do not take it from me. Let them tell you for themselves:

There are not a whole lot of Floridians who are itching to move from Florida to lockdown states, but there are thousands and thousands of people who are seeking to leave the lockdowns behind for the greener pastures in Florida.

We have long been known as the Sunshine State but, given the unprecedented lockdowns we have witnessed in other states, I think the Florida sun now serves as a beacon of light to those who yearn for freedom.

As we begin this legislative session, I look forward to working with Senate President Wilton Simpson and House Speaker Chris Sprowls. You both have already demonstrated leadership on issues that matter to Floridians and I know you will be great partners for progress.

When the initial fallout from COVID began, there was a lot of concern about whether we could afford to continue with the progress we have already made on priorities like protecting our water resources and K-12 education.

Forecasts were dire. The 2021 legislative session was shaping up to be a fiscal nightmare.

I am pleased to report that our current fiscal outlook is much better than the bleak forecasts from last spring.

As many of you are probably aware, when the pandemic hit, I vetoed $1 billion from this years budget. I also instructed our executive agencies not to spend all of the appropriated funds because we did not know for sure how much revenue we would be taking in.

Because Floridas economy is open, revenue is coming in at levels far higher than even the most recent revised estimates. For the last three months December to February preliminary estimates peg the increase in revenue at more than $800 million over and above the December revenue estimation.

Florida is below the national average in unemployment and much lower than our peer states of Texas, New York and California. We also anticipate downward revisions of Decembers unemployment numbers to reflect even stronger jobs numbers.

As international travel is reinstated and tourism picks up (and I hope the federal government will allow our cruise ships to sail again), the employment outlook should improve even more.

Throughout the pandemic, Florida has not touched one red cent from our rainy day fund.

The bottom line is that we saved Floridas economy and as a result our budget outlook is positive.

The priorities weve championed from water resources to education to infrastructure can be honored.

Let us get it done.

I reject reductions in funding for K-12 education. Last session, the Legislature answered my call to increase the average minimum salary for teachers, taking Florida from the bottom half of states to the top 5. Let us keep this momentum going let us do more this year!

We should not forget that Florida continues to make great strides in K-12 education.

Just last week, the College Board released data showing that Florida ranks No. 2 in the nation in the percentage of graduating seniors who have passed Advanced Placement exams.

Florida leads the nation in school choice.

We are beginning to place a strong and long overdue emphasis on vocational education.

Florida has launched an ambitious civics initiative so that students can understand the principles that make our country unique.

Florida is leading on education and we must continue to do so.

We also must continue to protect our natural resources and invest in improvements in water quality. I ask that you continue to fund the key projects from the EAA reservoir in the south to the projects in the northern Everglades that will impact our state for generations to come, and reaffirms our commitment to Everglades restoration and access to safe, clean water for our communities.

I am also proposing the creation of the Resilient Florida program under the Department of Environmental Protection. Through this initiative, Florida will invest $1 billion into projects that help our communities adapt to the threats posed by flooding from intensified storms and sea level rise. I am encouraged by similar proposals from the Legislature to address this important issue for our communities, and I look forward to signing into law a program that will make a difference.

Florida is and must remain a state dedicated to law and order. When riots broke out across the nation last year, we saw cities ruined by violent mobs. Law enforcement was targeted and lawlessness prevailed. This was not and must never be tolerated in the state of Florida.

As we saw rioting in other states last year, I called up the National Guard, mobilized mobile field force teams from the FHP, and worked with local officials like Carlos Gimenez and Lenny Curry to ensure that places like Miami and Jacksonville did not suffer the same fate as Minneapolis and Kenosha. Florida handled it well. But we need to do more.

Working with President Simpson, Speaker Sprowls and law enforcement groups across the state, we have proposed the strongest anti-rioting, pro-law enforcement reforms in the nation.

We will not permit localities to jeopardize the safety of their citizens by indulging in the insane fantasy of defunding law enforcement.

We will not allow our cities to burn and violence to rule the streets.

And we will not leave any doubt in the minds of those who wear the uniform that the state of Florida stands with you.

To paraphrase an old Merle Haggard song, when you mess with the men and women of law enforcement you are walking on the fightin side of me.

Speaker Sprowls and Senator Simpson have also been leaders in supporting legislative reforms to protect Floridians from the power of Big Tech.

This is real-life 2021, not fictitious 1984, yet Big Tech wields monopolistic power over the public in ways that would have made the monopolists of the early 20th Century blush.

Floridians have a right to control their personal data and Big Tech should not be able to make billions of dollars off us without our informed consent.

Florida has always been a state that strongly supports free speech, and we cannot allow the contours of acceptable speech to be adjudicated by the whims of oligarchs in Silicon Valley.

Nor we can allow Floridians to be de-platformed or silenced with no means of recourse, and this is especially true of those who rely on these technology platforms for their livelihoods.

Finally, because Florida is dedicated to free and fair elections, we cannot allow Big Tech to interfere in our elections by putting a thumb on the scale for political candidates favored by Silicon Valley.

Speaking of elections, we should take a moment to enjoy the fact that Florida ran perhaps the most transparent and efficient election in the nation in 2020. People actually asked, why cannot these other states be like Florida? Such a sentiment would have been unthinkable 20 years ago.

We need to make sure our elections are transparent and run efficiently. There should be no ballot harvesting in the state of Florida. One person, one vote.

We also cannot allow private groups to pour millions of dollars into the administration of our elections. That is a public function and should be done free from this type of private interference.

Let us stay ahead of the curve on election administration we never want to see the chaos of 20 years ago rear its head in Florida again.

I know these issues are merely scratching the surface of what the House and Senate will tackle this session. Of course, I would be remiss if I did not lend my support to the COVID liability bills for business and health care; for the Speakers bills cracking down on the Chinese Communist Party and other foreign influence; general reforms to improve the states legal climate; reform of the emergency powers of local government; and continued support for infrastructure.

I have no doubt that you will send me a lot more than that.

At the close of the constitutional convention the famed elder statesman, Benjamin Franklin, was asked to be the first to sign the new Constitution. Franklin pointed to General Washingtons chair, the back of which had the design of a sun low on the horizon. There were days, Franklin remarked, when I thought this picture of a sun low on the horizon was a setting sun, but I now know its a rising sun a new day for America, a new dawn for freedom.

Our nation and our state have endured a tumultuous year. Floridians have responded in ways that would make our founders proud.

Because of those efforts, the sun is rising here in Florida and the Sunshine State will soon reach new horizons.

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WATCH: Gov. Ron DeSantis to give State of the State Address - WMBB - mypanhandle.com

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30th anniversary of Penguins-Whalers trade: Why would Hartford ever give away Ron Francis? – TribLIVE

Posted: at 5:01 am

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As a Pittsburgh sports fan growing up in Connecticut, I wasnt sure how to feel about the Penguins trade with the Hartford Whalers in March of 1991.

I knew the players that the Penguins were getting were good and filled a need in Pittsburgh.

But, man, John Cullen was fifth in the league in scoring! And what if Zarley Zalapski honed all those offensive tools and became more physical and defensively stout?

My doubts were erased, though, when I got on the school bus while wearing a Penguins cap.

You guys stole Ron Francis and Ulf, grumbled the Whalers fan. You (expletive).

I stammered back. Yeah, but you guys got John Cullen and Zarl,

Go (expletive), he snarled.

Well, Im pretty sure if I did that on the bus, Id get suspended. But I let it go because the kid was older and bigger than me (which isnt saying much).

Regardless, the Whalers fan version of Scott Farkas was onto something.

Thats where we begin our second installment of looking back at The Trade of 1991 as Francis recalls the chain of events that eventually led to the Penguins big steal at the trade deadline. And the teams first Stanley Cup.

The explanation surrounding why the Penguins made the trade that they did on the night of March 4, 1991 is pretty clear. They had offense to spare. And they needed to get more defensive responsibility and toughness.

So you get a two-way specialist in Selke Trophy candidate Ron Francis to replace the offensively oriented John Cullen as a second-line center complement to Mario Lemieux.

You give up a young puck-moving defensemanwhich Hartford neededin Zarley Zalapski and get the nasty, net-front defensive presence of Ulf Samuelsson.

You give up the size, grit and enforcement up front in minor league prospect Jeff Parker. And you get an NHL-ready version on the blue line instead with mass and reach in Grant Jennings.

Got it.

The dynamics at play in Hartford were more complex, though. Why would they ever be willing to get rid of a 28-year-old Francis, who left the team as the franchises career leader with 264 goals, 557 assists and 821 points in 714 games?

He was team captain. He appeared to be well-liked by the other players. Almost every Whalers fan seemed to love him.

But what Francis didnt have was a contract beyond the end of the year. Nor did he have a good relationship with coach Rick Ley, who had taken the C off his chest and had given it to Pat Verbeek. Something that Jennings would describe as a gut punch for Francis.

Rocky relationships with Ley werent restricted to Francis.

Rick Ley had some different kinds of relationships with different players. Thats a nice way of putting it, Jennings said. He was going off on a lot of players. Having personal meetings with guys like myself and Ronny. Lots of people. I could sense I was on the block for sure.

But I had no idea they were making the deal they did.

As the Hartford Courant described things the day after the trade, Francis contentious contract negotiations as he was playing out his $370,000 option year were very much an undercurrent to the process.

The paper quoted the centerman as saying that Whalers general manager (and former Penguins GM) Eddie Johnston contacted him a few weeks before the deadline in an attempt to reopen negotiations.

Francis thought that meant the Whalers genuinely wanted to keep him, as they insisted that signing him after the trade deadline would be no problem.

In retrospect, they were trying to do a lot of things to get me to say, I want out. Which I didnt, Francis told me during Wednesdays Breakfast With Benz podcast. Then they called a couple of weeks before the deadline and said they were happy with me and wanted to sign me. So I said great, well talk after the deadline.

And then they traded me. Interesting times.

Francis admitted during our conversation that things were not great between him and Johnston during that last year in Hartford. Which, for most people who have met either person, is almost impossible to fathom. If you show me one person who has had a bad interaction with Francis, thatd be exactly one more person who has ever told me theyve had a bad experience with Johnston.

And vice versa.

Before ever meeting either Johnston or Francis, I heard lots of words to describe them. For Johnston, they are usually affable, ingratiating, friendly, outgoing and jovial.

I often only hear only two words to describe Francis, consummate gentleman.

After interacting with both on multiple occasions since then, I cant argue. So, for a lot of Penguins fans who are aware of Johnstons and Francis individual reputations, the notion of static between the two men may be hard to picture.

But Francis is now on his second go-round as a general manager himself. After holding that title with the Carolina Hurricanes (ironically, the team that used to be the Whalers), Francis is now the GM for the expansion Seattle Kraken. And he seems to have a better grasp now on why things were so tense then.

Looking back, I cant point the finger directly back at E.J. You work directly for the owner sometimes. Thats where a lot of this stuff came from, Francis said.

Bingo!

Its a great deal for the Hartford Whalers, owner Richard Gordon declared after the trade was announced.

History would look back and disagree. In time, so would Gordon himself.

Johnston would eventually return to Pittsburgh and coach Francis. Hes still with the organization as a community relations liaison at the age of 85. During a team documentary about the 1991 Stanley Cup season, Johnston told AT&T Sportsnet, We had no choice. He said if (Francis) is here on Monday, you guys (Johnston and his staff) wont be here. Get Ronny out of there.

According to Penguins GM Craig Patrick in that same documentary, Johnston then started calling every general manager in the league. And he put together the best package to pry Francis out of Connecticut.

By 2006, Gordon was telling the Hartford Courant, I consider the trade we made the worst trade in the history of hockey.

Now, on that front, history may look back and agree.

A quick return

Three games into their Penguins careers, the three ex-Whalers returned to Hartford with their new team for a road game.

I remember being floored by the support of the fans, Francis recalls. Pictures along the glass wishing me well. I had been there 10 years. A lot of good friends. Good to see those folks after the deal.

Cullen scored. Francis didnt. But he had an assist, and the Penguins won 5-2.

Theyd go 8-2-1 over the first 11 games after the trade, climbing from third place in the Patrick Division to first. During that stretch, the Pens allowed just 2.36 goals per game. They had allowed 5.60 goals per game in their five-game winless streak before the deal.

The plan at the time of the trade was falling into place.

Sometime after Game 3, Ulfy and I went out for a bite to eat, and I said this team can win the Cup. We felt that it had that much talent, Francis recalls.

And they did. Twice. As Francis contributed 28 points in 38 total regular-season and playoff games in 91. Jennings and Samuelsson contributed everything they were supposed to on the blue line.

For the ex-Penguins on the Whalers, it didnt go as well. They failed to win any of their final seven games. Then they lost to the Boston Bruins in six games during the first round of the Adams Division playoffs.

Zalapski made the All-Star team in 93 and topped out with 65 points that season after a career-high 20 goals in 92. But he never quite lived up to his fourth overall draft-choice status or Olympic pedigree that he had coming into the league.

For his part, Parker only played four games with the Whalers before suffering career-ending head and knee injuries.

Cullen was pretty good with 16 points in the final 13 regular-season contests and nine points in six playoff games. Plus 77 more the next season contributing 102 total points in 109 regular-season games during his time in Hartford.

But it was never the same for him there as it was in Pittsburgh.

Ronny was such an icon, such a legend. Hartford loved Ronny, Cullen said. You cant replace anybody else in their shoes. You have to stand on your own two feet. But it was tough. If I had bad games, people would boo. It was not a great situation for me in Hartford.

Jennings did have some sympathy for the fans the trio left behind in Hartford.

Every time we won a series and would go to the next level, it would make it look that much better for Pittsburgh and worse for Hartford, Jennings said. The fans took that on the chin. I felt sorry for them. But that was the decision management made. And they had to live with it.

The Final 20

Francis recalls the atmosphere in the locker room between the second and third periods of that Stanley Cup-clinching Game 6 against the Minnesota North Stars. It was 6-0 at the time.

It started with one guy throwing out a typical hockey cliche. Keep the shifts short! Then the next cliche. Get it over center ice and get it in deep. And then it was Third man high!

And it kept getting louder and louder and louder until Tom Barrasso said, Take a deep breath. Were up 6-0. Were going to be fine.

It was Paul Coffey, however, who said something even more sage as the clock got down to two minutes left at 8-0.

I remember standing on the bench watching the clock wind down the final seconds, Francis continued. And Paul Coffey said, As great as this moment is, its just going to get better every day of your life.

A very true statement from a guy who had won a few cups before that one.

Samuelsson and Jennings were around through most of 1995, at least long enough to see Cullen return to Pittsburgh for a year.

Francis would stay in Pittsburgh for 1992. He was an indispensable force on the team while Lemieux missed time due to injury during the teams second Stanley Cup run. And then for six more years beyond that, as he patched up his relationship with Johnston and played under him as a coach for four years.

A relationship Francis says is still good to this day.

In all, the Hall of Famer had 713 total points in 630 combined games played for the Penguins between the regular season and the playoffs.

So, in the end, I guess Hartfords Scott Farkas was right.

But I bet hes still ticked off about it. And that makes me smile.

You can hear Tim Benzs entire conversation with Ron Francis here.

Tim Benz is a Tribune-Review staff writer. You can contact Tim at tbenz@triblive.com or via Twitter. All tweets could be reposted. All emails are subject to publication unless specified otherwise.

Categories:Penguins/NHL | Sports | Breakfast With Benz | Tim Benz Columns

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A Lifelong Love Affair Is Coming to an End for This Revered Tennis Teacher – UVA Today

Posted: at 5:01 am

While growing up as the son of a steel worker in a blue-collar, football-loving town in western Pennsylvania, Ron Manilla didnt know much about tennis.

So when a monsignor at the Catholic school he had just started attending as a ninth-grader tried to recruit him to a tennis team that was short on players, Manilla who had played only football and basketball to that point scoffed.

I said, No way Im playing that sport. Thats a sissy sport, Manilla recalled. He said, Why dont you show up at the tennis courts tomorrow and Ill show you who the sissy is.

So I went out there and he just pulverized me up and down I thought I was going to die, Manilla said, laughing. And that was it. I just realized it was a really fun sport, a really tough sport, and ever since that day Ive been hitting tennis balls.

More than a half-century after first picking up a racket, Manilla is retiring. The longtime tennis director at the University of Virginia Foundation-owned Boars Head Sports Club a fixture in the Charlottesville tennis community, on the UVA tennis scene and in the Special Olympics world has announced that March 12 will be his last official day as a full-time teaching professional.

The man with the jovial disposition, the Santa Claus-like beard and the million-watt smile will be missed.

He not only touched the lives of many because of the racket in your hand, but also with the delivery of life lessons along the way, Boars Head General Manager Russ Cronberg said. Without a doubt, Ron has directly shaped the importance of tennis in our community.

When I think of the Boars Head Sports Club and of the people who make that place so special, I think of Ron Manilla, UVA mens tennis head coach Andres Pedroso said. Hes someone who treats everyone with consideration, is so kind to everybody, a friend to everybody in the organization and all the members. He says hello to everybody, is very flexible with everybody and is just a very friendly person who has been a joy to be around.

Hes been a major asset for not only the Boars Head, but also for UVA tennis and the Special Olympics.

Manilla, who has taught tennis to three generations of Charlottesville families, smiles when thinking back to his earliest days in the sport. He grew up in the small steel-mining town of Farrell before attending high school in Erie and then going on to play tennis at Gannon University (also in Erie), where he served as team captain.

The more Manilla played, the more his love for the game grew. His favorite players were Bjrn Borg and Evonne Goolagong.

After graduating with a degree in psychology in 1973, Manilla worked as a guidance counselor for troubled youth in Erie and served as Gannons head coach.

I knew how to play, but I didnt know much about coaching, Manilla said. There werent many books about it back then. I just had to start analyzing stroke technique and that kind of thing and just go from there.

Tired of Pennsylvanias cold weather, and with his older brother, Larry, living in Culpeper, Manilla moved to Charlottesville in 1976. He again worked with troubled youths through Charlottesvilles Community Attention program and taught tennis part-time at the YMCA, as well as in the city and county parks and recreation departments. Manilla combined his passions for teaching tennis and helping kids via a program he helped institute for latch-key children.

I considered myself like a grass-roots pro. I think that was the type of style that I used for the rest of my career, Manilla said. It was about being community-oriented and teaching tennis to as many people as I possibly could.

Manilla started as the full-time YMCA tennis director in the late 1970s. Soon after, he started the Get Acquainted Tennis Club, an adult program that he ran for 33 years worth of Saturday mornings at Charlottesville High School.

We went after players who had played, but who had quit playing due to jobs and raising kids, Manilla said. At times, we had over 200 people coming. It was so cool to help get so many players back to the game.

He was a darn good coach. You could see the quality of his tennis expertise in the way he worked with [players]. He was able to impart to them the tennis guidance they really needed.

- Lloyd Rauppfather of Special Olympics tennis player Chris Raupp

Manilla took over as director of the Atlantic Coast Athletic Club on Four Seasons Drive in the mid-1980s before coming to the Boars Head in 1995. It gave me an opportunity to really expand my career both as a tennis teacher and a tournament director, Manilla said. I had a staff, a beautiful facility and was able to get into a whole new style of teaching.

Manilla has been instrumental in bringing a pair of professional tournaments to the Boars Head that, starting in 2002, Charlottesville community members have flocked to.

Many fans still talk about the womens event held in 2003 that featured Russian superstar Anna Kournikova. Spectators started showing up at 7:30 a.m. for the 5 p.m. match. The place became so packed that some onlookers climbed trees to get a glimpse of the action. The match wound up being the last of Kournikovas career; she retired immediately after.

Over the years, several other high-profile players have come to play in the events including former No. 1 players Jelena Jankovi and Samantha Stosur, recent Australian Open finalist Jennifer Brady, former UVA star Danielle Collins, teen sensation Coco Gauff, career grand slam doubles winner Leander Paes, all-time aces leader Ivo Karlovic and former top-10 player Jack Sock.

All the players always say he is one of the best tournament directors, said former UVA star Treat Huey, who has enjoyed a successful doubles career on the pro tour since graduating in 2008. Hes always talking to everybody and asking them if he can do anything to help anybody.

He does such a professional job with those tournaments, UVA womens tennis head coach Sara OLeary said. I think whats amazing is all the great players who come to play in them because he runs them so professionally and does such a great job. Players know these are great tournaments and are so well-run.

In 2008, Manilla started the Xperience Special Olympics tennis tournament at the Boars Head.

We could have no better champion for Special Olympics than Ron Manilla, David Thomason, vice president of advancement for Special Olympics Virginia, said. Ron has opened so many doors for us in the Charlottesville community and far beyond. He opened the doors of Boars Head Sports Club for sure, but, as importantly, he opened the doors for connection with a community that, at his prompting and through his example, has enthusiastically and generously embraced Special Olympics Virginias core values of respect, inclusion and unity, and allowed people of all abilities to succeed on the court, in school, at work, and in the community.

Rons approach has exemplified what we want to achieve at SOVA using sport as a catalyst for attitudinal and societal change.

In 2018, Manilla served as the Special Olympics coach for Virginia at the USA Games in Seattle. The next year, he was a national coach at the World Games in Abu Dhabi, Dubai.

Local resident Chris Raupp, a Special Olympian who has worked in food preparation for Aramark at UVAs Newcomb Hall dining room since 1992, played under Manilla on those teams, winning a gold medal in doubles at the World Games.

He was a darn good coach, said Lloyd Raupp, Chris father. You could see the quality of his tennis expertise in the way he worked with [players]. He was able to impart to them the tennis guidance they really needed. He was able to work with them psychologically as well calming them down mentally and physically. And he would yell, too. He wasnt wishy-washy about it. He was there to get [players] to play the best that they could.

At the singles event in Abu Dhabi, Chris Raupp finished fourth and didnt medal after coming down with an illness.

We saw Ron consoling him afterward and telling him, Hey, you did good. You are the fourth-best player in the world. Dont you forget that, Sue Raupp, Chris mother, said.

He and Chris are best friends, she added. He talks about how he loves Chris, and Chris loves him.

Manilla has four children of his own with his former wife, Diana Marchibroda. At one point, all four worked in tennis, and in 2008 the Mid-Atlantic United States Tennis Association named the Manillas the Virginia Family of the Year.

At the time, Manillas oldest son, Dominic, had just graduated from Old Dominion University and was starting his career as a coach; Danny, who also played at ODU, was teaching tennis in Leesburg; Anna, who played for Western Albemarle High School, was working as a USTA league coordinator; and Joey, after also starring at WAHS, was playing at Christopher Newport University.

That was the coolest thing that could ever happen to a dad whose life is tennis, said Manilla, breaking into a wide smile. That was really cool. All four of my kids stayed in tennis. It was very humbling to me that theyd want to do it.

Treat Huey still gets a chuckle when he thinks back to the time UVA played a match against ODU. A normally staunch supporter of the Cavalier teams, Ron Manilla was cheering loudly for Dominic, a player on the Monarchs team.

He was a really big UVA fan, but he wasnt that day for sure, said Huey, laughing. And I thought that was really cool.

Manillas talent as an instructor was recognized in 2013 when the Professional Tennis Registry named him an International Master Pro, a distinction tantamount to a lifetime achievement award.

Tim Rose, the chief executive officer of the UVA Foundation, which operates the Boars Head Resort, said Manilla helped elevate the clubs tennis reputation on the East Coast.

Manilla also gave Rose his first-ever lesson. I was jumping up to swat my serve, emulating what I had seen on TV, Rose recalled. Ron suggested we start with just trying to get it over the net!

Rose said Manilla has been a teacher, a colleague and a friend.

Members of the Boars Head staff likely share a similar sentiment. Around the club, its commonplace to see them immersed in deep conversation with Manilla.

Pedroso said Manilla always seems to be in a good mood.

Every time you see him, he says hello genuinely, Pedroso said. And he stays relaxed. Ron just kind of takes everything in stride. He just has a special presence about him where you want to be around him. He lightens the mood and makes being around him very pleasant. I think thats why hes been around for so long. People want to be around people like that.

During a recent workday, Manilla gave a lesson to a 3- and a 4-year-old, then followed it up with a session with a woman in her 90s.

Manilla still plans to give lessons when he can and will stay involved in hosting the clubs professional tournaments, as well as the Xperience, but at age 70, he will no longer be on the court feeding balls to players for 27 to 30 hours a week.

When you turn my age, you start to think about things that you should be doing, Manilla said. Ive done this for so long. Is it that Im tired and just ready to quit? No, because I still have a passion for it. Do I really want to give that up? Well no, I really dont want to, but thats all Ive ever done feeding balls to people. And its been wonderful. Ive created great friendships.

But you just get to this point where you [realize] you havent done much of anything else. It was a hard decision to make, but I feel it was the right thing to do. I want to see my grandkids and get involved in their lives and be able to drive down to Norfolk and watch ODU [where Dominic is now head coach] play every single match.

I consider myself very lucky to have had all the experiences that Ive had. I just feel very grateful for everything.

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LogicBio Therapeutics to Present at Upcoming Investor Conferences – PRNewswire

Posted: March 3, 2021 at 2:05 am

LEXINGTON, Mass., Feb. 26, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood, today announced that chief executive officer Frederic Chereau will be presenting an overview of the company at upcoming investor conferences.

About LogicBio Therapeutics, Inc.

LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene delivery and gene editing platforms to address rare and serious diseases from infancy through adulthood. The company's proprietary GeneRideTM platform is a new approach to precise gene insertion that harnesses a cell's natural DNA repair process leading to durable therapeutic protein expression levels. LogicBio's cutting-edge sAAVyTM capsid development platform is designed to support development of treatments in a broad range of indications and tissues. The company is based in Lexington, MA. For more information, visit https://www.logicbio.com/.

Media Contacts:

Adam DaleyBerry & Company Public RelationsW: 212-253-8881C: 614-580-2048[emailprotected]

Jenna UrbanBerry & Company Public RelationsW: 212-253-8881C: 203-218-9180[emailprotected]

Investor Contacts:

Matt Lane Gilmartin Group 617-901-7698[emailprotected]

SOURCE LogicBio Therapeutics, Inc.

https://www.logicbio.com

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Orchard Therapeutics Reports 2020 Financial Results and Reviews Recent Accomplishments – BioSpace

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BOSTON and LONDON, March 02, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today reported financial results for the year ended December 31, 2020, as well as recent accomplishments, 2021 strategic priorities and upcoming milestones, and related organizational leadership updates.

Frank Thomas, president and chief operating officer said, It is gratifying to witness the positive momentum Orchard has already established in early 2021 driven by solid execution. Our compelling data in neurodegenerative disorders at the WORLDSymposium and successful completion of the $150 million financing exemplify this recent progress and showcase a growing appreciation for the potential of HSC gene therapy. We look forward to continuing our work in the year ahead and delivering further benefit for patients and our shareholders."

Recent Accomplishments

February 2021

January 2021

2021 Corporate Priorities and Upcoming Milestones

Orchard previously outlined the following key corporate objectives and upcoming expected milestones:

Organizational Leadership Updates

Given the progress on key development programs, Anne Dupraz has been appointed to the expanded role of chief development officer. In addition to overseeing the companys regulatory strategy, Ms. Dupraz will lead product development with the goal of ensuring a seamless approach to moving Orchards programs through to potential regulatory approval. Ms. Dupraz possesses more than 20 years of experience in the clinical and regulatory fields and has deep expertise in advanced therapies, having been involved in more than 50 different tissue, cell and gene-based therapy development programs in her career.

Ran Zheng, chief technical officer, and Andrea Spezzi, chief medical officer, are stepping down from their respective leadership positions with Orchard to pursue other opportunities. Orchard has initiated a global search for permanent replacements for both of these roles.

Fourth Quarter 2020 Financial Results

Research and development expenses were $22.6 million for the three months ended December 31, 2020, compared to $30.9 million in the same period in 2019. R&D expenses include the costs of clinical trials and preclinical work on the companys portfolio of investigational gene therapies, as well as costs related to regulatory, manufacturing, license fees and milestone payments under the companys agreements with third parties, and personnel costs to support these activities. The company expects R&D expenses to grow slightly in the upcoming periods as the company continues to advance its programs through later stages of development.

Selling, general and administrative expenses were $16.2 million for the three months ended December 31, 2020, compared to $18.5 million in the same period in 2019. The decrease was primarily due to realization of savings associated with an updated strategy and corporate restructuring announced in May 2020.

Net loss attributable to ordinary shareholders was $33.6 million for the three months ended December 31, 2020, compared to $45.4 million in the same period in 2019. The decline in net loss as compared to the prior year was primarily due to savings realized in our operating expenses as a result of the companys updated strategy and corporate restructuring. The company had 98.3 million ordinary shares outstanding as of December 31, 2020.

Thomas continued, "Our burn rate has declined from prior periods as we see the positive impact of our May 2020 corporate restructuring take hold, providing a longer runway and greater financial flexibility, aided by our recent financing. We are investing to support execution for the highest value programs in our portfolio while also dedicating capital to our longer-term strategy to expand into larger indications.

Cash, cash equivalents and investments as of December 31, 2020, were $191.9 million compared to $325.0 million as of December 31, 2019, with the decrease primarily driven by cash used to fund operations in 2020. In the fourth quarter of 2020, the cash used to fund operations was approximately $12.0 million after the receipt of approximately $19.2 million from R&D tax credit refunds related to 2019 qualifying activities under the tax code in the UK. The company expects that its cash, cash equivalents and investments as of December 31, 2020, along with gross proceeds of $150.0 million from the February 2021 private placement, will support its currently anticipated operating expenses and capital expenditure requirements into the first half of 2023. This cash runway excludes the $50 million available under the companys credit facility and any non-dilutive capital received from potential future partnerships or priority review vouchers granted by the FDA following future potential U.S. approvals.

About Libmeldy / OTL-200

Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, has been approved by the European Commission for the treatment of MLD in eligible early-onset patients characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

Libmeldy is not approved outside of the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

Libmeldy was developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, the therapeutic potential of Libmeldy (OTL-200) and Orchards product candidates, including the product candidates referred to in this release, Orchards expectations regarding its ongoing preclinical and clinical trials, including the timing of enrollment for clinical trials and release of additional preclinical and clinical data, the likelihood that data from clinical trials will be positive and support further clinical development and regulatory approval of Orchard's product candidates, and Orchards financial condition and cash runway into the first half of 2023. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchards ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates; the delay of any of Orchards regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates or the receipt of restricted marketing approvals; the inability or risk of delays in Orchards ability to commercialize its product candidates, if approved, or Libmeldy, including the risk that Orchard may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter ended September 30, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

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Major NIH study seeks to understand unusual forms of diabetes Washington University School of Medicine in St. Louis – Washington University School of…

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Researchers seek participants with diabetes that doesnt fit type 1 or type 2 categories

Most diabetes patients have either type 1, which is managed with insulin injections, or type 2, treated with other medications. Researchers at Washington University School of Medicine in St. Louis are part of a national research network aiming to discover rare forms of diabetes as a first step toward providing more effective treatments for forms of the disorder that don't fit neatly into either type 1 or type 2.

Washington University School of Medicine in St. Louis is part of a national research network aiming to discover rare forms of diabetes as a first step toward providing more effective treatments.

Most patients diagnosed with diabetes have either type 1, which is managed with insulin injections because patients cant make their own insulin, or type 2, in which patients either dont make enough insulin or dont respond well to the insulin their bodies produce. People with type 2 diabetes are treated with medications.

But an increasing number of patients dont fall into these two general categories, and they need a more precise diagnosis and a more individualized treatment. The Rare and Atypical Diabetes Network (RADIANT), funded by the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health (NIH) will involve about 2,000 people nationwide who will be screened for unusual forms of diabetes.

We are looking for new and different forms of diabetes, particularly in immigrant and minority populations, where it has been reported that atypical forms of diabetes are more common, said Fumihiko Urano, MD, PhD, the lead investigator at the Washington University clinical site. We also are looking for rare types of diabetes that result from specific gene mutations. Using precision medicine, we have tools now to identify, and possibly to treat more effectively, these rare forms of disease.

Urano, the Samuel E. Schechter Professor of Medicine and a professor of pathology and immunology, plans to recruit 200 patients with diabetes in the next year at the Washington University site. He and the other network researchers envision building a comprehensive database of genetic, clinical and descriptive data about previously unidentified forms of diabetes to provide more information about those forms of the illness to doctors and researchers, allowing them to recognize atypical forms of diabetes more quickly, as well as to develop new or more personalized treatment strategies.

Those eligible to participate include individuals diagnosed with type 2 diabetes before the onset of puberty, those with type 2 diabetes who are not overweight, and people with a family history of diabetes, particularly those from families where most family members with diabetes were diagnosed before the age of 18. Investigators also are seeking those with very abnormal cholesterol or lipid levels and an unusual distribution of body fat, as well as people whose diabetes appears to wax and wane. Those not eligible for the study include people who have a high likelihood of having an already known form of diabetes and women who are pregnant.

Study subjects will be asked to complete surveys and to provide measurements of height, weight and blood pressure. They also will undergo genetic testing and be asked to provide researchers with detailed family histories.

Next, experts will review surveys and test results to determine whether an individuals diabetes can be considered atypical or rare.

Most people with atypical diabetes are diagnosed, at least initially, with type 1 or type 2 diabetes, but they tend to experience a different disease course, different complications or unusual responses to standard diabetes medications.

Urano has experience treating an extremely rare and atypical form of diabetes known as Wolfram Syndrome. That disorder features blood sugar problems, but unlike more common forms of diabetes, it is caused by a single genetic mutation and progresses to include serious vision, balance and other neurological problems.

As part of the RADIANT study, researchers will select certain participants to gather detailed clinical and biological information through a wide range of tests, including genome sequencing, blood sampling and physical exams; and participants may get additional specialized tests based on the specific characteristics of their form of diabetes.

Family members of enrolled participants also may be invited to participate in the study, particularly if the condition appears to be inherited. Members of minority groups are particularly encouraged to apply for the study because different forms of atypical diabetes although rare tend to be more common in minority populations than in Caucasians.

Its extremely frustrating for people with atypical diabetes when their diabetes seems so different and difficult to manage, said the studys project scientist, Christine Lee, MD, of the NIHs National Institute of Diabetes and Digestive and Kidney Diseases. Through RADIANT, we want to help patients and the broader health-care community by finding and studying new types of diabetes to shed light on how and why diabetes can vary so greatly.

Urano added, Our goal is to further clarify diabetes in its many different forms. By focusing on rare forms of diabetes, the study should help us better understand the spectrum of diabetes to improve the lives of people who have these rare forms of diabetes, and to assist the people who care for them.

Nationwide, the study is being led by researchers at the University of South Florida, the Baylor College of Medicine in Houston, and the University of Chicago.

For more information, people who have forms of diabetes that seem different from common types of the disorder may call Stacy Hurst at 314-747-3294, e-mail AtypicalDiabetes@wustl.edu or visit http://www.atypicaldiabetesnetwork.org for more information about the study and how to join.

This study is supported by the National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health (NIH). Grant numbers U54 DK118638 and U54 DK118612.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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The Genome Odyssey Review: From Code to Clinic – The Wall Street Journal

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In the months after Carson Miller was born, his parents Danny and Nikkia young couple living in Northern Californianoticed that he was having trouble with basic movements, like rolling over and crawling. He managed to take a few steps at 12 months but never got beyond that. When his brother Chase was born a year later, the parents anxiety turned to dismay as the younger boy also struggled with motor activity. Desperate to understand what was wrong, Danny and Nikki took their wheelchair-bound children to a series of doctors, enduring test after test; yet a diagnosis remained elusive. Finally, the family saw a group of medical detectivespart of an academic consortium called the Undiagnosed Diseases Networkwho subjected the familys DNA to a comprehensive analysis. The doctors at last identified the problem: an unusual defect in the energy machinery of the boys cells. Diagnosis firmly in hand, the family could now turn their attention to treatment.

The fact that critical information lurks within the three billion or so pairs of letters representing our genetic instructions was a key driver of the Human Genome Project, begun in 1990 and completed, with suitable fanfare, 13 years later. But translating DNA sequence into actionable insight hasnt been easy. This is a major theme of The Genome Odyssey, Euan Ashleys impassioned, firsthand account of the effort to bring genomic data into clinical practice and help patients like Carson and Chase.

For starters, says Dr. Ashley, a cardiologist and geneticist at Stanford, there was the priceprohibitively high early on. Thanks to advances in technology, the cost of sequencing an individuals DNA has declined a million-fold since 2003the equivalent, he says, of a Ferrari plummeting from $350,000 to less than 40 cents. The time required to decode a genome has plunged to days rather than years or months. When Jazlene, a newborn girl with a dangerously abnormal heart rhythm, arrived at Stanford in 2014, Dr. Ashley and his colleagues were able to identify the genetic cause within days and practice more precise medicine by fine-tuning the infants therapy.

At the level of DNA, humans are 99.9% identical, Dr. Ashley reminds us. The challenge, for doctors, lies in figuring out which differences in the remaining 0.1% are medically pertinent. For many families, the diagnosis of a rare genetic disease marks the end of one journey and the beginning of a second one, focused on learning from similar patients and partnering with researchers to search for a cure.

While Dr. Ashley concentrates on genetic disease, he also highlights the promise of DNA superhumanspeople who have been dealt an unusually good genetic hand. One example: Sharlayne Tracy, a college student in Dallas who enrolled in a University of Texas Southwestern study of cardiovascular health and was found to have astonishingly low levels of bad cholesterol. DNA analysis revealed a genetic defect disrupting PCSK9, a protein involved in cholesterol metabolism. The discovery prompted several pharmaceutical companies to develop medications targeting this protein. Two of the drugs, for the treatment of stubbornly high cholesterol, have been approved by the FDA.

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The Promises of CRISPR Genome Editing in Biomedicine – Labiotech.eu

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Tony Ho, Head of Research and Development at CRISPR Therapeutics, offers his insights into the advantages of CRISPR gene editing over other technologies and what the future holds for the genome editing field.

For decades, gene editing required engineering DNA-cutting enzymes, such as zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), for every specific genetic modification. The advent of CRISPR-Cas9, a gene-editing tool that only requires a custom guide RNA molecule to find its target, revolutionized the field.

CRISPR gene editing has quickly risen in popularity as a research tool because it makes genome editing much simpler and faster than ever before. The technology is also being researched as a potential treatment for a wide range of genetic diseases.

That is the case of CRISPR Therapeutics, a company that is running clinical trials testing the technology in two blood conditions as well as in cancer. Tony Ho, who leads research efforts at the company, shares his views on the potential of the technology and his expectations for the future of gene editing.

Genome editing technologies have been developed at a rapid pace over the past decade. Within the past five years, a novel system has been developed based on fundamental research of bacterial systems immunity to viral infections.

CRISPR-Cas9 is based on RNA-guided targeting and is much simpler and faster than earlier methods. The ease of design, together with the remarkable specificity, efficiency, and versatility of the CRISPR-Cas9 system have revolutionized the field of genome editing and enabled rapid development of novel therapies.

The advent of programmable nucleases has accelerated the development of gene editing from concept to clinic.Current advances in genome editing tools allow us not only to target monogenic diseases but also polygenic diseases, such as cancer and diabetes. Genomic editing also provides a degree of precision not previously possible by other therapeutic approaches through its ability to target individual cell types.

It also allows us to build on synthetic biology [techniques] that confer novel abilities to the cells. With these abilities, we can develop potentially curative therapies that may not be possible using traditional modalities.

The CRISPR-Cas9 system can modify DNA with greater precision than existing technologies. An advantage the CRISPR-Cas9 system offers over other mutagenic techniques, like ZFN and TALEN, is its relative simplicity and versatility.

Additionally,CRISPR-Cas9 has the potential of simultaneous multiple loci editing, making the technology easier, more efficient, and more scalable compared to other genome editing technologies.The programmability of the CRISPR-Cas9 system allows rapid prototyping of different therapeutic approaches, which leads to faster development cycles compared to other genome editing approaches.

While manufacturing is considered a challenge for the field, it is not a fundamental limitation. Companies are committed to overcoming challenges in order to bring medicines to patients at scale.

Additionally, its important to push the boundaries of what genome editing tools can offer to medicine. For example, its critical to move the field beyond therapeutic areas of rare diseases and hematologic malignancies, and into common diseases, like cancer and diabetes, where there is the potential to continue to see a dramatic impact and benefit to patients.

We expect to see increased enthusiasm for the gene editing field, particularly around CRISPR-Cas9, as it is a powerful platform that is facile and scalable. We believe the next generation of cell therapies will be driven by increased edits. We have published papers at oncology conferences that demonstrate that CRISPR-Cas9 can generate perfectly viable and enhanced CAR-T cells with 10 or more edits.

CRISPR-Cas9 is a revolutionary tool that can change the future of medicine. At CRISPR Therapeutics, we have five programs in the clinic, led by our two hematology programs, where we have demonstrated that a one-time CRISPR-based treatment can result in a functional cure for patients with sickle cell disease and beta-thalassemia.

We also have three ongoing clinical trials for allogeneic CAR-T therapies targeting B-cell malignancies, multiple myeloma, and renal cell carcinoma. Excitingly, we showed positive topline data in patients with relapsed or refractory B-cell malignancies, which demonstrate the potential of CRISPR gene editing for the treatment of cancers. We believe a number of cancers could be cured with CRISPR-based treatments, and we could replace a number of diseased or defective organs with regenerative medicine.

There is a world full of possibilities for CRISPR applications and I am very hopeful about what the future can bring.

This interview is part of Labiotechs report The Genome Editing Toolbox: ZFNs, TALENs, and CRISPR in Biomedicine. Become a member to get full access to this and many other reports offering a deep insight into the hottest areas of the biotech industry.

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Tenaya Therapeutics Secures $106 Million in Series C Funding to Accelerate Pipeline of Potentially Curative Therapies for Heart Disease – Business…

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SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Tenaya Therapeutics, a biotechnology company whose mission is to discover, develop and deliver curative therapies that address the underlying causes of heart disease, today announced that it has secured $106 million in Series C funding. Founded by leading cardiovascular scientists from the Gladstone Institutes and UT Southwestern (UTSW), Tenaya is advancing programs across three therapeutic platforms to address heart disease: Gene Therapy, Cellular Regeneration and Precision Medicine.

The Series C financing was led by RTW Investments, LP and adds new investors RA Capital Management, Fidelity Management & Research Company and funds and accounts advised by T. Rowe Price Associates, Inc. with additional participation from all existing investors including The Column Group, Casdin Capital, GV and others.

The new funding will allow Tenaya to advance its lead gene therapy program towards clinical studies; progress new programs towards IND-enabling studies; build on existing drug discovery and development capabilities across its three platforms; and invest in cGMP manufacturing capabilities.

Heart disease remains the leading cause of death in the world despite improvements in standards of care, said Faraz Ali, Chief Executive Officer of Tenaya. There is tremendous promise in the use of vastly more sophisticated tools for drug discovery available today to pioneer new classes of targeted treatments for heart failure, including potentially one-time curative gene therapies for genetic cardiomyopathies. We appreciate the strong support of new and existing investors who are helping us advance therapies to improve and extend the lives of patients.

Emerging Portfolio

Tenaya is advancing diverse and differentiated therapeutic approaches to gene therapy and small molecules for both prevalent and rare forms of heart disease:

Drug Development Capabilities

Since its Series B funding round announced in 2019, Tenaya has added to the depth and breadth of its drug discovery capabilities to support the efficacy, safety and differentiation of current and future products:

AAV Manufacturing

Tenaya also announced important steps towards internalizing its AAV manufacturing capabilities, expertise and IP to support its emerging portfolio of gene therapy products:

Tenaya has successfully built on its distinguished scientific heritage to advance a bold research strategy and differentiated drug discovery capabilities uniquely focused on heart disease, said Roderick Wong, M.D., Managing Partner and Chief Investment Officer at RTW Investments, LP. We are strong believers in the potential for new approaches in this area of high unmet need and are encouraged by early results we are seeing in the industry with the use of AAV to treat genetic forms of heart disease. We are excited to partner with Tenaya to accelerate their leadership position in this exciting field and to advance novel therapies that can benefit individuals and families fighting heart disease.

About Tenaya Therapeutics

Tenaya Therapeutics is shaping the future of heart disease treatment driven by a bold mission: to discover, develop and deliver curative therapies that address the underlying drivers of heart disease. Tenaya is developing therapies for rare genetic disorders as well as for more prevalent heart conditions through three platforms: Gene Therapy, Cellular Regeneration and Precision Medicine. Founded by leading cardiology researchers at the Gladstone Institutes and UT Southwestern, Tenaya is backed by a top-tier syndicate of investors. For more information, please visit http://www.TenayaTherapeutics.com and follow us on LinkedIn.

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Pharming Group, in collaboration with Invitae Corporation, launches genetic testing program ‘navigateAPDS’ in US and Canada – PRNewswire

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-New program expected to improve access to genetic testing for activated PI3K delta syndrome (APDS), an ultra-rare immunodeficiency disease

- Program has potential to advance clinical research in APDS as a result of earlier diagnosis

LEIDEN, The Netherlands, March 2, 2021 /PRNewswire/ -- Pharming Group N.V. ("Pharming" or "the Company") (Euronext Amsterdam: PHARM/Nasdaq: PHAR), a global, commercial stage biopharmaceutical company developing innovative protein replacement therapies and precision medicines for the treatment of rare diseases and unmet medical needs, in collaboration with Invitae Corporation (NYSE: NVTA, "Invitae"), a leading medical genetics company, announces the launch of a sponsored genetic testing program, navigateAPDS, designed to assist clinicians in identifying patients and their family members with activated PI3K delta syndrome (APDS), which may lead to earlier diagnosis.

APDS is an ultra-rare primary immunodeficiency disease caused by a genetic mutation affects approximately 1-2 people per million. Patients are often misdiagnosed with other immunodeficiencies or autoimmune disorders and often have a protracted course to obtain a correct diagnosis. A definitive diagnosis can be made only by a genetic test. Current treatment is generally limited to supportive therapies such as antibiotics and the use of immunoglobulin replacement therapy. There is no approved therapy for the treatment of APDS, however, clinical trials are currently ongoing, including Pharming's pivotal-stage development program for leniolisib, a small molecule phosphoinositide 3-kinase delta (PI3K) inhibitor, under development by Novartis and Pharming to treat patients with APDS.

Pharming's support of the program will facilitate genetic testing and counselling for eligible individuals in the United States and Canada at no charge. NavigateAPDS will use the Invitae Primary Immunodeficiency Panel (PI), which analyzes up to 407 genes that are associated with inherited disorders of the immune system. In addition to providing genetic testing to individuals who may present with a clinical picture known to be associated with APDS, navigateAPDS will offer pre-test and post-test genetic counseling through a third party, and all blood relatives of patients found to have a P/LP variant for APDS are qualified to be tested through the program. By offering access to the full PI panel, physicians and patients are more likely to identify the underlying cause and potential diagnosis without the need for additional expanded patient testing.

Sijmen de Vries, Chief Executive Officer commented:

"Our partnership with Invitae is an important step towards simplifying access to testing, which may allow for an expedited and accurate diagnosis for patients suffering from a primary immunodeficiency such as APDS. Earlier diagnosis can favorably impact disease management and could have a positive effect on long-term outcomes and patients' quality of life. The program is also a key component in advancing clinical research by identifying the underlying causes of APDS, which will help us better understand these patients' potential to respond to investigational precision medicines."

Robert Nussbaum, M.D., Chief Medical Officer of Invitae commented:

"Genetic information is a powerful tool that can improve outcomes for patients and their families, both by supporting more rapid diagnosis of ultra-rare diseases such as APDS and also by enabling access to clinical trials. Partnerships like our work with Pharming may enable the clinical trials necessary to develop precision therapies in areas of significant unmet need."

To learn more about the navigateAPDS program, visit http://www.invitae.com/navigateapds.

About APDS

APDS is an ultra-rare primary immunodeficiency first fully described in 2013 that affects approximately 1-2 people per million. APDS occurs when there is an abnormal change in either one of two specific genes, the PIK3CD gene or the PIK3R1 gene. The genes follow an autosomal dominant mode of inheritance which means one copy of the altered gene from either biological parent is sufficient to cause the disorder. The genes are involved in making parts of a protein that helps in the growth and division of white blood cells, particularly the B-cell and T-cell lymphocytes. APDS is present at birth and signs and symptoms start early in childhood with affected individuals having increased susceptibility to a myriad of inflammatory conditions including recurrent severe respiratory tract infections, chronic benign lymphoproliferation, hematopoietic malignancies, and/or autoimmune diseases. Patients are often misdiagnosed with other immunodeficiencies or autoimmune disorders and often have a protracted course to obtain a correct diagnosis. A definitive diagnosis can only be made by a genetic test and, once appropriately identified, regular specialized health check-ups and customized treatment plans are required. Current treatment is generally limited to supportive therapies such as antibiotics and the use of immunoglobulin replacement therapy. There is no approved therapy for the treatment of APDS, however, clinical trials are currently ongoing.

To learn more, visit http://www.allaboutapds.com.

About Pharming Group N.V.

Pharming Group N.V. is a global, commercial stage biopharmaceutical company developing innovative protein replacement therapies and precision medicines for the treatment of rare diseases and unmet medical needs.

The flagship of our portfolio is our recombinant human C1 esterase inhibitor, or rhC1INH, franchise. C1INH is a naturally occurring protein that downregulates the complement cascade in order to control swelling in affected tissues.

Our lead product, RUCONEST is the first and only plasma-free rhC1INH protein replacement therapy. It is approved for the treatment of acute hereditary angioedema, or HAE, attacks. We are commercializing RUCONEST in the United States, the European Union and the United Kingdom through our own sales and marketing organization, and the rest of the world through our distribution network.

We are also developing rhC1INH for subsequent indications, including pre-eclampsia, acute kidney injury and we also investigating the clinical efficacy of rhC1INH in COVID-19.

In addition, we are studying our oral precision medicine, leniolisib (a phosphoinositide 3-kinase delta, or PI3K delta, inhibitor), for the treatment of activated PI3K delta syndrome, or APDS, in a registration enabling Phase 2/3 study in the United States and Europe.

Furthermore, we are also leveraging our transgenic manufacturing technology to develop next-generation protein replacement therapies most notably for Pompe disease, which program is currently in the preclinical stage.

For more information please visit the company's website: http://www.pharming.com.

About Invitae

Invitae Corporation (NYSE: NVTA) is a leading medical genetics company, whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website at http://www.invitae.com.

Forward-looking Statements

This press release contains forward-looking statements, including with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, Pharming's ability to overcome the challenges posed by the COVID-19 pandemic to the conduct of its business, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in Pharming's 2019 Annual Report and its report for the nine months ended 30 September 2020, the events and circumstances discussed in such forward-looking statements may not occur, and Pharming's actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Pharming as of the date of this release.

Inside Information

This press release relates to the disclosure of information that qualifies, or may have qualified, as inside information within the meaning of Article 7(1) of the EU Market Abuse Regulation.

For further public information, contact:

Pharming Group, Leiden, The NetherlandsSijmen de Vries, CEO: T: +31 71 524 7400Susanne Embleton, Investor Relations Manager: +31 71 524 7400/[emailprotected]

Invitae, San Francisco, USACorporate CommunicationsLaura D'AngeloE: [emailprotected]

FTI Consulting, London, UKVictoria Foster Mitchell/Mary Whittow/Alex Shaw, T: +44 203 727 1000

LifeSpring Life Sciences Communication, Amsterdam, The NetherlandsLeon MelensT: +31 6 53 81 64 27E: [emailprotected]

SOURCE Pharming Group N.V.

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Pharming Group, in collaboration with Invitae Corporation, launches genetic testing program 'navigateAPDS' in US and Canada - PRNewswire

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