Category Archives: Genetic Therapy

The financing was led by T. Rowe Price funds and accounts, with participation from Farallon, Wellington Management Company and existing investors Atlas Venture, Fidelity, Invus, Casdin, Deerfield, Foresite Capital and an entity associated with SVB Leerink. Cowen served as exclusive placement agent for the offering.

Generation Bios non-viral platform moves beyond the limitations of existing gene therapy by enabling re-dosable long-lasting gene therapies for severe diseases on a global scale. The platform combines three unique technologies: a novel cell-targeted lipid nanoparticle delivery system (ctLNP), a proprietary closed-ended DNA construct (ceDNA) and a high-capacity capsid-free biologics manufacturing process. In addition to the liver, Generation Bio is developing gene therapies for patients with skeletal muscle and eye diseases.

Our vision is to develop re-dosable long-lasting gene therapies manufactured at a scale that leaves no patient or family behind, said Geoff McDonough, M.D., president and chief executive officer of Generation Bio. Since our founding, we have had the support of high-quality investors who share our excitement about our potential to lead a new generation of gene therapy as we advance our lead programs toward the clinic.

Generation Bios non-viral platform is designed to extend the reach of gene therapy through its potential to enable:

About Generation Bio

Generation Bio is a biotechnology company leading a new generation of re-dosable long-lasting gene therapy on a scale to potentially benefit more families living with a broader range of diseases around the world. The companys powerful non-viral platform combines three unique technologies: a novel cell-targeted lipid nanoparticle delivery system (ctLNP), a proprietary closed-ended DNA construct (ceDNA) and a high-capacity capsid-free biologics manufacturing process. In addition to its lead liver-targeted programs in hemophilia A and phenylketonuria (PKU), Generation Bio is developing gene therapies for patients with diseases of skeletal muscle and the eye. Generation Bio was founded by Atlas Venture and is headquartered in Cambridge, Mass.

For more information, please visit http://www.generationbio.com or follow us at @generationbio.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200110005089/en/

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Generation Bio Announces $110 Million Series C Financing to Advance Lead Programs for Hemophilia A and PKU - BioSpace

Its capacity to produce AAV (Adeno Associated Virus) materials at large-scale will support Genethon and Sareptas project to take micro-dystrophin to clinical trials this year

Corbeil-Essonnes, France, January 9, 2020 Yposkesi, a leading full-service Contract Development and Manufacturing Organization (CDMO) for preferred access and reserved capacity for cGMP grade viral vector production, today announces that it has been selected to produce clinical and large-scale commercial AAV micro-dystrophin material within the co-development program between Genethon and Sarepta Therapeutics focused on Duchenne muscular dystrophy.

Genethon and Sarepta recently announced they are extending their collaboration on developing an innovative gene therapy for Duchenne muscular dystrophy, which has demonstrated significant efficacy in pre-clinical testing. Yposkesis selection to produce clinical and large-scale commercial batches is based on its innovative proprietary suspension production process using a distinct producer clone and a specific transfecting agent.

Yposkesi is currently doubling the size of its production facilities to increase volume capacity of its bioreactors, enabling it to meet its clients growing needs. With the construction of an additional 5,000m2 site underway, Yposkesi plans to deliver commercial batches in 2022/2023.

Yposkesi is proud to have been selected to produce and deliver AAV material at large-scale using its proprietary suspension-based process for Duchenne muscular dystrophy, a neuromuscular disease that requires high doses, said Alain Lamproye, CEO of Yposkesi. The significant investments made in honing our expertise, expanding our facilities, as well as developing innovations to increase production capacity and process efficiency are aimed at serving gene therapy developers, especially those currently facing a bottleneck in manufacturing.

About Duchenne muscular dystrophyDuchenne muscular dystrophy is a rare, progressive genetic disease that affects all the muscles of the body in approximately one in 3,500 boys. It is the most common neuromuscular disease in children. It is linked to abnormalities in the DMD gene, which is responsible for the production of dystrophin, a protein that is essential for the proper functioning of the muscles. This gene has the characteristic of being one of the largest in our genome (2.3 million base pairs of which more than 11,000 are coding). Because of this size, it is technically impossible to insert the complete DNA of dystrophin in a viral vector (or even the only 11,000 coding base pairs), as is usual in gene therapy.

About YposkesiYposkesi is a leading Contract Development & Manufacturing Organization (CDMO) for gene therapy vector manufacturing. Created in November 2016 in Corbeil-Essonnes (France) as a spin-off from the world-class gene therapy pioneer Genethon, Yposkesi provides integrated services covering bioprocess development (USP & DSP) from small/pilot to large-scale production, analytical development, GMP manufacturing of lentiviral and AAV vectors and regulatory support. Its current facility consists of a 50,000ft2 (approx. 5,000m2) building, operating multiple manufacturing suites for bulk drug substance and Fill&Finish. By 2022/23, Yposkesi will increase its global footprint to 100,000ft2 (approx. 10,000m2) with a second large-scale facility designed for EMA and FDA compliance. Capitalizing on the more than 25 years expertise of Genethon, Yposkesi invests significantly in innovation in bioprocessing to deliver on high-quality projects, cost-effectively. Yposkesi has also entered into several strategic partnerships including those with Axovant Gene Therapies, Servier and Orchard Therapeutics.www.yposkesi.com

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Yposkesi selected to produce clinical and large-scale commercial material of micro-dystrophin for Genethon and Sarepta Therapeutics program - BioSpace

Whoever comes out on top of the current race to gain pioneering approvals for new AAV-delivered gene therapies will have to look over their shoulders to watch the next tech wave forming on the horizon for gene therapy 2.0.

One of those next-gen players, Generation Bio, just brought in $110 million of venture cash to cover the cost of the rest of their preclinical journey toward something completely new in the field. The latest round brings the biotech which now has about 80 staffers up to $235 million in total since its inception about 3 years ago. That will fuel the rest of its preclinical stage of development as it looks to break into human studies in the back half of 2021.

That kind of 4-plus year timeline before the first human dosing could test the endurance level of a venture player. But Generation CEO Geoff McDonough looks over the past 2 years advancing a new lipid nanoparticle delivery system for their closed-end DNA therapies working to the day when gene therapies can be produced and sold for far less than the $2 million-or-so price tag today and sees lots of fast-paced advances.

I think the reality is we didnt have an expectation at the outset (on timelines), McDonough tells me. Recognizing the novel work needed to build the platform, the investors knew it would take time and money to bring them up to a GMP level.

I would say for a 40-year problem, adds the CEO, 2 years seems pretty good.

The founding tech at Generation was designed to do what AAV treatments do in the nucleus, offering enduring expression, while allowing manufacturing at a biologic scale with a more economical, capsid-free production method. Taking a page from the tech handbooks at companies like Alnylam and Moderna, theyre building a gene therapy that they believe can do much better than the fragile, one-time-only pioneers. And without the $1 million production cost that keeps wholesale prices in the low 7-figure range.

Theyre looking for much greater economy, eventually taking these therapies to much broader ailments and out of the realm of rare diseases with a new approach that they believe can be infinitely redosable on an as-needed basis.

Thats the big picture.

Generations team is working on 2 lead programs for hemophilia A and phenylketonuria (PKU) to go into IND-enabling studies. Theyve now identified Wilson disease and Gaucher disease as likely starting points for the next steps as they move past the liver to skeletal muscle and the retina and then other tissues. And McDonough the former CEO at Sobi is looking down the road 12 to 18 months when hed like to turn to the public markets with an IPO to fund the first clinical-stage work.

In the meantime, hed like to concentrate on opening another new chapter of the company on the dealmaking side.

It felt very important not to partner initially, says McDonough. The investors wanted to retain ownership of platform. We just had tremendous good fortune we didnt need to do that for finance reasons. But now that they have a better grasp of the technology and what needs to be done, its time to partner probably later in the year.

T. Rowe Price funds and accounts led the round, with Farallon and Wellington Management Company jumping in alongside. Existing investors Atlas Venture, Fidelity, Invus, Casdin, Deerfield, Foresite Capital and an entity associated with SVB Leerink came back to stay in the syndicate. Cowen served as exclusive placement agent for the offering.

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With $110M to add to the bankroll, Generation Bio sets its sights on engineering a revolution in the gene therapy field - Endpoints News

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These reports, excerpted and edited by Barrons, were issued recently by investment and research firms. The reports are a sampling of analysts thinking; they should not be considered the views or recommendations of Barrons. Some of the reports issuers have provided, or hope to provide, investment-banking or other services to the companies being analyzed.

American Water Works AWK-NYSE Buy Price $120 on Jan. 8 by Janney Montgomery Scott

American Water Works acquisition activity has been robust recently, with five announced deals in the past 30 days, totaling $108 million. Illinois and Pennsylvania accounted for the bulk of the deals, with New Jersey also generating a small transaction. This provides reinforcement to our positive investment thesis, and we maintain our Buy rating and $153 fair value on the shares, based on a price/earnings ratio of just over 35 times our 2021 earnings-per-share estimate of $4.32.

Newtek Business Services NEWT-Nasdaq Underperform Price $22.83 on Jan. 8 by Raymond James

We are downgrading our rating to Underperform from Market Perform.

We believe that there are a number of earnings headwinds ahead, with difficult growth targets in the Small Business Administration 7(a) lending business and a tougher comparison for Newteks merchant services segment. There is potential for upside versus our expectations from the conventional lending joint venture, but we do not believe that opportunity overwhelms the risk we see from other segments at this stage.

While there are always other levers to pull, such as one-time dividends from control subsidiaries, such earnings sources could result in a weaker quality of returns and [might] impact the sustainability of the current premium multiple valuation.

Royal Caribbean Cruises RCL-NYSE Overweight Price $130.44 on Jan. 7 by J.P. Morgan

We are raising our 2020 year-end price target to $154, from $135, on a slightly higher 2021 estimated P/E multiple of 13 times, from 11.4 times previously (no change to estimates).

A higher multiple is warranted, given that Royal proved in 2019 that it could comfortably grow core yields in the current elevated-capacity growth environment. Thirteen times is 1.0-1.5 times below the companys long-term average multiple and still well below the broader market multiple, and fair, in our view, given where we are in the economic cycle (late), and the inherently significant operating leverage in the cruise-line business model.

Cytokinetics CYTK-Nasdaq Buy Price: $10.83 on Jan. 6 by H.C. Wainwright & Co.

This morning, Cytokinetics announced the opening of enrollment in the Redwood-HCM study, a Phase 2 clinical trial testing CK-274 for the treatment of hypertrophic cardiomyopathy [HCM, a genetic disorder characterized by an abnormally thick heart muscle].

CK-274 has been optimized to ensure rapid symptom relief for HCM patients, which is particularly important in this patient population, and very valuable compared with gene-therapy approaches (which probably require longer periods of time, after administration, before becoming effective in relieving symptoms). We reiterate our Buy rating and price target of $30.

Lyft LYFT-Nasdaq Buy Price $43.58 on Jan. 2 by CFRA

Our Buy opinion reflects our outlook on Lyfts attractive end-markets/geographic exposure, scalable and recurring-based business model, as well as our expectation for considerable margin expansion through 2021. We see Lyft taking market share in the still nascent mobility-as-a-service space, with overseas expansion as a potential significant growth opportunity long term (the company is currently operating in the U.S. and Canada only). We like Lyfts initiatives and partnerships [for] autonomous capabilities.

Risks include slower-than-expected adoption of mobility-as-a-service, lower-than-expected revenue per active rider, greater-than-projected insurance costs, higher-than-expected expense growth, and greater-than-forecast intermediate-term cash burn.

Our 12-month $70 target price is based on our discounted-cash-flow analysis.

Bio-Techne TECH-Nasdaq Overweight (Volatile) Price $217.16 on Jan. 7 by Stephens

Since 2013, Bio-Techne has expanded its portfolio of assets from legacy reagents/diagnostics, moving into instruments and new clinical applications, which has resulted in growth accelerating from the low-single to the low-double digits. We see this continuing as Bio-Techne begins to benefit from cell- and gene-therapy capabilities, along with Exosome (liquid biopsy), in coming years. All of which should result in Bio-Techne achieving its targets of $1.2 billion in revenue and 40% margins by 2023.

Since CEO Chuck Kummeth joined the company in 2013, Bio-Techne has transformed into a more-diversified, higher-growth life-science company. Given the outlook for accelerating organic growth and expanding margins, we [give it] an Overweight (Volatile) rating, with a $250 price target.

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The Investment Allure of American Water Works - Barron's

WORCESTER (AP) The family of a prominent Boston-area physical therapist and co-founder of Christmas in the City, a volunteer-run charity to help homeless children, has launched a new fund at the University of Massachusetts Medical School to support amyotrophic lateral sclerosis, or ALS, research under the direction of world-renowned ALS researcher Dr. Robert H. Brown Jr.

Jake and Sparky Kennedy announced this week the Jake Kennedy ALS Fund at UMass Medical School. The focus for raising $1 million to help find a treatment and cure for ALS touches the family personally. And the clock is ticking.

ALS is a progressive and ultimately fatal neurological disease that attacks the motor neurons in the brain, brain stem and spinal cord. Most people with ALS die from respiratory failure, usually within three to five years from when the symptoms first appear, according to the National Institute of Neurological Disorders and Stroke. About 10% of people with ALS survive for 10 years or more.

Jake Kennedy, 64, co-founder of Kennedy Brothers Physical Therapy, was recently diagnosed with ALS, a disease that already has stormed through his family.

His father, Chris Kennedy, former dean of students at Northeastern University, died from ALS in 1989, a year after he retired and the disease was diagnosed.

Jakes youngest brother, Jimmy, died from ALS in 1997 at age 31.

In 2016, his brother and physical therapy business co-founder, Richard Ratt Kennedy, was diagnosed with the disease while training for his 32nd Boston Marathon.

One of Jake and Sparkys four children, Zachary Kennedy, is working on the front lines toward a cure.

Zack Kennedy, 31, of Worcester, received his Ph.D. in biomedical sciences in August from UMass Medical School, where he was mentored by Brown. Zack works now in the lab overseen by Anastasia Khvorova and collaborates with Brown and others racing to develop gene therapy to halt ALS.

Jake has a form of ALS known as Bulbar onset, which affects his voice and swallowing first. He and Sparky, who live in Salem, New Hampshire, spoke to the Telegram & Gazette in a phone interview Thursday.

This is a very optimistic time in the research world, Jake said. Dr. Brown is very, very optimistic about finding a cure for ALS.

The couples children wanted to start the ALS fund at UMass to carry on their fathers legacy, said Sparky.

The ALS gene is in the family and we wanted it to go to Dr. Brown, she said.

Jakes wide personal and professional network would be a source of support for growing the research fund, which she hoped would push beyond the $1 million target.

Jake is really loved in the city of Boston, she said. Everybodys come out this year to help us, knowing the family struggles with this gene.

In Worcester, researchers have reason to be optimistic. Laboratory studies on animals have shown some positive findings. Several drugs are in the pipeline.

Its very easy to be passionate about the research, Zack said Thursday, while working in the lab at UMass. I hope to be part of the drug that will end a lot of family suffering.

He said that with the advent of gene therapy, basic research is accelerating and more therapies are coming online.

There are 30 to 40 gene mutations linked to ALS.

About 10% of ALS cases are inherited, Zack said. Advances made in genetic research could also help address sporadic, or not-inherited ALS and other related neurological diseases, including some forms of dementia.

Zack was working on defects in one gene in particular, C9ORF72.

Were currently working to knock down that gene and silence it, reduce its expression, he said.

If researchers can alter the gene and deliver it into the spinal cord or brain, it could reprogram the processes leading to cell death. Delivering the genetic therapy safely and effectively to the right place also brings another set of challenges.

All these things are big problems were tackling here, Zack said.

He believed that a drug could be developed within five years that could significantly slow down, if not stop, the progression of ALS.

Im honored to be on the team thats on the vanguard of developing these drugs, he said.

Knowing his familys strong history of ALS, Zack said the weight of trying to find a cure was heavy, but one he gladly takes on.

He has been tested for the genetic mutation linked to ALS and learned he does not have it. But he worries about those who might.

Still, he said, it was also wonderful to be in a position where I can help. I do feel very lucky to be in this position.

The can-do spirit to fight ALS is another outgrowth of the generosity shown by the Kennedys when they founded Christmas in the City for homeless children in Boston 30 years ago.

Zack recalled helping to prepare gifts and activities for what has grown to 6,000 children at a family fun extravaganza hosted by volunteers at the Seaport convention center.

His parents started the program with a small group of volunteers for 165 homeless kids, he said, when he and his siblings were young, and they would get so many presents from the large extended Kennedy family.

My parents wanted to make sure we knew what it was really about, Zack said.

Jakes brother and business partner, Richard, serves as president of the Angel Fund for ALS Research, which is also dedicated to supporting ALS research at UMass Medical School.

Together with a $1 million grant from the ALS Association, funded by proceeds from the Ice Bucket Challenge, UMass Medical School has received more than $2.1 million in private contributions toward ALS research over the last few years, on top of government-funded grants, according to a medical school spokeswoman.

Jake is planning to run the Boston Marathon, his 38th, in April. He has met the qualifying time standard for entry but he said he would also be raising money for the ALS fund.

My speech is going downhill, but physically Im fine, he said.

Hes excited and upbeat about the future for biomedical research to find a cure for ALS. And despite knowing the devastating path that confronts those with the disease, he doesnt feel sorry for himself.

Im the luckiest man who ever lived, Jake said.

He counted his blessings of his wife, his children, his family, his job and Christmas in the City.

He said, That will never change.

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The race is on for ALS cure: Fund launched at UMass Medical School - GazetteNET

Searching for new methods of attacking serious central nervous system diseases, Biogen has signed a deal that could be run into the hundreds of millions of dollars to essentially license a transcriptional map of the brain and scan it for new drug targets.

The partnership, worth $15 million upfront and far more in milestones, is with a new bioinformatics startup called CAMP4 Therapeutics. Founded in 2018 year by the Whitehead Institutes Richard Young and Harvard Medical Schools Leonard Zon, CAMP4 takes genes considered affiliated with a disease and maps out the various ways cells express those genes and turn them on or off. They then take that map and in the biological equivalent of stretching a war map across a table in a bunker mark up all the different methods of attack.

The more we know about a gene, it doesnt mean we can drug that gene, CEO Josh Mandel-Brehm, who worked at Biogen before joining CAMP4, told Endpoints News. Many genes are not quote-un-quote druggable, so then we have an interesting problem: I think I understand the genetics of the disease, but how do I drug it? And thats where our map solves for this problem.

Alongside the $15 million upfront payment, CAMP4 will be eligible for up to $96 million in milestones for each of the initial targets it supplies, and up to $173 million for each subsequent one.

Biogen is not disclosing what genes or diseases they will target, but the collaboration will focus on microglial cells. These macrophages are found in the central nervous system and have long been considered a key factor in neurodegenerative diseases such as Alzheimers and Parkinsons.

Biogen will bring CAMP4 the genes it believes are central to diseases in the central nervous system, and CAMP4 will inform them all the ways the gene is being expressed and where Biogen might go about turning up or down the expression.

So you say hey I want to move this particular gene for this disease, how should I do that? I want to change the expression of it,' Mandel-Brehm said, describing how they work with partners. Well show you the different nodes, if you will: hey you can drug this, you can drug this, you can drug this. Its modality agnostic.

CAMP4, founded out of Polaris Partners, is one of a couple of new biotechs that build on a discovery Young had back in 2015. The longtime transcriptional biologist published work showing that cells modify gene expression using a series of insulated neighborhoods, 3-D loops of DNA that control gene expression within them. CAMP4 maps those (along with other aspects of the transcriptome). Flagship Pioneerings new Omega Therapeuticsalso tries to drug them.

Since launching in 2018, CAMP4 has done extensive work on mapping genes for liver diseases, including NASH, as part of an in-house development program. Theyve also partnered with synthetic RNAi company Alnylam and are working to expand their maps of cells in the brain, heart, muscles, immune system, blood and kidneys, which they could then license out or use to develop drugs in-house.

The way I think about the diseases were going after is BFP: Big Fucking Problems,Mandel-Brehm said, citing NASH and other diseases that lack effective treatments. And Its well-validated and understood: If youre drugging something thats tied to the gene, youre much more likely to be successful.

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Targeting the BFPs of the CNS, Biogen licenses map of genetic neighborhoods in the brain - Endpoints News