Archive for the ‘Gene Therapy Research’ Category

Recently, Sangamo Therapeutics (SGMO) announced an extensive collaboration with Biogen (BIIB) to develop gene therapies for neurological diseases, comprising of Alzheimer's disease and Parkinson's disease. Sangamo is to receive a $350M from an upfront payment and proceeds from a stock purchase. The Biogen collaboration will be added to an impressive list that comprises of other big-name players such as Pfizer (PFE) and Gilead (GILD). Sangamos partnerships and collaborations are one of my primary reasons for making an early investment in Sangamo, so, I am even more bullish on SGMO following a collaboration with one of the leading neurological companies in the world.

I intend to review the details of the Biogen deal and why I am even more bullish on SGMO. In addition, take a look at the companys current market valuation in order to defend a long-term investment. Finally, I take a look at the charts to lay out a plan for starting a position in SGMO.

The Biogen collaboration will utilize Sangamo's ZFP technology to control the expression of tau and alpha-synuclein genes that are thought to be responsible for Alzheimer's, Parkinson's, and 10 other neurological targets. In return, Sangamo is expected to receive a $125M upfront payment and $225M in proceeds from a stock purchase at $9.21 per share. In addition, Sangamo is eligible for up to $2.37B in other milestone payments and could claim "high-single-digit to sub-teen double-digit royalties on net sales."

In terms of responsibilities, Sangamo will be take on the early research activities and the development of the AAV delivery vectors and ZFP-TFs. Once Sangoma has finished their preclinical work, Biogen will take over and will take the programs through the regulatory pathway and commercialization.

This collaboration is a significant event for Sangamo for a number of reasons. Personally, I find it extremely bullish that one of the worlds leading neurological companies has tapped Sangamos technology to take on some of the worlds most notorious neurological diseases. In addition, the details of the collaboration show that Biogen is dedicated to Sangamo with an impressive financial commitment and is now one a large shareholder of the company.

If these neurological programs are successful, they could be the first products that actually slow or stop the progression of these diseases with a one-time treatment. Obviously, this would be a medical breakthrough and could change the way we treat some of the worst diseases known to man.

The Sangamo-Biogen collaboration will use the company's ZFP technology to develop genomic therapeutics that switch genes on and/or off in the brain that are responsible for neurological diseases. Sangamo has announced that the ST-501 program will target tau and the ST-502 program will target alpha-synuclein. These programs will employ Sangamo's zinc finger protein transcription factors ZFP-TFs, which is a genome regulation technology that uses adeno-associated viruses AAVs.

ZFP-TF has already shown some success in neurological diseases such as Huntington's and ALS. Sangamo presented preclinical data at the companys R&D Day, which showed that more than half of the ZFP-TFs tested reduced the total alpha-synuclein levels by greater than 50% in ex-vivo cell culture system. So, it looks as if ZFP-TF could address the underlying source, by repressing the genes expression at the DNA level with a one-time administration. As a result, I am feeling optimistic that Sangamo's differentiated ZFP-TF technology will show several benefits over other therapeutic approaches.

In my previous article, I discussed how Sangamo has partnerships and collaborations with some of the most distinguished biotech and pharmaceutical companies in the world onboard (Figure 1). Perhaps the most impressive collaborations are Sangamos hemophilia A gene therapy candidate, SB-525, being partnered with Pfizer (PFE). Sangamo transferred their SB-525 IND to Pfizer, which activated a $25M milestone payment from Pfizer. Now, SB-525 will be entering its late-stage of development and could be the companys first licensed product.

Figure 1: SGMO Partnerships (Source: SGMO)

In addition, the company has a partnership with Sanofi (SNY) for sickle cell disease and beta-thalassemia. Sangamo also has collaborations with Takeda Pharmaceuticals (OTCPK:TKPHF) and Gileads (GILD) Kite Pharma. It is important to note that these partners entered agreements with Sangamo at an early stage of development and were willing to agree to big financial commitments to Sangamo in order to get a piece of the action.

Thus far, Sangamo has brought in around $700M in license fees, milestones, and equity from these partnerships or collaborations. Sangamo could pull in up to a cumulative $6.34B in potential milestone payments from these partnerships.

Looking at the names in figure 1, we can say the company has a knack for attracting and closing major deals. Not only am I bullish about the details of these partnerships, but I also bullish about managements ability and willingness to sign big-name partners. Most developmental biotech/pharma companies would kill to get one of those names associated with their company and technology. Meanwhile, Sangamo has five big names willing to make major commitments before the company has marketed therapies. These partnerships should supply significant milestone payments that can be used to fund the companys impressive pipeline and OpEx. Looking at figure 2, we can see that Street analysts expect Sangamo to start collecting on these milestone payments and eventually royalties in the coming years (Figure 2).

Figure 2: Estimated Annual Earnings (Source: Seeking Alpha)

Currently, the SGMO is fairly valued for its expected 2020 annual revenue, however, it is considerably undervalued when looking at the companys estimated ~$550M annual revenue in 2025, which is a forward price-to-sales of 1.50x. The biotech industrys average price-to-sales of 5x, so we should expect the market to start to recognize the opportunity and the share price will start to be priced in line with the rest of the industry.

Can the company hit $550M? I would point to the estimated $6.5B in potential milestone payments. The company has only collected about $700M, which leaves over $5B of potential milestones to get dispersed in the coming years, therefore, $550M in revenue isnt impossible.

What is more, the company expects their near-term cash from Biogen, combined with the company existing cash to be sufficient to potentially get through Pfizer's first BLA filing of SB-525 for Hemophilia A and beyond. So, it possible that we have little-to-no dilution in the coming quarters.

This historic market sell-off has hammered small-cap biotechs and SGMO has not been immune. Admittedly, the charts are far from looking bullish, so I am waiting for SGMO to show some signs of support before hitting the buy button.

Figure 3: SGMO Daily (Source: Trendspider)

I am looking for a double or triple bottom to ensure a level of support has been formed. Once the stock is able to clear the high of the formation before committing to a buy. Although SGMOs valuation points to the buy, I am not going to bind my cash to a ticker that is being dragged down with the rest of the market.

Disclosure: I/we have no positions in any stocks mentioned, but may initiate a long position in SGMO over the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Original post:
Sangamo: Adding Another Partnership To The Bull Thesis - Seeking Alpha

SARASOTA, Fla. and NOVATO, Calif., March 16, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc.Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that it has dosed the first patient in its KIK-AS (Knockdown of UBE3A-antisense in Kids with Angelman Syndrome) study of GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS).

The Phase 1/2 open-label, multiple-dose, dose-escalating study will enroll 20 patients to evaluate the safety, tolerability, and potential efficacy of GTX-102 in pediatric patients with Angelman syndrome. This is the first investigational study testing an antisense oligonucleotide as a potential therapy to treat AS. Further details can be referenced at: https://clinicaltrials.gov/ct2/show/NCT04259281.

Today is an important milestone with the dosing of the first patient in the KIK-AS study, stated Dr. Scott Stromatt, GeneTxs Chief Medical Officer GTX-102 has the potential to address the underlying deficiency that causes Angelman syndrome and we are excited, grateful and humbled to be leading this scientific quest. We look forward to the results of this study and sharing them with the Angelman community.

The GeneTx team has achieved a tremendous accomplishment rapidly advancing this program into the clinic, and GTX-102 may one day provide patients with Angelman syndrome with the first targeted therapy and a potentially transformative option for this devastating disease, said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx.

Chicagos Rush University Medical Center is the first clinical site to begin enrolling patients in the KIK-AS study, with additional sites being planned in Boston, Cincinnati, Denver, Los Angeles, New York and Ottawa, Canada.

When I held the syringe with this investigational treatment in my hand to inject it for the first time, I thought about the scientific advances in genomic and molecular medicine to produce potential treatments that bring hope of changing the disease course and ameliorating severity of symptoms in those with Angelman syndrome, said Elizabeth Berry-Kravis, site principal investigator at Rush. This is an exciting time for the field of neurodevelopmental disorders as we embark on a path to understanding the outcomes of treatments directed at correcting the underlying molecular causes of disease.

Pending additional site activation, GeneTx Biotherapeutics expects to report preliminary data from the first cohorts in the study in early 2021.

About Angelman Syndrome

Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is typically not inherited but instead occurs spontaneously.

Individuals with Angelman syndrome have developmental delay, balance issues, motor impairment, and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age.

About GTX-102

GTX-102 is an investigational antisense oligonucleotide designed to target and inhibit expressionof UBE3A-AS. Nonclinical studies show that GTX-102 reduces the levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the CNS. Reactivation of paternal UBE3A expression in animal models of Angelman syndrome has been associated with improvements in some of the neurological symptoms associated with the condition. GTX-102 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA). InAugust 2019, GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx.

About GeneTx Biotherapeutics

GeneTx Biotherapeutics LLC is a startup biotechnology company singularly focused on developing and commercializing a safe and effective antisense therapeutic for the treatment of Angelman syndrome. GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. GeneTx licensed the rights to antisense technology intellectual property from The Texas A&M University System in December 2017.

AboutUltragenyx Pharmaceutical Inc.

Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyxs strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the Company's website atwww.ultragenyx.com.

Ultragenyx Forward-Looking Statements

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our partnership with GeneTx, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with theSecurities and Exchange CommissiononFebruary 14, 2020, and its subsequent periodic reports filed with theSecurities and Exchange Commission.

Contacts:

GeneTxPaula Evans630-639-7271Paula.Evans@GeneTxBio.com

Ultragenyx Investors & MediaDanielle Keatley415-475-6876dkeatley@ultragenyx.com

See the original post here:
GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome - BioSpace

[Editors note: 7 Biotech Stocks to Buy and Hold in 2020 was previously published in December 2019. It has since been updated to include the most relevant information available.]

If it were not for the surge in stock prices in the fourth quarter, biotechnology stocks would have ended the year in double-digit losses. Instead, they had a banner end of the year and now are suffering or succeeding based on the coronavirus and broad market pressures.

At a macroeconomic level, markets now believe the government regulators will not scrutinize drug pricing. The government is starting to realize that high healthcare costs are not due solely to rising drug prices. So, biotech companies that raise prices to offset higher research and development costs may do so in 2020.

Mega mergers in 2019, like AbbVie (NYSE:ABBV) buying Allergan (NYSE:AGN), signaled the undervaluation in the sector. In 2020, price-to-earnings valuations may expand to correct the market discount.

There are seven biotechnology stocks in 2020 that investors should hone in on. Get ready to pop these in your portfolio.

Source: Pavel Kapysh / Shutterstock.com

Biogen (NASDAQ:BIIB) rose from $220 to $300 in October when the company said it would resume filing for approval to bring its Alzheimers therapy drug to market. Then on Dec. 6, the company presented Phase 3 results for Aducanumab.

Over an 18-month period, it enrolled 3,285 patients over the two studies. The results showed that with a high dose over a long period of time, there was a meaningful slowing of decline in Alzheimers patients. And so, if the drug passes regulatory review, BIIB stock will live up to its hype.

The Aducanumab data showed mixed results. Although a higher dosage benefited patients, the side effects on the higher dosage are not clear.

Biogen reported steady revenue growth in the third quarter. Revenue grew 5% to $3.6 billion as earnings per share grew 17% to $8.39 (on a GAAP basis). Its multiple sclerosis drug portfolio is resilient. But Biogen is focused on addressing the intellectual property challenge with Tecfidera, a drug that treats relapsing forms of MS. This is offset by the launch of Vumerity, a drug also used to treat people with relapsing forms of MS.

Biogen reported revenue for Spinraza growing in the double-digits year-over-year and quarter-over-quarter. This drug treats patients with spinal muscular atrophy (SMA). Since Spinraza has a well-characterized safety profile, Biogen will run further studies to evaluate the benefits of higher doses to achieve greater efficacy.

Analysts who offer a price target on BIIB stock have an average of a $306.75 price target.

Source: Michael Vi / Shutterstock.com

Amgen (NASDAQ:AMGN) trades with analyst price targets that average $248. But at a trailing P/E of 18.6 times, the companys Otezla acquisition from Celgene (NASDAQ:CELG) is accretive to full-year results.

Last year Amgen announced a collaboration with BeiGene (NASDAQ:BGNE). Forming collaborations are in Amgens growth strategy. Since 2011, it grew by expanding to 100 countries, including China and other emerging markets. BeiGene, which represents a strategic investment in China, offers strong oncology expertise. It also has good commercial and clinical capabilities.

In 2020 and beyond, the Amgen-BeiGene collaboration may accelerate the commercialization of Amgens approved oncology products in the region. It paid $2.7 billion for a 20.5% equity stake, which was a 36% premium to BeiGenes 30-day average share price.

In Q3, Amgen reported EPS of $3.66. Revenue declined 2.9% to $5.7 billion. Despite a global sales decline, Amgen reported double-digit sales for a multitude of drugs. If generic competition lessens in 2020 and drug pricing improves, Amgen stock could continue trending higher.

Source: Casimiro PT / Shutterstock.com

Gilead Sciences (NASDAQ:GILD) surged early as a coronavirus darling before getting sucked back down into range by the prevailing market forces. And as investors wait for the stock to build an uptrend, they may collect a dividend that yields around 4%.

The U.S. approved Gileads Descovy for PrEP (pre-exposure prophylaxis). This drug is indicated to reduce the risk of sexually acquired HIV-1. Filgotinib was validated in the European Union and the company submitted a new drug application in Japan. Filgotinib is a JAK1 inhibitor that treats rheumatoid arthritis. If approved, it will compete with AbbVies (NYSE:ABBV) Humira.

Setting aside Gileads viability as a coronavirus vaccine leader, investors should consider Gilead for 2020 because of its strong cash flow generation. It produced $2.6 billion in cash from operations in the third quarter and now has $25.1 billion in cash and investments.

That healthy cash flow allowed the company to easily spend on R&D. It paid $5.5 billion for global research collaboration activities and it also invested in Galapagos (NASDAQ:GLPG). Galapagos gives Gilead access to many compounds, including six molecules that are in clinical trials. For example, Gilead gains rights to a Phase 3 candidate that treats idiopathic pulmonary fibrosis. GLPG1972 is a Phase 2b candidate that treats osteoarthritis.

Gilead could grow its revenue beyond the 12.7% rate. If its purchase of Kite Pharmaceuticals yields new products brought to market, the stock is set to blast off in late 2020 and beyond.

Source: Shutterstock

Regeneron (NASDAQ:REGN), which is getting better known for its blockbuster drug Dupixent, broke out of a downtrend late last year and hasnt stopped growing.

The company reported an EPS of $6.67 as revenue soared 23.1% year-over-year to $2.1 billion. Markets incorrectly worried over generic pressure on Eylea, which treats advanced wet age-related macular degeneration. Eylea sales grew 14% to $1.9 billion.

Dupixent has a growing addressable market. As it gains approvals worldwide, sales might even accelerate. In 2020, Regeneron will continue investing in researching activities for the drug to have additional indications.

For example, late this year it will have data in combination with Aimmunes (NASDAQ:AIMT) AR101 for treating peanut allergies. It will also have a data readout for its interleukin-33 (IL-33) antibody called Regeneron 3500 for treating atopic dermatitis and COPD (chronic obstructive pulmonary disease).

Regeneron continues to develop drugs in the oncology market. It launched Libtayo, an anti-PD-1 therapy that treats cutaneous squamous cell carcinoma. It also has new data from Libtayo in treating lung cancer.

At a P/E near 20 times, Regeneron still has plenty of upside potential. Even if the companys revenue were to slow to the 4% range in the next five years, the stock is still worth around $526.

Source: Shutterstock

Crispr Therapeutics (NASDAQ:CRSP), which is based in Switzerland, almost doubled in 2019. So after that performance, why should investors expect any more upside in 2020?

CRSP reported encouraging results for a potential immune-evasive cell replacement therapy for diabetes. From its press release, it said: The data demonstrate that the CyT49 pluripotent stem cell line, which has been shown to be amenable to efficient scaling and differentiation, can be successfully edited with CRISPR.

The results are further proof that regenerative medicine and gene editing may lead to cures in various diseases. Crispr is currently focusing on chronic diseases like diabetes.

Optimism for CRSP stock is so strong that a 4.3 million stock offering at $64.50 barely hurt the stock. The stock sale will add $274.1 million of cash, which Crispr may use to fund its ongoing clinical studies in sickle cell disease and beta-thalassemia.

Digging into the details, Crispr said that it successfully treated its first patient in the CLIMB SCD-121 study. The neutrophil engraftment had 46.6% hemoglobin F four months later. That suggests a curative response. Since the study is ongoing, the company will continue to inform investors of the safety profile of the treatment and its efficacy.

Crispr is ushering in an innovative form of therapy through gene editing. And markets like what the future holds.

Source: Shutterstock

Editas Medicine (NASDAQ:EDIT) has negligible revenue and is losing money, but its collaboration with Celgene gives it the resources in advancing its pipeline.

Editas reported a $70 million payment from its Celgene collaboration. This payment is in recognition of the work it did so far. It also includes contributions it will make to the collaboration. Its first patient will receive a doss of EDIT-101 by early 2020. This medicine treats subjects suffering from LCA10 or Leber congenital amaurosis. In Q3, Editas presented data for its USH2A study. It produced up to 60% corrected gene expression.

Looking ahead, additional collaborations with Celgene could give EDIT stock a lift. On its conference call, the company said:

All of that work is rolled into the new collaboration with Celgene. As you can imagine, as a leader and we believe the leader in T cell medicines for oncology including arguably the best CD19 and the best BCMA CAR-T programs that have a strong interest in maintaining their leadership position and gene editing is certainly an important part of what are likely to be the next generation of T cell medicines for oncology.

Editas collaboration with Celgene continues to progress nicely. Wall Street, which has a $42.50 price target, is also optimistic that the company will reward investors in 2020.

Source: Shutterstock

Innoviva (NASDAQ:INVA) has been trading sidewas all year until the recent mass-sell-off.

Last year, Innoviva booked $69.2 million in gross royalty revenues from GlaxoSmithKline(NYSE:GSK). This included royalties of $46.4 million connected to sales of Relvar/Breo Ellipta. This drug is a combination inhaled corticosteroid that treats patients with COPD (chronic obstructive pulmonary disease). Anoro Ellipta, which treats COPD and is taken once daily, brought in royalties of $11.6 million.

The company had $2.8 million in legal and related fees. Theravance Biopharma (NASDAQ:TBPH) initiated an arbitration. In the final decision, the Theravance Respiratory Company would reimburse those legal costs.

Global Relvar/Breo Ellipta sales fell 10% globally and by 32% in the U.S. Pricing discounts hurt revenue, and that revenue was partially offset by volume growth. Market share gains in various European markets and in Japan offset the overall revenue declines. Anoro Ellipta net sales grew 18% globally and were up 17% in the U.S. The negative impact of higher rebates was offset by higher sales volumes.

For 2020, Innoviva has a lower operating cost basis, helped by ending its Brisbane office lease. So, if the company reduces rebate offers and continues to grow its sales volume, revenue and profits may rebound.

As of this writing, Chris Lau held shares of INVA.

Here is the original post:
7 Biotech Stocks to Buy and Hold in 2020 - Investorplace.com

HOUSTON--(BUSINESS WIRE)--Kuur Therapeutics today announced the launch of its new business in partnership with Baylor College of Medicine and Baylors Center for Cell and Gene Therapy. Houston-based Kuur Therapeutics will advance the work of its predecessor, Cell Medica, to develop anti-cancer therapies using its innovative chimeric antigen receptor natural killer T cell (CAR-NKT) therapy platform.

IP Group, Baylor College of Medicine and Schroeder Adveq are investing to support two phase 1 studies: GINAKIT 2 (autologous CAR-NKT cells in neuroblastoma) and ANCHOR (allogeneic CAR-NKT cells in CD19 malignancy). The ongoing GINAKIT2 study is now enrolling patients at the third dose level and the ANCHOR study IND has recently been approved by the FDA, with first patient treatment expected in 1H 2020. The funding will also support the preclinical development of an allogeneic CAR-NKT product for treatment of hepatocellular carcinoma, ahead of an IND submission anticipated in 1H 2021.

In conjunction with the new investment, Kevin S Boyle, Sr, was named CEO of Kuur, succeeding Chris Nowers. Mr Boyle joined Cell Medica as CFO in February 2018. Kevin previously held senior finance roles at both NASDAQ-listed and private equity backed companies. He is an accomplished capital markets professional, having raised over $2.0 billion in equity and debt capital.

Kuurs novel CAR-NKT platform is a next-generation technology of engineered immune cells with enhanced functions for the treatment of hematological and solid tumors. It utilizes the unique properties of NKT cells, a specialized type of innate lymphocyte, which shares properties with both T and NK cells. This platform, developed in the laboratory of Baylor Principal Investigator Leonid Metelitsa, is exclusively licensed to Kuur by its partner and collaborator, Baylor College of Medicine.

Annalisa Jenkins, Chair of Kuurs Board, said: We are fortunate to have Kevin step up to the role of CEO. He will act as a change agent, leading the company during a crucial period for our clinical trials and working to secure the additional capital required to progress our two lead CAR-NKT products through the clinic.

The Board would also like to thank Chris for his exceptional leadership during a transition period that has resulted in a company on the right path forward for its investors, with a focus on its important collaborations.

Kevin S Boyle, Sr, Kuurs CEO, said: I am excited to lead Kuur Therapeutics at such a pivotal moment. We are making final preparations to take our off-the-shelf program into the clinic and believe the allogeneic approach holds huge promise for unlocking the potential of CAR therapies for large patient populations. Compared with patient-specific autologous CAR products, it is immediately available for treatment and less expensive to manufacture.

Leonid S. Metelitsa, BCM Principal Investigator, said: My goal is to make a difference in the lives of cancer patients, especially children, and Im excited to be working with the Kuur team to make this goal a reality. I believe that the NKT-cell platform technology, developed in my laboratory and progressed to first-in-human clinical testing in close collaboration with colleagues at BCM and Texas Childrens Hospital, offers a unique route to next-generation off-the-shelf CAR therapies for a broad range of malignancies.

- ENDS -

Notes to Editors

About Kuur Therapeutics

Kuur Therapeutics, headquartered in Houston, is a clinical-stage biotechnology company focused on transforming the treatment of solid and hematological cancers by developing next generation chimeric antigen receptor-natural killer T cell (CAR-NKT) therapies. Developing a portfolio of primarily allogeneic therapies, the companys revolutionary platform spanning both hematological and solid tumors is being created in partnership with Baylor College of Medicine and Baylors Center for Cell and Gene Therapy.

For further information, please visit http://www.kuurtx.com. Follow Kuur Therapeutics on LinkedIn

About Kuurs CAR-NKT cell technology

One of the challenges with allogeneic therapies is that infusing a patient with donor-derived lymphocytes can induce graft versus host disease (GvHD), a potentially life-threatening condition in which the infused cells recognize the patients tissues as foreign. The NKT cells used in Cell Medicas CAR-NKT platform have an invariant T cell receptor (iTCR) that does not distinguish between self- and non-self tissues, making them unlikely to induce GvHD when given to another person. Kuurs CAR-NKT cells are also engineered to secrete IL-15, to prolong persistence and enhance anti-tumor activity.

Baylor has previously reported evidence of clinical activity and a good safety profile in neuroblastoma patients treated at low dose levels of autologous CAR-NKT cells at the 2019 American Society for Gene and Cell Therapy conference.

About Baylor College of Medicine

Baylor College of Medicine (www.bcm.edu) in Houston is recognized as a premier academic health sciences center and is known for excellence in education, research and patient care. It is the only private medical school in the greater southwest and is ranked 16th among medical schools for research and 5th for primary care by U.S. News & World Report. Baylor is listed 21st among all U.S. medical schools for National Institutes of Health funding and number one in Texas. Located in the Texas Medical Center, Baylor has affiliations with seven teaching hospitals and jointly owns and operates Baylor St. Lukes Medical Center, part of CHI St. Lukes Health. Currently, Baylor trains more than 3,000 medical, graduate, nurse anesthesia, physician assistant and orthotics students, as well as residents and post-doctoral fellows.

Follow Baylor College of Medicine on Facebook (http://www.facebook.com/BaylorCollegeOfMedicine) and Twitter (http://twitter.com/BCMHouston).

About the Baylor College of Medicine Center for Cell and Gene Therapy

The Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital, and Houston Methodist Hospital in Houston, Texas is led by Helen E Heslop, MD, DSc (Hon), Director and Malcolm K Brenner, MD, PhD, Founding Director. The Center for Cell and Gene Therapy provides an infrastructure to rapidly translate novel cell and gene therapy protocols from the laboratory to the clinic. The comprehensive approach of the center brings a wide variety of scientists and clinicians together to develop strategies for the treatment of cancer, HIV and cardiovascular disease. Patient facilities include the adult stem cell transplant unit at Houston Methodist Hospital and the pediatric stem cell transplant unit at Texas Children's Hospital.

Go here to see the original:
Kuur Therapeutics Launches to Develop and Commercialize Off-the-shelf CAR-NKT Cell Therapies Targeting Hematological and Solid Tumors - Business Wire

Several government organizations and private institutions are investing in the regenerative medicine industry, for research and development. New development is undertaken as per the rules and regulations standardized by organizations like Food and Drug Administration (FDA) in the USA, the European Medicines Agency (EMA) in Europe and other regional authorities.

Request a sample of this report @https://www.adroitmarketresearch.com/contacts/request-sample/52

From being able to identify and heal life threatening diseases, to their prevention, the health and medicine sector has witnessed a revolutionary development over the years, with regenerative medicine being a major innovation.

The average life expectancy of a human being has increased, thus giving rise to the geriatric population. That, coupled with the increasing number of neurodegenerative, orthopedic, oncological, genetic and chronic disorders, constitutes the major factors that are fuelling the growth of the regenerative medicine market size. It has provided a tremendous opportunity to manufacturers, to satisfy this demand of the market. They are collaborating with leading research specialists, labs, pharmaceutical and biotechnology companies, and investors, towards the invention of more regenerative medicines, like gene therapy, nanotechnology, and tissue engineering. The regenerative medicine industry is evolving as one of the most cost-effective and definitive healthcare solutions. Regenerative medicine product analysis shows cell therapy fetching the highest revenue in the forecast period followed by tissue engineering, gene therapy and small molecules and biologics.

Browse the complete report @https://www.adroitmarketresearch.com/industry-reports/regenerative-medicine-market

North America is expected to hold the highest regenerative medicine market share which invests in oncology and stem cell research. Presence of organizations such as National Cancer Institute, Alliance for Regenerative Medicine, National Institutes of Health and International Society for stem cell research supports the research and development of regenerative medicine which is accelerating the growth in this region. The Asia Pacific region is expected to witness the fastest growth during the forecast period, owing to the population growth in this region. Countries like China and Japan are introducing flexible regulations towards the clinical adoption of regenerative medicine techniques. The government is also actively participating in funding towards the infrastructure required for the research and development. Thus, boosting the global regenerative medicine market further.

What to expect from the upcoming report on Global Regenerative Medicine Market:

Growth projections of the global regenerative medicine market during the forecast period (2018-2025).

-Areas of growth and concern in the field of regenerative medicine.

Economic factors likely to influence the market.

Trends, drivers, opportunities, restraints, and challenges in the development and use of regenerative medicine.

The involvement of government and other private organizations that are elevating the research and development of regenerative medicine.

Analysis of the regional demographics affecting the market.

Information about the competitors in the market, and the initiatives taken by them to improve this market.

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Regenerative Medicine Market 2020 Global Share, Growth, Size, Opportunities, Trends, Regional Overview, Leading Company Analysis, And Key Country...

New research has found that there are 362 cell and gene therapies in clinical pipelines in the US, an increase from 2018.

A new report from Americas Biopharmaceutical Companies has revealed that there are 362 cell and gene therapies in development in the US. Roughly a third of the therapies, 132, are potential treatments for rare diseases.

The research also highlights that the rate of R&D in this field is growing, as in 2018, a Pharmaceutical Research and Manufacturers of America (PhRMA) report on the cell and gene therapy pipeline found 289 therapies in clinical development in the US.

There are currently nine cell or gene therapy products approved by the US Food and Drug Administration (FDA).

Cell and gene therapies represent two overlapping fields of biomedical research with similar aims, which target DNA or RNA inside or outside the body. Gene therapies use genetic material, or DNA, to alter a patients cells and treat an inherited or acquired disease, whereas cell therapy is the infusion or transplantation of whole cells into a patient for the treatment of an inherited or acquired disease.

According to the report, the novel cell and gene therapies range from early to late stages of clinical development and are focused on a variety of diseases and conditions from cancer, genetic disorders and neurologic conditions.

Some of the cell and gene therapies in the pipeline include:

Another finding highlighted by the report is the 60 RNA therapeutics in development. Whilst not a kind of cell or gene therapy,RNA interference (RNAi) and antisense RNA use a genes DNA sequence to turn it off or modify the gene expression. So, these treatments can potentially inhibit the mechanism of disease-causing proteins.

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Almost 400 cell and gene therapies in US pipeline, finds report - European Pharmaceutical Review

Barth syndromeis a rare metabolic disease caused by mutation of a gene calledtafazzinorTAZ. It can cause life-threatening heart failure and also weakens the skeletal muscles, undercuts the immune response, and impairs overall growth. Because Barth syndrome is X-linked, it almost always occurs in boys. There is no cure or specific treatment.

In 2014, to get a better understanding of the disease,William Pu, MD, and colleagues at Boston Childrens Hospital collaborated with the Wyss Institute to create a beatingheart on a chip model of Barth syndrome. The model used heart-muscle cells with theTAZmutation, derived from patients own skin cells.It showedthatTAZis truly at the heart of cardiac dysfunction: the heart muscle cells did not assemble normally, mitochondria inside the cells were disorganized, and heart tissue contracted weakly. Adding a healthyTAZgene normalized these features, suggesting that gene replacement therapy could be a viable treatment.

But to fully capture Barth syndrome and its whole-body effects, Pu and colleagues needed an animal model. The animal model was a hurdle in the field for a long time, says Pu, director of Basic and Translational Cardiovascular Research at Boston Childrens and a member of the Harvard Stem Cell Institute. Efforts to make a mouse model using traditional methods had been unsuccessful.

As described in the journalCirculation Research, most mice with the whole-bodyTAZdeletion died before birth, apparently because of skeletal muscle weakness. But some survived, and these mice developed progressive cardiomyopathy, in which the heart muscle enlarges and loses pumping capacity. Their hearts also showed scarring, and, similar to human patients with dilatedcardiomyopathy, the hearts left ventricle was dilated and thin-walled.

Mice lackingTAZjust in their cardiac tissue, which all survived to birth, showed the same features. Electron microscopy showed heart muscle tissue to be poorly organized, as were the mitochondria within the cells.

Pu, Wang, and colleagues then used gene therapy to replaceTAZ, injecting an engineered virus under the skin (in newborn mice) or intravenously (in older mice). Treated mice with whole-bodyTAZdeletions were able to survive to adulthood.TAZgene therapy also prevented cardiac dysfunction and scarring when given to newborn mice, and reversed established cardiac dysfunction in older mice whether the mice had whole-body or heart-onlyTAZdeletions.

Thats where the challenge will lie in translating the results to humans. Simply scaling up the dose of gene therapy wont work: In large animals like us, large doses risk a dangerous inflammatory immune response. Giving multiple doses of gene therapy wont work either.

The problem is that neutralizing antibodies to the virus develop after the first dose, says Pu. Getting enough of the muscle cells corrected in humans may be a challenge.

Another challenge is maintaining populations of gene-corrected cells. While levels of the correctedTAZgene remained fairly stable in the hearts of the treated mice, they gradually declined in skeletal muscles.

The biggest takeaway was that the gene therapy was highly effective, says Pu. We have some things to think about to maximize the percentage of muscle cell transduction, and to make sure the gene therapy is durable, particularly in skeletal muscle."

Reference: Wang et al. (2020).AAV Gene Therapy Prevents and Reverses Heart Failure in A Murine Knockout Model of Barth Syndrome.Circulation Research.https://www.ahajournals.org/doi/abs/10.1161/CIRCRESAHA.119.315956.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Gene Therapy Reverses Heart Failure in Mouse Model - Technology Networks

News Release

Monday, March 9, 2020

New strategy could be applied to other infectious diseases.

A new approach to direct the body to make a specific antibody against HIV led to sustained production of that antibody for more than a year among participants in a National Institutes of Health clinical trial. This drug-delivery technology uses a harmless virus to deliver an antibody gene into human cells, enabling the body to generate the antibody over an extended time. With further development, such a strategy could be applied to prevent and treat a wide variety of infectious diseases, according to the study investigators.

Researchers from NIHs National Institute of Allergy and Infectious Diseases (NIAID) reported the findings on March 9 in an oral presentation at the 2020 Conference on Retroviruses and Opportunistic Infections (CROI).

Antibodies are immune system proteins that help prevent or clear infections. Traditional vaccines induce the immune system to generate protective antibodies. Another approach to preventing infections is to deliver monoclonal antibodies preparations of a specific antibody designed to bind to a single target directly into people. Monoclonal antibodies also are used therapeutically, with many already approved for treating cancer, autoimmune diseases and other conditions and others being evaluated for treatment of infectious diseases, such as Ebola virus disease.

Administering proteins to people requires periodic injections or infusions to retain protective or therapeutic levels, which can be challenging, particularly in resource-limited settings. Delivery of antibody genes using a virus as a carrier, or vector, offers a potential alternative.

Monoclonal antibodies hold enormous promise for preventing and treating both established and emerging infectious diseases, said NIAID Director Anthony S. Fauci, M.D. Novel delivery platforms such as viral vectors could facilitate the future development and deployment of antibody-based prophylaxis and therapy, and these findings are a promising first step in that direction.

The drug-delivery system developed by scientists at NIAIDs Vaccine Research Center (VRC) uses adeno-associated virus serotype 8 (AAV8) to deliver an antibody gene. AAVs small viruses that do not cause disease in humans have proven to be safe, well-tolerated vectors for gene therapy. In a previous study in animal models, VRC researchers found that using AAV8 to deliver genes for antibodies against simian immunodeficiency virus (SIV), the monkey equivalent of HIV, led monkeys to safely produce high levels of anti-SIV antibodies and protected them from acquiring SIV.

Building on this preclinical work, researchers designed a Phase 1 clinical trial known as VRC 603. It aims to assess the safety and tolerability of an AAV8 vector carrying an anti-HIV antibody gene in adults living with well-controlled HIV, and to evaluate whether it could cause human cells to produce the antibody. The vector carries the gene for an anti-HIV monoclonal antibody called VRC07, which was originally isolated from the blood of a person with HIV.

VRC07 is a broadly neutralizing antibody (bNAb), meaning it can stop a wide range of HIV strains from infecting human cells in the laboratory. Other clinical studies are underway to determine whether bNAb infusionscan protecthumansfrom acquiring HIV. Scientists also are evaluating whether combinations of HIV bNAbs can suppress the virus in people living with HIV.

The CROI presentation by Joseph P. Casazza, M.D., Ph.D., principal investigator of VRC 603, described initial results from the first eight participants in the ongoing trial, which is being conducted at the NIH Clinical Center in Bethesda, Maryland. Each of these individuals, aged 30 to 60 years, received a single dose by intramuscular injection of one of three different dose levels of AAV8-VRC07. They continued taking daily antiretroviral therapy.

Following injection with AAV8-VRC07, all eight participants produced VRC07 at levels detectable in the blood. VRC07 production reached an early peak four to six weeks after injection, then decreased, and slowly began to increase again roughly 16 weeks after the injection. The researchers have monitored the five participants who received low or intermediate AAV8-VRC07 doses for one and a half to two years. For three of these five individuals, antibody levels one year after injection were higher than those observed at four to six weeks. The three volunteers who received the highest AAV8-VRC07 dose have so far been monitored for five months to one year. Two produced VRC07 at concentrations higher than those seen in the low and intermediate dose groups.

Study participants have not experienced any major side effects due to AAV8-VRC07. Some volunteers experienced transient mild tenderness at the injection site or mild muscle pain.

To the best of our knowledge, this marks the first time that an AAV-based technology to deliver an antibody gene has resulted in safe and sustained levels of that antibody in blood, said NIAID VRC Director John Mascola, M.D. We hope that further development of this technology will yield a drug-delivery strategy applicable to a broad range of infectious diseases.

Administration of monoclonal antibody-based therapies sometimes results in a persons immune system developing antibodies against the therapy. Only three of the eight VRC 603 participants developed antibodies against VRC07; it is not yet clear whether these anti-drug antibodies could reduce VRC07s ability to neutralize HIV. The VRC 603 participants HIV was kept under control with continued antiretroviral therapy during the trial.

The concentrations of VRC07 observed in the study participants were lower than the antibody concentrations observed in animal studies of the AAV8-based technology. The VRC researchers are analyzing data from VRC 603 to better understand the factors that determine how much bNAb is produced by human cells. They also are continuing to monitor the VRC 603 participants and to enroll new volunteers into the trial.

AAV8-VRC07 was developed by VRC scientists in collaboration with David Baltimore, Ph.D., of the California Institute of Technology and Alejandro Balazs, Ph.D., of the Ragon Institute of MGH, MIT and Harvard. AAV8-VRC07 was manufactured by the Clinical Vector Core of the Center for Cellular and Molecular Therapeutics at the Childrens Hospital of Philadelphia. More information about the VRC 603 trial is available on ClinicalTrials.gov using identifier NCT03374202.

NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

JP Casazzaet al. Durable HIV-1 antibody production in humans after AAV8-mediated gene transfer. Oral presentation at the 2020 Conference on Retroviruses and Opportunistic Infections (CROI). Presented March 9, 2020.

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Drug-delivery technology leads to sustained HIV antibody production in NIH study - National Institutes of Health

HORAMA, a clinical stage gene therapy company in ophthalmology, today announced that Rodolphe Clerval has been appointed as Chief Business Officer (CBO). Rodolphe has a strong track record of executing strategic partnerships, business development deals and financing efforts. He will be responsible for business and corporate development, supporting HORAMA in operating a portfolio of gene therapy programs in inherited retinal dystrophies.

We are delighted to welcome Rodolphe, who brings us solid international experience in business development and corporate development strategy, acquired in biotech and pharmaceutical companies. His precious skills will allow us to prepare the next steps and to optimize the potential of our gene therapy products in ophthalmic diseases, for which there is a strong and unmet medical need , says Christine Placet, CEO, HORAMA.

Rodolphe Clerval (44) has over 18 years international experience in the pharma and biotech industry. Prior to joining HORAMA, Mr. Clerval was co-founder and Chief Business Officer at Enterome. During his tenure at Enterome, he has executed over 15 transactions, including major industrial partnerships with Takeda, J&J, BMS, Abbvie and Nestle Health Sciences, totalizing over 100m in upfront and R&D payments and in equity investments. He was also actively involved in fundraising rounds. Rodolphe previously worked with TcLand Expression and Genzyme in business development, and with Natixis as sell-side equity analyst. He started his career at Aventis Animal Nutrition as a scientist.

Rodolphe graduated with a degree in Biochemical Engineering from Polytech Marseille and he is a Certified European Financial Analyst from EFFAS/SFAF.

"HORAMA is a remarkable company, combining top gene therapy research, clinical stage programs with a strong team of industry leaders. I am thrilled to join HORAMA and to support the company in delivering best-in-class therapies in inherited retinal dystrophies", said Rodolphe Clerval, CBO, Horama.

Gene therapy market (source: FiorMarkets and Grand View Research, Inc)

Gene therapy is being developed with an aim to treat rare conditions with limited or no treatment options.Genetic disorders occur due to gene mutations, which can result in incorrect protein synthesis. Gene therapy is used to introduce a healthy gene into cells to allow the synthesis of a functional protein. Growing awareness and acceptance of gene therapy for various disease treatments are favouring market growth.The global gene therapy market is estimated to reach $5.5 billion by 2026, while the global ophthalmology market is projected to grow to $43 billion by 2026 (April 2019 report issued by Grand View Research, Inc.).

About HORAMA

At HORAMA, we believe in gene therapy to treat a broad range of inherited disorders.Our focus is on Inherited Retinal Dystrophies with our lead clinical program targeting patients with PDE6B gene mutations, a condition which leads to progressive vision loss in children and adults leading to legal blindness.Our team is pushing the boundaries of gene therapy by advancing next generation delivery platforms that will improve effectiveness and coverage of gene transfer to address multiple diseases. For more information, please go to: http://www.horama.fr.

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Contacts

HORAMA Christine Placet c.placet@horama.fr

Press: ALIZE RP Caroline Carmagnol Tel: +33 (0)6 64 18 99 59caroline@alizerp.com

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HORAMA Strengthens Its Management Team With the Appointment of Rodolphe Clerval as Chief Business Officer (CBO) - Yahoo Finance

DetailsCategory: DNA RNA and CellsPublished on Friday, 13 March 2020 09:52Hits: 219

HAIFA, Israel I March 12, 2020 I HAIFA, Israel, March 12, 2020 - Pluristem Therapeutics Inc. (Nasdaq:PSTI) (TASE:PSTI), a leading regenerative medicine company developing a platform of novel biological products, today announced it has signed a collaborative agreement with the BIH Center for Regenerative Therapy (BCRT) and the Berlin Center for Advanced Therapies (BeCAT) at Charite University of Medicine Berlin to expand its existing framework and research agreement and conduct a joint project evaluating the therapeutic effects of Pluristems patented PLX cell product candidates for potential treatment of the respiratory and inflammatory complications associated with the COVID-19 coronavirus.

PLX cells are allogeneic mesenchymal-like cells that have immunomodulatory properties that induce the immune systems natural regulatory T cells and M2 macrophages, and thus may prevent or reverse the dangerous overactivation of the immune system. Accordingly, PLX cells may potentially reduce the fatal symptoms of COVID-19 induced pneumonia and pneumonitis. Previous pre-clinical findings of PLX cells revealed significant therapeutic effects in animal studies of pulmonary hypertension, lung fibrosis, acute kidney injury and gastrointestinal injury which are potential complications of the severe COVID-19 infection. Clinical data using PLX cells demonstrated the strong immunomodulatory potency of PLX cells in patients post major surgery. Taken together, PLX cells potential capabilities with the safety profile observed from clinical trials involving hundreds of patients worldwide potentially position them as a therapy for mitigating the tissue-damaging effects of COVID-19.

The collaboration with Charit researchers will allow us to expedite our program to potentially enable the use of PLX cells to treat patients infected with COVID-19 that have respiratory and immunological complications. The fact that PLX is available off-the-shelf, combined with our ability to manufacture large scale quantities, is a key advantage in case a large number of patients may need respiratory support. The primary target is to prevent the deterioration of patients towards Acute Respiratory Distress Syndrome (ARDS) and sepsis. We intend to start the joint collaboration immediately, with an aim to bringing much needed treatment to a rapidly expanding global health threat, stated Yaky Yanay, Pluristem President and CEO.

Prof. Hans-Dieter Volk, Director of the BCRT at Charite University Medicine Berlin, commented, Through our long-term collaboration with Pluristem, we have a thorough understanding of PLX cells and their mechanism of action. Charites unique knowledge, which includes research and clinical expertise in the immunopathogenesis of viral infections and critically ill patients, provides us an accelerated framework in which we believe PLX cells can be explored as a potential therapy for patients infected with COVID-19.

About BIH Center for Regenerative TherapiesThe BIH Center for Regenerative Therapies (BCRT) is a cooperative translational research institution of the Charit University Hospital in Berlin and the Berlin Institute of Health (BIH). The mission of the BCRT is to develop a translational platform for Regenerative Therapies from bench-to-bedside. The clinical platforms -- Immune, muskuloskleletal, and cardiovascular system -- are cross-linked by cross-field clinical fields (cachexia/sarcopenia, genetic diseases) and technology and translation support platforms. There are extended experiences in clinical trials with cell therapy, including phase 1-3 trials with PLX cells.

About Berlin Center for Advanced Therapies (BeCAT)The Berlin Center for Advanced Therapies is a spin-off of the BCRT focusing on translation of cell and gene therapies in the major research fields of regenerative medicine and cancer. It consists of four research fields (endogenous regeneration, tissue engineering, anti-cancer immunotherapy, and rare diseases) and three technology platforms (manufacturing, product characteristics and biomarker, and clinical development and regulatory affairs.

About Pluristem TherapeuticsPluristem Therapeutics Inc. is a leading regenerative medicine company developing novel placenta-based cell therapy product candidates. The Company has reported robust clinical trial data in multiple indications for its patented PLX cell product candidates and is currently conducting late stage clinical trials in several indications. PLX cell product candidates are believed to release a range of therapeutic proteins in response to inflammation, ischemia, muscle trauma, hematological disorders and radiation damage. The cells are grown using the Company's proprietary three-dimensional expansion technology and can be administered to patients off-the-shelf, without tissue matching. Pluristem has a strong intellectual property position; a Company-owned and operated GMP-certified manufacturing and research facility; strategic relationships with major research institutions; and a seasoned management team.

SOURCE: Pluristem Therapeutics

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Pluristem and Charit University of Medicine Berlin Join Forces Targeting Potential Treatment for Respiratory and Inflammatory Intratissue...

Myst Therapeutics (Myst), a preclinical stage biotechnology company focused on developing selected TIL-based autologous T cell therapy products for cancer, announced the appointment of five leading experts in the areas of immunotherapy, tumor infiltrating lymphocytes, gene therapy, and oncology diagnostics to its Scientific Advisory Board.

"Myst is fortunate to attract these world-leading immunotherapy and cancer researchers to our advisory board," said TJ Langer, President and Chief Executive Officer of Myst. "Their experience and insight will be invaluable for the development and progression of our PuriT product pipeline."

James Mul, Ph.D. - Senior Member, Associate Center Director, and Michael McGillicuddy Endowed Chair in Melanoma Research/Treatment at Moffit Cancer Center. Dr. Mul has pioneered immune cell-based therapies and ushered them from the laboratory through Phase I and II clinical trials. He also leads work to engineer improvements to cell-based therapies through manipulation of tumor microenvironment and immune biological processes. Dr. Mul received his Ph.D. at the Fred Hutchinson Cancer Research Center, and fellowship training at the National Cancer Institute.

Michael Lotze, M.D. - Professor of Surgery, Immunology, and Bioengineering, University of Pittsburgh School of Medicine; Vice Chair of Research, Department of Surgery; Associate Director for Strategic Partnerships, University of Pittsburgh Cancer Institute; Asst. Vice Chancellor, UPSHS. Michael Lotze has been a forerunner in immune-oncology and gene therapy for three decades. He initiated the first gene therapies at the NIH and has treated over 100 patients at the University of Pittsburgh. He is a co-inventor on 10 patents, has authored over 500 scientific papers, and published numerous books and chapters on tumor biology and immunology. Dr. Lotz is the former CSO of Lion Biotechnologies (now Iovance Biotherapeutics).

Pamela Ohashi, Ph.D. - Co-Director of the Campbell Family Institute for Breast Cancer Research, Senior Scientist at the Princess Margaret Cancer Centre, Professor in the Departments of Medical Biophysics and Immunology at the University of Toronto, and the Director of the Tumor Immunotherapy Program at the Princess Margaret Cancer Centre. Dr. Ohashi is an expert on T cell behavior in the tumor microenvironment where her work has contributed to numerous Phase I and Phase II clinical trials. Dr. Ohashi is the author of over 150 scientific publications and has received numerous awards, including the American Association of Immunologists Pharmingen Investigator Award and the National Cancer Institute of Canadas William E. Rawls Award.

Chantale Bernatchez, Ph.D. - Associate Professor, Department of Melanoma Medical Oncology - Research, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX. Dr. Bernatchez is the director of the MD Anderson TIL Laboratory, where she leads the development of tumor infiltrating lymphocyte-based therapies. She currently supports a major Phase II clinical trial in Stage IV melanoma patients, the first of its kind outside of the National Cancer Institute, and is planning future trials utilizing novel techniques for promoting T cell survival and anti-tumor activity. Chantale received her Ph. D. from Laval University, Quebec and conducted postdoctoral training at both Laval University and MD Anderson Cancer center.

Alex Aravanis, M.D., Ph.D. - Dr. Aravanis is the Chief Scientific Officer, Head of R&D, and Co-Founder at GRAIL, the leader in early cancer detection diagnostics, and is commanding one of the largest clinical study programs in genomic medicine. Previously, Alex was Senior Director, R&D for Illumina, where he developed clinical assays for RNA and DNA analysis from fixed tissues, whole exome analysis, massively parallel single cell transcriptomics, and liquid biopsy using cell-free nucleic acids. Dr. Aravanis served as Vice President of Development and Chief Science Officer of Sapphire Energy Inc. and was a Co-Founder and Vice President of Engineering of Pria Diagnostics, LLC. Alex holds a PhD in Electrical Engineering, and an MD from Stanford University.

"These superb researchers are recognized for their significant discoveries and contributions in the field of tumor immunology and immunotherapy," stated James Mul, Ph.D., Associate Center Director and Michael McGillicuddy Endowed Chair in Melanoma Research and Treatment at Moffitt Cancer Center. "Their extensive experience in this field will be invaluable to Myst as the company advances its immunotherapy products to the marketplace."

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About the PuriT Platform

Clinical evidence has demonstrated that a patients' tumor infiltrating lymphocytes (TIL) recognize tumor specific molecules, traffic to the tumor, respond to tumor antigen, and selectively eradicate tumors. Clinical studies using these cells have shown durable objective clinical responses in patients with advanced metastatic disease, including refractory melanoma, breast, cervical, and colorectal cancer. Mysts PuriT platform builds on these successes by enriching and rejuvenating TIL to further improve responses. These encouraging results highlight PuriT as an emerging modality capable of providing personalized therapy options for patients spanning a broad spectrum of cancers.

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Myst TherapeuticsTJ Langertj@mysttherapeutics.com

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Myst Therapeutics Appoints Leading Cancer Immunotherapy Experts to Its Scientific Advisory Board - Yahoo Finance

Mar 12th 2020

BEING ABLE to see all the details of the genome at once necessarily makes medicine personal. It can also make it precise. Examining illness molecule by molecule allows pharmaceutical researchers to understand the pathways through which cells act according to the dictates of genes and environment, thus seeing deep into the mechanisms by which diseases cause harm, and finding new workings to target. The flip side of this deeper understanding is that precision brings complexity. This is seen most clearly in cancer. Once, cancers were identified by cell and tissue type. Now they are increasingly distinguished by their specific genotype that reveals which of the panoply of genes that can make a cell cancerous have gone wrong in this one. As drugs targeted against those different mutations have multiplied, so have the options for oncologists to combine them to fit their patients needs.

Cancer treatment has been the most obvious beneficiary of the genomic revolution but other diseases, including many in neurology, are set to benefit, too. Some scientists now think there are five different types of diabetes rather than two. There is an active debate about whether Parkinsons is one disease that varies a lot, or four. Understanding this molecular variation is vital when developing treatments. A drug that works well on one subtype of a disease might fail in a trial that includes patients with another subtype against which it does not work at all.

Thus how a doctor treats a disease depends increasingly on which version of the disease the patient has. The Personalised Medicine Coalition, a non-profit advocacy group, examines new drugs approved in America to see whether they require such insights in order to be used. In 2014, it found that so-called personalised medicines made up 21% of the drugs newly approved for use by Americas Food and Drug Administration (FDA). In 2018 the proportion was twice that.

Two of those cited were particularly interesting: Vitrakvi (larotrectinib), developed by Loxo Oncology, a biotech firm, and Onpattro (patisiran), developed by Alnylam Pharmaceuticals. Vitrakvi is the first to be approved from the start as tumour agnostic: it can be used against any cancer that displays the mutant protein it targets. Onpattro, which is used to treat peripheral-nerve damage, is the first of a new class of drugssmall interfering RNAs, or siRNAsto be approved. Like antisense oligonucleotides (ASOs), siRNAs are little stretches of nucleic acid that stop proteins from being made, though they use a different mechanism.

Again like ASOs, siRNAs allow you to target aspects of a disease that are beyond the reach of customary drugs. Until recently, drugs were either small molecules made with industrial chemistry or bigger ones made with biologynormally with genetically engineered cells. If they had any high level of specificity, it was against the actions of a particular protein, or class of proteins. Like other new techniques, including gene therapies and anti-sense drugs, siRNAs allow the problem to be tackled further upstream, before there is any protein to cause a problem.

Take the drugs that target the liver enzyme PCSK9. This has a role in maintaining levels of bad cholesterol in the blood; it is the protein that was discovered through studies of families in which congenitally high cholesterol levels led to lots of heart attacks. The first generation of such drugs were antibodies that stuck to the enzyme and stopped it working. However, the Medicines Company, a biotech firm recently acquired by Novartis, won approval last year for an siRNA called inclisiran that interferes with the expression of the gene PCSK9thus stopping the pesky protein from being made in the first place. Inclisiran needs to be injected only twice a year, rather than once a month, as antibodies do.

New biological insights, new ways of analysing patients and their disease and new forms of drug are thus opening up a wide range of therapeutic possibilities. Unfortunately, that does not equate to a range of new profitable opportunities.

Thanks in part to ever better diagnosis, there are now 7,000 conditions recognised as rare diseases in America, meaning that the number of potential patients is less than 200,000. More than 90% of these diseases have no approved treatment. These are the diseases that personalised, precision medicine most often goes after. Nearly 60% of the personalised medicines approved by the FDA in 2018 were for rare diseases.

Zolgensma is the most expensive drug ever brought to market.

That might be fine, were the number of diseases stable. But precision in diagnosis is increasingly turning what used to be single diseases into sets of similar-looking ones brought about by distinctly different mechanisms, and thus needing different treatment. And new diseases are still being discovered. Medical progress could, in short, produce more new diseases than new drugs, increasing unmet need.

Some of it will, eventually, be met. For one thing, there are government incentives in America and Europe for the development of drugs for rare diseases. And, especially in America, drugs for rare diseases have long been able to command premium prices. Were this not the case, Novartis would not have paid $8.7bn last year to buy AveXis, a small biotech firm, thereby acquiring Zolgensma, a gene therapy for spinal muscular atrophy (SMA). Most people with SMA lack a working copy of a gene, SMN1, which the nerve cells that control the bodys muscles need to survive. Zolgensma uses an empty virus-like particle that recognises nerve cells to deliver working copies of the gene to where it is needed. Priced at $2.1m per patient, it is the most expensive drug ever brought to market. That dubious accolade might not last long. BioMarin, another biotech firm, is considering charging as much as $3m for a forthcoming gene therapy for haemophilia.

Drug firms say such treatments are economically worthwhile over the lifetime of the patient. Four-fifths of children with the worst form of SMA die before they are four. If, as is hoped, Zolgensma is a lasting cure, then its high cost should be set against a half-century or more of life. About 200 patients had been treated in America by the end of 2019.

But if some treatments for rare diseases may turn a profit, not all will. There are some 6,000 children with SMA in America. There are fewer than ten with Jansens disease. When Dr Nizar asked companies to help develop a treatment for it, she says she was told your disease is not impactful. She wrote down the negative responses to motivate herself: Every day I need to remind myself that this is bullshit.

A world in which markets shrink, drug development gets costlier and new unmet needs are ceaselessly discovered is a long way from the utopian future envisaged by the governments and charities that paid for the sequencing of all those genomes and the establishment of the worlds biobanks. As Peter Bach, director of the Centre for Health Policy and Outcomes, an academic centre in New York, puts it with a degree of understatement: if the world needs to spend as much to develop a drug for 2,000 people as it used to spend developing one for 100,000, the population-level returns from medical research are sharply diminishing.

And it is not as if the costs of drug development have been constant. They have gone up. What Jack Scannell, a consultant and former pharmaceutical analyst at UBS, a bank, has dubbed Erooms lawEroom being Moore, backwardsshows the number of drugs developed for a given amount of R&D spending has fallen inexorably, even as the amount of biological research skyrocketed. Each generation assumes that advances in science will make drugs easier to discover; each generation duly advances science; each generation learns it was wrong.

For evidence, look at the way the arrival of genomics in the 1990s lowered productivity in drug discovery. A paper in Nature Reviews Drug Discovery by Sarah Duggers from Columbia University and colleagues argues that it brought a wealth of new leads that were difficult to prioritise. Spending rose to accommodate this boom; attrition rates for drugs in development subsequently rose because the candidates were not, in general, all that good.

Today, enthused by their big-science experience with the genome and enabled by new tools, biomedical researchers are working on exhaustive studies of all sorts of other omes, including proteomesall the proteins in a cell or body; microbiomesthe non-pathogenic bacteria living in the mouth, gut, skin and such; metabolomessnapshots of all the small molecules being built up and broken down in the body; and connectomes, which list all the links in a nervous system. The patterns they find will doubtless produce new discoveries. But they will not necessarily, in the short term, produce the sort of clear mechanistic understanding which helps create great new drugs. As Dr Scannell puts it: We have treated the diseases with good experimental models. Whats left are diseases where experiments dont replicate people. Data alone canot solve the problem.

Daphne Koller, boss of Insitro, a biotech company based in San Francisco, shares Dr Scannells scepticism about the way drug discovery has been done. A lot of candidate drugs fail, she says, because they aim for targets that are not actually relevant to the biology of the condition involved. Instead researchers make decisions based on accepted rules of thumb, gut instincts or a ridiculous mouse model that has nothing to do with what is actually going on in the relevant human diseaseeven if it makes a mouse look poorly in a similar sort of way.

But she also thinks that is changing. Among the things precision biology has improved over the past five to 10 years have been the scientists own tools. Gene-editing technologies allow genes to be changed in various ways, including letter by letter; single-cell analysis allows the results to be looked at as they unfold. These edited cells may be much more predictive of the effects of drugs than previous surrogates. Organoidsself-organised, three-dimensional tissue cultures grown from human stem cellsoffer simplified but replicable versions of the brain, pancreas, lung and other parts of the body in which to model diseases and their cures.

Insitro is editing changes into stem cellswhich can grow into any other tissueand tracking the tissues they grow into. By measuring differences in the development of very well characterised cells which differ in precisely known ways the company hopes to build more accurate models of disease in living cells. All this work is automated, and carried out on such a large scale that Dr Koller anticipates collecting many petabytes of data before using machine learning to make sense of it. She hopes to create what Dr Scannell complains biology lacks and what drug designers need: predictive models of how genetic changes drive functional changes.

There are also reasons to hope that the new upstream drugsASOs, siRNAs, perhaps even some gene therapiesmight have advantages over todays therapies when it comes to small-batch manufacture. It may also prove possible to streamline much of the testing that such drugs go through. Virus-based gene-therapy vectors and antisense drugs are basically platforms from which to deliver little bits of sequence data. Within some constraints, a platform already approved for carrying one message might be fast-tracked through various safety tests when it carries another.

One more reason for optimism is that drugs developed around a known molecule that marks out a diseasea molecular markerappear to be more successful in trials. The approval process for cancer therapies aimed at the markers of specific mutations is often much shorter now than it used to be. Tagrisso (osimertinib), an incredibly specialised drug, targets a mutation known to occur only in patients already treated for lung cancer with an older drug. Being able to specify the patients who stand to benefit with this degree of accuracy allows trials to be smaller and quicker. Tagrisso was approved less than two years and nine months after the first dose was given to a patient.

With efforts to improve the validity of models of disease and validate drug targets accurately gaining ground, Dr Scannell says he is sympathetic to the proposal that, this time, scientific innovation might improve productivity. Recent years have seen hints that Erooms law is being bent, if not yet broken.

If pharmaceutical companies do not make good on the promise of these new approaches then charities are likely to step in, as they have with various ASO treatments for inherited diseases. And they will not be shackled to business models that see the purpose of medicine as making drugs. The Gates Foundation and Americas National Institutes of Health are investing $200m towards developing treatments based on rewriting genes that could be used to tackle sickle-cell disease and HIVtreatments that have to meet the proviso of being useful in poor-country clinics. Therapies in which cells are taken out of the body, treated in some way and returned might be the basis of a new sort of business, one based around the ability to make small machines that treat individuals by the bedside rather than factories which produce drugs in bulk.

There is room in all this for individuals with vision; there is also room for luck: Dr Nizar has both. Her problem lies in PTH1R, a hormone receptor; her PTH1R gene makes a form of it which is jammed in the on position. This means her cells are constantly doing what they would normally do only if told to by the relevant hormone. A few years ago she learned that a drug which might turn the mutant receptor off (or at least down a bit) had already been characterisedbut had not seemed worth developing.

The rabbit, it is said, outruns the fox because the fox is merely running for its dinner, while the rabbit is running for its life. Dr Nizars incentives outstrip those of drug companies in a similar way. By working with the FDA, the NIH and Massachusetts General Hospital, Dr Nizar helped get a grant to make enough of the drug for toxicology studies. She will take it herself, in the first human trial, in about a years time. After that, if things go well, her childrens pain may finally be eased.

This article appeared in the Technology Quarterly section of the print edition under the headline "Kill or cure?"

Read more here:
New drugs are costly and unmet need is growing - The Economist

DUBLIN, March 13, 2020 /PRNewswire/ -- The "Nitric Oxide - Therapeutics, Markets and Companies" report from Jain PharmaBiotech has been added to ResearchAndMarkets.com's offering.

Share of drugs where NO is involved in the mechanism of action is analyzed in the worldwide pharmaceutical market for 2018 and is projected to 2023 and 2028 as new drugs with NO-based mechanisms are introduced into the market. Various strategies for developing such drugs are discussed.

Several companies have a product or products involving NO and free radicals. The report includes profiles of 35 companies involved in this area of which 9 have a significant interest in NO-based therapeutics. Other players are pharmaceutical and biotechnology companies as well as suppliers of products for NO research. Unfulfilled needs in the development of NO-based therapeutics are identified. Important 18 collaborations in this area are tabulated.

There are numerous publications relevant to NO. Selected 500 references are included in the bibliography. The text is supplemented with 26 tables and 30 figures. It is concluded that the future prospects for NO-based therapies are bright and fit in with biotechnology-based approaches to modern drug discovery and development. It is anticipated that some of these products will help in meeting the unfulfilled needs in human therapeutics.

The report contains information on the following:

The report describes the latest concepts of the role of nitric oxide (NO) in health and disease as a basis for therapeutics and development of new drugs. Major segments of the market for nitric oxide-based drugs are described as well as the companies involved in developing them.

Nitric oxide (NO) can generate free radicals as well as scavenge them. It also functions as a signaling molecule and has an important role in the pathogenesis of several diseases. A major focus is delivery of NO by various technologies. Another approach is modulation of nitric oxide synthase (NOS), which converts L-arginine to NO. NOS can be stimulated as well as inhibited by pharmacological and gene therapy approaches.

Important therapeutic areas for NO-based therapies are inflammatory disorders, cardiovascular diseases, erectile dysfunction, inflammation, pain and neuroprotection. The first therapeutic use of NO was by inhaltion for acute respiratory distress syndrome (ARDS). NO-donors, NO-mimics and NOS modulators are described and compared along with developmental status. NO-related mechanisms of action in existing drugs are identified.

Various pharmacological approaches are described along with their therapeutic relevance. Various approaches are compared using SWOT (Strengths, Weaknesses, Opportunities, Threats) analysis. NO-based therapies are compared with conventional approaches and opportunities for combination with modern biotechnology approaches are described.

For more information about this report visit https://www.researchandmarkets.com/r/m3rdb1

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Nitric Oxide Industry Outlook to 2028 - Pathways, Physiology, Disease, Pharmacology, Therapeutic Applications, Drugs, Therapy Markets, Companies -...

Tim Kelly, PhD

Tim Kelly joins AskBio to lead manufacturing of clinical- and commercial-scale AAV vectors to be used in gene therapy.

RESEARCH TRIANGLE PARK, N.C., March 12, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio), a leading, clinical-stage adeno-associated virus (AAV) gene therapy company, today announced the appointment of Tim Kelly, PhD, as President of Manufacturing. He will oversee all manufacturing functions at AskBio and its Viralgen affiliate for the production of clinical- and commercial-scale AAV vectors. Prior to joining AskBio, Dr. Kelly was the President and Chief Executive Officer at KBI Biopharma, a contract services organization that provides drug development and biomanufacturing services to pharmaceutical and biotechnology companies globally.

AskBio currently has clinical studies underway in late-onset Pompe disease and congestive heart failure. To meet the growing demand for AAV gene therapies, the company is investing in manufacturing innovation, talent and capacity that will allow it to effectively and efficiently serve patient populations.

Our goal at AskBio is to continue advancing production technology to drive down costs to make gene therapies accessible to all patients who may benefit from treatment. I am delighted that Tim has joined the company to help us shape the future of manufacturing, said Sheila Mikhail, JD, MBA, Chief Executive Officer and co-founder at AskBio. He brings a wealth of experience successfully leading therapeutic development and manufacturing and fostering the entrepreneurial, patient-focused culture that drives us at AskBio.

In January, Viralgen broke ground on a 300,000 square foot commercial facility in San Sebastin, Spain, with production expected to start in the spring of 2022, complementing the clinical-scale production currently carried out at its existing cGMP facility.

AskBios technology is truly transforming human health, and I am incredibly excited to help translate our innovations into reliable delivery of AAV gene therapy products to patients in need, said Dr. Kelly.

More about Tim KellyDr. Kelly has more than 20 years of experience in the development and manufacture of therapeutic proteins. He has overseen biopharmaceutical services for over 320 molecules at all stages of development and commercialization and supported numerous successful FDA and international regulatory inspections throughout his career. He began his tenure at KBI Biopharma in 2005, initially acting as Vice President of Biopharmaceutical Development, where he led the establishment and growth of KBIs analytical development, formulation development and cGMP laboratory services business. He subsequently served as Executive Vice President of Operations with responsibility for KBIs development and manufacturing functions in North Carolina and Colorado before becoming President and Chief Executive Officer. Prior to KBI, he directed the quality control function for Diosynth Biotechnology, where he supported clinical and commercial biopharmaceutical products. Dr. Kelly earned his PhD in molecular genetics and biochemistry from Georgia State University.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process called Pro10 and an extensive AAV capsid and promoter library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy 2i/R9 and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). Learn more at https://www.askbio.com or follow us on LinkedIn.

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/512b3baa-aaff-4a92-8539-152021f4527d

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Tim Kelly Joins AskBio as President of Manufacturing to Lead AAV Vector Production for Gene Therapy - GlobeNewswire

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Gene Therapy Industry Market 2020 |Global Industry Analysis By Trends, Size, Share, Company Overview, Growth And Forecast By 2026| Latest Research...

Familialhypercholesterolemia (FH) is one of the most clinically relevant monogenicdisorders contributing to the development of atherosclerotic cardiovasculardisease (ASCVD). The prevalence of FH was estimated to be 1 in 200 to 1 in 250 individualsin studies in which genetic testing was conducted on large community populationsamples.1 However, the disease often remains undetected and thusuntreated, with only 10% of individuals with FH receiving adequate diagnosisand treatment.2

Notingthe recent accumulation of studies on FH, the authors of a Nature ReviewsCardiology article sought tosummarize the key elements of a model of care for the condition that canbe adapted as new evidence emerges.1 Selected points are highlightedbelow.

Screening and detection. A combination of selective, opportunistic (eg, genetic screening of blood donors), systematic, and universal screening approaches is recommended to improve the detection of FH. Universal screening of children and childparent (reverse) cascade testing is potentially a highly effective method for detecting patients with FH at a young age, before they develop ASCVD32 [and] might be particularly relevant to communities with gene founder effects, noted the review authors. All children with FH should ideally be detected from the age of 5 years or earlier if homozygous FH (hoFH) is suspected.

Diagnosis. In the United States, elevated levels of low-density lipoprotein cholesterol (LDL-C) and a family history of FH are the main phenotypic criteria for FH diagnosis in children. Patients with hoFH, heterozygous FH (heFH), and polygenic hypercholesterolemia may also present with overlapping LDL-C levels, posing a challenge for the development of a standardized diagnostic tool for FH.

Genetic testing. Aninternational expert panel recently endorsed genetic testing in the care ofpatients with FH as it would [allow] a definitive diagnosis, improve[e] riskstratification, address the increasing need for more potent therapies, improve[e]adherence to treatments, and increase[e] the precision and cost- effectivenessof cascade testing.1,3 However, genetic testing remains underuseddue to issues such as cost, low access to genetic counseling, and lack ofclinician knowledge in this area.

Clinical risk assessment.Cumulative lifetime exposure to elevated LDL-C is the key factor driving ASCVDrisk in asymptomatic patients with FH, further underscoring the need for timelydiagnosis and risk stratification. In addition to phenotypic and geneticfactors, imaging of subclinical atherosclerosis, might be the most usefulclinical tool for assessing risk in FH.1 For example, imaging ofcoronary artery calcium can be used to predict coronary events in asymptomaticmiddle-aged patients with FH taking statins, and computed tomography coronaryangiography can be used to assess plaque burden and to intensify therapy.

Care of adults.Emerging evidence continues to support aggressive cholesterol-lowering therapyand lifestyle management in patients with FH from as young as 8 years tomaximally mitigate the cumulative cholesterol burden of risk. The review authorsemphasize the importance of patient-centered care and shared decision making,although health literacy is a challenge that may need to be addressed with somepatients.

Whilethere is insufficient evidence to develop strictly defined LDL-C treatmenttargets, current evidence-based recommendations stipulate that in adultpatients with FH, statin therapy and diet should initially be targeted toachieve a 50% reduction in LDL-cholesterol level and an LDL-cholesterol level<1.8 mmol/l (70 mg/dl) or <2.6 mmol/l (100 mg/dl) for primaryprevention, and <1.4 mmol/l (55 mg/dl) or <1.8 mmol/l (70 mg/dl) forsecondary prevention or for patients at very high risk.1

The addition of ezetimibe is indicated in patients who do not achieve the recommended LDL-C levels with statins alone. The use of a proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor as a third-line therapy is recommended in those patients or in patients who are intolerant to statins. The addition of a PCSK9 inhibitor in patients with heFH can further reduce LDL-C levels by approximately 60% and lead to recommended treatment targets in more than 80% of patients. However, these agents should not be used during pregnancy, as they cross the placenta and their impact on fetal development has not yet been determined.

Care of children. Extensive evidence supports the treatment of FH starting in childhood, as [m]odest and sustained reductions in LDL- cholesterol levels from early life can have a major effect on reducing mortality associated with ASCVD. Initial therapy is based on lifestyle management in early childhood, with the addition of statins by age 10 years in children with HeFH and upon diagnosis in children with hoFH. Ongoing research is investigating the efficacy and safety of PCSK9 inhibitors in children with heFH or hoFH.4,5

Radical therapies and novel approaches. Lipoprotein apheresis may be required insevere cases of FH, including in pregnant women, and liver transplantationremains the only curative therapy for patients with severe hoFH.

In ongoing studies, an array of novel treatment approaches are being examined, including functional LDL receptor gene transfer therapy in patients with hoFH and targeted RNA-based therapies to lower elevated lipoporotein(a) levels.6-8

Reviewauthors also emphasized the importance of clinical registries, patient supportgroups and networks, and the need for structured research programs that areunderpinned by actionable dissemination and implementation strategies,research skills and training among service providers, and sustainable fundingmodels. They stated that a major challenge is translating new evidence intohealth policy and routine care. Systems approaches for supporting healthorganizations and providers in addressing these gaps in care and serviceprovision are essential.

We spoke with Seth Shay Martin, MD, MHS, associate professor ofmedicine at the Johns Hopkins University School of Medicine in Baltimore,Maryland, and director of the Advanced Lipid Disorders Program of the Ciccarone Center atJohns Hopkins.

Cardiology Advisor: What are examplesof the latest advances in knowledge or practice pertaining to FH?

Dr Martin: A big advance inpractice has been the introduction of PCSK9 inhibitors. When added to statinsand ezetimibe, this class of medications can lower LDL-C by 60% sometimes the reduction can be lower, but inmy experience the effect is commonly approximately 60%. This leads to patientscoming back to clinic really satisfied.

Cardiology Advisor: What is the optimalapproach for the treatment of these patients, and what are some of the toptreatment challenges?

Dr Martin: The optimal approach is to follow the 2018 American Heart Association/American College of Cardiology multi-society guidelines, which recommend a combination approach of lifestyle modification with first-line maximal statin therapy, followed by the addition of ezetimibe and PCSK9 inhibitors. The LDL-C threshold at which additional therapy should be considered is70 mg/dL in high-riskpatients with ASCVD and FH. In patients with isolated FH (termed severe hypercholesterolemia by the guidelines,based on LDL-C levels 190 mg/dL), the LDL-C threshold is 100 mg/dL.

Cardiology Advisor: What are otherrelevant treatment implications for clinicians who treat these patients?

Dr Martin: One of the joys intaking care of a patient with FH is taking care of a family. It is a geneticdisorder with a 50% chance of being passed from parent to child. It is key toperform cascade testing to identify other members of the family; family visitsto the clinic can be beneficial for all.

Cardiology Advisor: What are remaining needs in thisarea?

Dr Martin: There is a great need for increasing awareness and diagnosis rates for FH. This is what our center is working to do as partners of the FH Foundation and as a CASCADE FH Registry site.

References

Go here to read the rest:
Reviewing Evidence on the Screening, Diagnosis, and Care of Familial Hypercholesterolemia - The Cardiology Advisor

Institutes participating in the partnership include the Stanford University School of Medicine, the Fred Hutchinson Cancer Research Center, and Parker Institute for Cancer Immunotherapy.

The Cocoon platform is a closed automated cell therapy manufacturing platform enabling decentralized process development. A transportable cassette that internalizes all of the media, agents, and consumables used in the process is attached inside and the Cocoon closes and begins processing.

Each Cocoon develops therapy for one patient, therefore the technology is patient-scale, and the process can be scaled with many Cocoons, attached on Cocoon trees operating at the same time.

Under the agreement, Lonzas experts will work collaboratively with research teams of the partners to transfer some of their existing cell-based immunotherapies, which are in pre-clinical phase, to the Cocoon bioprocessing system.

Subsequently, the process development will be shared between the partners facilities and Lonzas R&D site in Shady Grove (MD), US.

Once these therapies enter the clinic, whether manufacturing is at the institutes or elsewhere, the Cocoon platform will enable this, Eytan Abraham, head of personalized medicine at Lonza, told us.

Asked about the potential immunotherapies examined, Abraham said that they target a combination of hematological malignancies, solid malignancies, and processes that use non-viral delivery of the gene of interest.

Use of the Cocoon technology can potentially benefit the organizations development projects in several ways, including increased process control, reductions in costs, manpower, time and space requirements, as well as offering superior scalability thereby enabling treatment of larger patient populations.

Further than that, Lonza expects the partnerships to help assess the technology and evaluate the platforms potential to manufacture a range of cell therapies comparable to those manufactured through other processes currently available.

Through these collaborations we are both showcasing the Cocoon advantages and capabilities, but also learning what is needed for decentralized based manufacturing of the next wave of patient scale cell therapies, Abraham told us.

He added that, accordingly, the company will continue to evolve the system to answer these needs, whether they increase cell numbers, improve in-process analytics, integration of additional technologies, such as magnetic cell separation and electroporation, or scaling technologies.

Originally posted here:
Lonza partners with three institutes on Cocoon system - BioPharma-Reporter.com

BURLINGTON, Mass., March 12, 2020 (GLOBE NEWSWIRE) -- Flexion Therapeutics, Inc. (Nasdaq:FLXN) today reported financial results and recent business highlights for the quarter and the full year ended December 31, 2019.

2019 marked an exciting period of growth for both Flexion and ZILRETTA, said Michael Clayman, M.D., President and Chief Executive Officer. While our full-year sales of $73 million reflect the building market enthusiasm for ZILRETTA, we believe we are still just scratching the surface of its potential. Based on our recent label update, increasing utilization from existing accounts, a growing prescriber base, and most importantly, the strong clinical performance of the product, we have growing confidence that ZILRETTA can become the leading intra-articular therapy for the millions of people who confront osteoarthritis knee pain each year. Furthermore, we continue to investigate ZILRETTAs potential in new indications with our Phase 2 trial in shoulder OA and adhesive capsulitis.

Added Dr. Clayman, ZILRETTA is the engine that will drive our growth in the years ahead and facilitate our goal of becoming the leader in discovering, developing and commercializing new treatments for musculoskeletal conditions. We are making great strides towards this goal as evidenced by the progress of FX201 and FX301, two potentially transformative product candidates.

2019 Financial Highlights

The Company reported a net loss of $149.8 million for full-year 2019 as compared to a net loss of $169.7 million for full-year 2018. Net sales of ZILRETTA were $23.7 million for fourth-quarter 2019 and totaled $73 million for full-year 2019. The cost of sales for full-year 2019 was $10.0 million.

Research and development expenses were $69.6 million and $53.1 million for the years ended December 31, 2019 and 2018, respectively. The increase in research and development expenses year-over-year of $16.5 million was primarily due to an increase in salary and other employee-related costs for additional headcount and stock-based compensation expense and an increase in expenses related to portfolio expansion, ZILRETTA development and other program costs.

Selling, general and administrative expenses were $129.7 million and $121.3 million for the years ended December 31, 2019 and 2018, respectively. Selling expenses were $96.3 million and $87.3 million for the years ended December 31, 2019 and 2018, respectively. The year-over-year increase in selling expenses of $9.0 million was primarily due to salary and other employee-related costs and external costs related to marketing and reimbursement support activities. General and administrative expenses were $33.4 million and $34.0 million for the years ended December 31, 2019 and 2018, respectively, which represents a decrease of $0.6 million year-over-year.

Interest expense was $17.1 million and $15.7 million for the years ended December 31, 2019 and 2018, respectively.

As of December 31, 2019, the Company had approximately $136.7 million in cash, cash equivalents, and marketable securities compared with $258.8 million as of December 31, 2018.

ZILRETTA Commercial MetricsSince the commercial launch of ZILRETTA in November 2017 through December 31, 2019:

Recent News and 2019 Business Highlights

FX201 is a gene therapy product candidate designed to stimulate the production of an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), whenever inflammation is present within the joint. Based on preclinical data, the Company believes FX201 holds the potential to provide OA pain relief for a year or more and may slow disease progression.

Conference CallFlexions management will host a conference call today at 4:30 p.m. ET. The dial-in number for the conference call is 855-770-0022 for domestic participants and 908-982-4677 for international participants, with Conference ID #8074875. A live webcast of the conference call can also be accessed through the Investors tab on the Flexion Therapeutics website, and a replay will be available online after the call.

Indication and Select Important Safety Information for ZILRETTA(triamcinolone acetonide extended-release injectable suspension)

Indication:ZILRETTA is indicated as an intra-articular injection for the management of osteoarthritis pain of the knee.

Limitation of Use: The efficacy and safety of repeat administration of ZILRETTA have not been demonstrated.

Contraindication:ZILRETTA is contraindicated in patients who are hypersensitive to triamcinolone acetonide, corticosteroids or any components of the product.

Warnings and Precautions:

Intra-articular Use Only:ZILRETTA has not been evaluated and should not be administered by epidural, intrathecal, intravenous, intraocular, intramuscular, intradermal, or subcutaneous routes. ZILRETTA should not be considered safe for epidural or intrathecal administration.

Adverse Reactions:The most commonly reported adverse reactions (incidence 1%) in clinical studies included sinusitis, cough, and contusions.

Please seeZilrettaLabel.comfor full Prescribing Information.

About ZILRETTAOnOctober 6, 2017, ZILRETTA was approved by the U.S.FDAas the first and only extended-release intra-articular therapy for patients confronting osteoarthritis-related knee pain. ZILRETTA employs proprietary microsphere technology combining triamcinolone acetonide a commonly administered, short-acting corticosteroid with a poly lactic-co-glycolic acid (PLGA) matrix to provide extended pain relief. The pivotal Phase 3 trial on which the approval of ZILRETTA was based showed that ZILRETTA significantly reduced knee pain for 12 weeks, with some people experiencing pain relief through Week 16.

About Osteoarthritis (OA) of the KneeOA, also known as degenerative joint disease, affects more than 30 million adults living in the U.S. and accounts for more than$185 billionin annual expenditures. In 2016, more than 15 million Americans were diagnosed with OA of the knee and the average ageof physician-diagnosed knee OAhas fallen by 16 years, from 72 in the 1990s to 56 in the 2010s. The prevalence of OA is expected to continue to increase as a result of aging, obesity and sports injuries. Each year, approximately five million OA patients receive either an immediate-release corticosteroid or hyaluronic acid intra-articular injection to manage their knee pain.

About FX201FX201 is a locally administered gene therapy product candidate which utilizes a helper-dependent adenovirus (HDAd) vector, designed to stimulate the production of an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), whenever inflammation is present within the joint. Inflammation is a known cause of pain, and chronic inflammation is thought to play a major role in the progression of osteoarthritis (OA). By persistently suppressing inflammation, Flexion believes FX201 holds the potential to both reduce OA pain and modify the disease.

About FX301FX301 is a locally administered NaV1.7 inhibitor product candidate, known as funapide formulated for extended release in a thermosensitive hydrogel. The initial development of FX301 is intended to support administration as a peripheral nerve block for control of post-operative pain. Flexion believes FX301 has the potential to provide effective pain relief while preserving motor function and anticipates initiating clinical trials in 2021.

AboutFlexion TherapeuticsFlexion Therapeutics(Nasdaq:FLXN) is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with osteoarthritis, the most common form of arthritis. The company's core values are focus, ingenuity, tenacity, transparency and fun. Visitflexiontherapeutics.com.

Forward-Looking Statements This release contains forward-looking statements that are based on the current expectations and beliefs of Flexion. Statements in this press release regarding matters that are not historical facts, including, but not limited to, statements relating to the future of Flexion; our expectations regarding revenues for the year ended December 31, 2020; expected sales growth of ZILRETTA; expected clinical developments and clinical trial timelines; expected increases in the rate of individuals with OA of the knee; and the potential therapeutic and other benefits of ZILRETTA and Flexions product pipeline, are forward looking statements. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, risk that we may not achieve revenue expectations for 2020; the risk that we may not be able to successfully maintain an effective sales force to commercialize ZILRETTA; competition from alternative therapies; the risk that we may not be able to maintain and enforce our intellectual property, including intellectual property related to ZILRETTA; the risk that ZILRETTA may not be successfully commercialized or adopted; risks regarding our ability to obtain adequate reimbursement from payers for ZILRETTA; risks related to the manufacture and distribution of ZILRETTA, including our reliance on sole sources of supply and distribution; risks related to clinical trials, including potential delays, safety issues or negative results; risks related to key employees, markets, economic conditions, health care reform, prices and reimbursement rates; and other risks and uncertainties described in our filings with theSecurities and Exchange Commission(SEC), including under the heading "Risk Factors" in our Quarterly Report on Form 10-Q for the quarter endedSeptember 30, 2019filed with theSEConNovember 7, 2019and subsequent filings with theSEC. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of the statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

Contact:

Scott YoungVice President, Corporate Communications & Investor RelationsT: 781-305-7194syoung@flexiontherapeutics.com

Julie DownsAssociate Director, Corporate Communications & Investor RelationsT: 781-305-7137jdowns@flexiontherapeutics.com

Excerpt from:
Flexion Therapeutics Reports Fourth-Quarter and Full-Year 2019 Financial Results - GlobeNewswire

Genes may determine what characteristics are passed down from parent to offspring, but each cell expresses these genes differently based on external epigenetic modifications. Epigenetics dont alter the gene sequence (genotype), but they do influence cell behavior and function (phenotype). The study of epigenetics helps us understand how phenotypic changes lead to disease, stem cell differentiation, and essentially, what drives the fate of every cell in the human body.The epigenome is not consistent between cells, or even between cells of the same type. Individual modifications come and go throughout a cells lifetime. Therefore, scientists are faced with the steep challenge as they try to decipher the role of epigenetics in disease and development.[i] Understanding intercellular heterogeneity is key here. The epigenome must be examined at single-cell resolution.

Now, with the advancement of single-cell sequencing methods like the single-cell assay for transposase accessible chromatin (scATAC-seq), researchers have access to sophisticated techniques to map large cell populations, one cell at a time. The resulting epigenomic information provides unprecedented insight into the different cell types that come together to form organs and organ systems, as well as pathogenic modifications associated with disease.

Every single cell has unique epigenomic instructions that guide how it expresses its genes and these instructions are subject to change. A map locating epigenetic modifications in the genome would help scientists understand how epigenetics drives cellular differentiation. But until recently, epigenetic assays mainly focused on select regions of DNA or gave bulk results across an entire sample of cells.[ii] These assays were not designed to detect epigenetic patterns in individual cells.

Single-cell tools like scATAC-seq help us get a grasp on intracellular heterogeneity, differentiate between cell populations and map the role of epigenetics in the larger context of an organism. By building a collection of scATAC-seq data, scientists have begun generating a cell atlas to provide insight into the role of epigenetics during the intricate biological processes that occur throughout the human lifetime.

During ATAC-seq, a hyperactive transposase mutant, Tn5, binds to open chromatin (euchromatin) regions. Wherever Tn5 binds, it cleaves the DNA and attaches sequencing adapters. Then, after PCR amplification, ATAC fragments are sequenced to identify open chromatin regions. ATAC results indicate where nucleosomes are typically positioned in the cell sample and which regions of the genome are open for transcription factors to bind. As such, scientists use ATAC-Seq as a first-pass screening approach to identify changes in chromatin accessibility between samples.

ATAC-seq has many practical applications, but it cant account for the cell-to-cell variability thats often an important aspect of developmental processes and disease. So, researchers developed a new assay in which microfluidic technology is used to isolate individual cells before ATAC-seq.[iv] This assay provides epigenomic information at single cell resolution, earning it the name scATAC-seq.

The key to the scATAC-seq method is that it isolates genomes of individual cells early on to perform a separate ATAC-seq reaction on each individual cell. Then, open regions of the genome are cleaved by the Tn5 transposase, tagged with sequencing adapters and amplified with barcoded cell-identifying primers. Subsequently, the barcoded libraries of ATAC fragments, (each representing an individual cell) are pooled together and sequenced to reveal open chromatin regions of thousands of individual cells.

The first droplet-based iteration of the scATAC-seq method (dscATAC-seq) uses a single cell isolator to encapsulate thousands of individual nuclei in nanoliter-sized droplets for ATAC sequencing. It uses a custom Tn5 transposase to enhance library complexity and signal resolution. Compared to the original microfluidic method, the new workflow is faster and yields greater biological insight with less time and effort spent on sequencing. To demonstrate its power and potential, this technique has been used to conduct an unbiased analysis of the many different cell types and regulatory elements in a mouse brain. [v]

Figure 1:In scATAC-seq, droplet-based technology partitions thousands of whole cells or nuclei into individual nanoliter-sized droplets, enabling researchers to prepare a library of ATAC fragments for sequencing to reveal open chromatin regions. Credit:Bio-Rad Laboratories.

To capture single cell data on a truly massive scale, combinatorial indexing was next introduced into the dscATAC-seq workflow. This new method, called dsciATAC-seq, enables researchers to assess up to 50,000 cells in a single assay. Assaying a large volume of cells is possible because, in dsciATAC-seq, the hyperactive mutant transposase integrates a first set of barcodes as it cleaves open regions of chromatin in each nucleus. Because every cells DNA already carries a barcode, multiple cells can be loaded into a single droplet. Then, as usual, ATAC fragments are amplified with a second set of barcoded primers. After sequencing these fragments, the two sets of barcodes are used to derive epigenomic profiles for tens of thousands of cells.

Putting the dsciATAC-seq method to the test, researchers have studied immune cell clusters from human bone marrow derived cells to illustrate how the chromatin accessibility landscape in these cells changes according to different stimulants at the single cell level.5Although the number of cells that a single scATAC-seq experiment can evaluate has grown dramatically, it will take a continued concerted effort from scientists across many disciplines to create a comprehensive map of the human epigenome, encompassing data from trillions of cells.[vi] Furthermore, to help decode the patterns we find in the human epigenome, it may be valuable to gather information about the epigenomes of animals commonly used as research models. As each of these maps become increasingly detailed, scientists will gain a more thorough understanding of how biological process work and may apply this knowledge towards developing better treatments for complex diseases.

Reference:

[i] Egger, G., et al. Epigenetics in human disease and prospects for epigenetic therapy. Nature, 2004, 429, 457463. doi:10.1038/nature02625[ii] DeAngelis, J. T., Farrington, W. J., & Tollefsbol, T. O. An overview of epigenetic assays. Molecular biotechnology, 2008, 38(2), 179183. doi:10.1007/s12033-007-9010-y[iii] Buenrostro JD, Giresi PG, Zaba LC, Chang HY, Greenleaf WJ. Transposition of native chromatin for fast and sensitive epigenomic profiling of open chromatin, DNA-binding proteins and nucleosome position. Nature Methods, 2013, 10(12):1213-8. doi: 10.1038/nmeth.2688.[iv] Buenrostro JD, Wu B, Litzenburger UM, Ruff D, Gonzales ML, Snyder MP, Chang HY, Greenleaf WJ. Single-cell chromatin accessibility reveals principles of regulatory variation. Nature, 2015, 523(7561):486-90. doi: 10.1038/nature14590.[v] Lareau, C.A., Duarte, F.M., Chew, J.G. et al. Droplet-based combinatorial indexing for massive-scale single-cell chromatin accessibility. Nature Biotechnology 37, 916924 (2019) doi:10.1038/s41587-019-0147-6.[vi] Bianconi, E., Piovesan, A., Facchin F., Beraudi, A., Casadei. R., Frabetti, F., Vitale, L., Pelleri, M., Tassani. S., Piva, F., Perez-Amodio, S, Strippoli, P. & Canaider, S. An estimation of the number of cells in the human body. Annals of Human Biology, 2013, 40:6, 463-471. doi: 10.3109/03014460.2013.807878.

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Using Single Cells To Get the Whole Picture of the Epigenome - Technology Networks

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2020-2025 Global and Regional Anti-Senescence Therapy Industry Production, Sales and Consumption Status and Prospects Professional Market Research...

MarketsandResearch.biz is releasing the latest insights through extensive research titled Global Gene Therapy for Age-related Macular Degeneration Market 2020 by Manufacturers, Countries, Type and Application, Forecast to 2025 which provides a comprehensive researched abstract of the key players with considerable shareholdings at a global level regarding demand, sales, and income through offering better products and services. The perceptive research study gives an in-depth analysis featuring market scope, history, establishment, attractiveness, production, sales volume, and growth potentials. It presents a thorough lookout towards the ongoing market structure as well as a forecast for the global Gene Therapy for Age-related Macular Degeneration market between 2020 and 2025.

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Gene Therapy for Age-related Macular Degeneration Market Growth Forecast 2020-2025 RetroSense Therapeutics, REGENXBIO, AGTC - News Times

Woburn Mar 13, 2020 (Thomson StreetEvents) -- Edited Transcript of Flexion Therapeutics Inc earnings conference call or presentation Thursday, March 12, 2020 at 8:30:00pm GMT

* David A. Arkowitz

Flexion Therapeutics, Inc. - CFO

* Michael D. Clayman

Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director

Flexion Therapeutics, Inc. - VP of Corporate Communications & IR

The Benchmark Company, LLC, Research Division - Senior Healthcare Technology Research Analyst

Joh. Berenberg, Gossler & Co. KG, Research Division - Analyst

* Serge D. Belanger

Good afternoon, ladies and gentlemen. And welcome to the Flexion Therapeutics Fourth Quarter and Full Year 2019 Financial Results Conference Call. My name is Daniel, and I will be your coordinator for today. (Operator Instructions)

I'll now turn the call over to the company.

Scott Young, Flexion Therapeutics, Inc. - VP of Corporate Communications & IR [2]

Thank you, Dan. Good afternoon. This is Scott Young, Vice President for Corporate Communications and Investor Relations. Before we begin, I would call your attention to the metrics slide that we will discuss in today's presentation. Those slides can be viewed directly via the webcast, in the 8-K we issued this afternoon or under the Investors tab on flexiontherapeutics.com.

In addition, our Q4 earnings press release and an archive of this conference call, can also be found there. Today's call will be led by Flexion's Chief Executive Officer, Dr. Michael Clayman; and he is joined by David Arkowitz, Flexion's Chief Financial Officer; and Melissa Layman, Flexion's newly appointed Chief Commercial Officer.

On today's call, we will be making forward-looking statements that include commercial, financial, clinical and regulatory projections. Statements relating to future financial or business performance, conditions or strategies

matters, including expectations regarding net sales, operating expenses, cash utilization, clinical, regulatory and commercial developments and anticipated milestones are forward-looking statements within the meaning of the Private Securities Litigation Reform Act.

Flexion cautions that these forward-looking statements are subject to various assumptions, risks and uncertainties, which change over time. Additional information on the factors and risks that could affect Flexion's business, financial conditions and results of operations are contained in Flexion's Form 10-K for the year ended December 31, 2019, which was filed with the SEC and other filings, which are available at http://www.sec.gov as well as Flexion's website.

These forward-looking statements speak only as of the date of this call, and Flexion assumes no duty to update such statements. I will now

over to Flexion's CEO, Mike Clayman.

Michael D. Clayman, Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director [3]

--------------------------------------------------------------------------------

Thanks, Scott, and thank you all for joining. Today, I'll recap our commercial progress in 2019, review our life cycle management activities, provide an update on our pipeline and discuss our priorities for this year. After that, I'll turn it over to David for a deeper review of our commercial metrics and a summary of our financial performance, and then we will open the line and take questions.

To begin, as we reported today, we recorded full year ZILRETTA net sales of $73 million for 2019, which is fully in line with the preliminary unaudited revenue estimate we provided in early January. We are very pleased with our sales performance in 2019, which represents growth of more than 220%, compared to our 2018 full year net sales of $22.5 million. Those numbers tell a very compelling story, and they speak to the outstanding work of our field-based teams, the excellent coverage we have for ZILRETTA, the impact from our judicious use of volume-based rebates, but most importantly, our sales performance speaks to the remarkable experience that patients and clinicians are having with ZILRETTA. As you will see in the commercial metrics that David will review, by the end of 2019, our customers had purchased 175,000 units of ZILRETTA. While our data are limited to the account level, that number provides a reasonable surrogate for the number of patients who have been treated with ZILRETTA since it was introduced to the market in late 2017. And we know that many of those patients have received unprecedented pain relief from ZILRETTA. I say it is based on real-world feedback from the countless conversation I've had with grateful ZILRETTA patients and their physicians, feedback that is wholly consistent with our compelling clinical trial data.

In fact, last year, we became aware of several professional athletes who received ZILRETTA, namely Rod Woodson, the NFL Hall of Fame Defense named as one of the top 100 players of all time. Michael Eruzione, the captain of (inaudible) Gold medal winning U.S. Olympic Hockey team and the player who scored the game winning goal against the Russians during the Miracle On Ice; and Chris Dickerson, a former outfielder who played from the New York Yankees and other major league baseball teams. From snow blowing a driveway to playing with their children and grandchildren, to jogging for the first time in years, they all have profoundly moving stories about how ZILRETTA has helped each of them manage their knee OA pain, and thereby, improve their ability to participate in regular everyday activities. As part of our ongoing physician marketing initiatives, we brought all 3 athletes together at a major orthopedic conference last December, where they and their treating physicians, spoke to a standing room crowd of some of the country's leading sports medicine experts. They share their stories of how ZILRETTA has helped them, and the best words I can use to describe that session are inspiring and humbling.

While all are elite athletes, their ZILRETTA stories echo the scores of e-mails and letters I've received from patients who are so grateful to reengage in things they love doing with less pain. From gardening, playing around of golf, walking on the beach, hiking, each of these stories serves as a reminder to all of us that while we have impacted more than 175,000 patients, there are 15 million people who see their doctor every year for OA knee pain. And 5 million of them receive an intra-articular injection. The opportunity for ZILRETTA inflection is truly massive, which brings me back to our performance in 2019 and our goals for 2020.

Throughout the course of 2019, we saw existing practices increase their use of ZILRETTA and more than 1,600 new accounts start using the product. Today, with the benefit of 2 years in the market, we have actual claim

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play changes in the payer mix versus our assumptions prior to launch. We now see that the actual fix is skewed slightly more toward Medicare, which accounts for roughly 55% of the market versus previous estimates of 50%. 8% is still commercial, but Medicaid, VA and 340B plans are making up roughly 5% of the mix. We view this modest increase in Medicare patients to be incrementally positive as Medicare patients can be injected the same-day they visit without any need for prior authorization. In addition, it is important to point out that commercial coverage for ZILRETTA remains excellent. As we discussed in December, the recent approval of our NDA resulted in a significantly improved label, which most importantly, removed the onerous not intended for repeat administration wording in the limitation of use statement. Within days of the approval, we developed materials to help our MBMs communicate the label update and we have anecdotal feedback from the field that tells us that the changes have been very well received. While we clearly view the label update as a tailwind. We have always said that we continue to believe that the meaningful impact will be seen over the following quarters and years. From a commercial perspective, our progress in 2019 strengthens our belief that ZILRETTA can become the new standard of care for the intra-articular treatment for OA knee pain in the years ahead. While under normal circumstances, these factors would give us confidence that our 2020 ZILRETTA's net sales guidance of $120 million to $135 million is both credible and achievable. Like all businesses, we are acutely attuned to the potential impacts of the coronavirus global pandemic, and we are monitoring this dynamic situation very closely. To date, we have not seen any material impacts on ZILRETTA sales, our ability to access customers or to initiate our clinical trials. However, it is impossible to predict how the outbreak could evolve in the months ahead or what impacts more aggressive social distancing or other containment efforts might have on patients or practices. Regarding our supply chain, we believe we are in a very strong position. We do not source any of our key materials from China, and we presently have approximately 10 months worth of finished product inventory in our warehouses in the U.S. Furthermore, we have an additional 12 months of API, triamcinolone acetonide at our manufacturing facility in the U.K. Again, it is impossible to predict the long-term impacts of the outbreak. But we feel very good about our ability to provide ZILRETTA to patients over the quarters ahead.

Shifting to our clinical development activities, our Phase II trial to investigate the safety and efficacy of ZILRETTA in shoulder OA and adhesive capsulitis, also known as frozen shoulder syndrome continues to advance, and we anticipate data from that trial in 2020.

As we've previously discussed, these 2 conditions combined account for roughly 800,000 injections, and they present an opportunity for us to expand the use of ZILRETTA with a subset of orthopedics, who primarily focus on sports medicine and commonly treat these conditions with steroid injections.

With respect to our pipeline, we've also been making progress with our 2 drug candidates, FX201 and FX301. FX201 is our gene therapy, which holds the potential to provide OA pain relief for at least a year, improve function and potentially modify disease. As we announced last year, the IND for FX201 was accepted by the FDA, and we recently treated the first 2 patients in our Phase I dose-ranging study. We anticipate treating approximately 15 to 24 patients, who will be followed for 104 weeks with initial readout in 2021.

Now we'll move to FX301. Our NAV 1.7 inhibitor, [formulated]

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sensitive hydrogel for administration as a peripheral nerve block for control of postoperative pain. We've held our pre-IND meeting with FDA, and we remain on track to initiate our first FX301 clinical trial in

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unlike typical local anesthetics. We believe the [selective] pharmacology of FX301 has the potential to deliver at least 3 to 5 days of effective pain relief, while preserving motor function, which could enable ambulation, rapid discharge from the hospital and early rehabilitation following musculoskeletal surgery.

Finally, regarding our search for a Chief Commercial Officer. We were very excited to announce the appointment of Melissa Layman earlier this week, and I'm delighted that she is able to join us for today's call. As we've said repeatedly, in our search for a CCO, we were looking for someone who have had success leading an entire commercial enterprise, who had deep experience and expertise in each of the key commercial functional domains, who had a track record as a very strong leader and who would positively contribute to our already strong culture. Because Melissa fulfills all of these criteria and is simply a terrific person to interact with. We could not be more pleased to have her take the helm of our commercial organization. Before David summarizes our fourth quarter financials and walks through the commercial metrics, I'd like to give Melissa the opportunity to make a few remarks. Melissa?

--------------------------------------------------------------------------------

Melissa Layman, [4]

--------------------------------------------------------------------------------

Thank you, Mike, and thank you for the kind words. It is such a pleasure to be here today and such an honor to join Flexion. While I've barely been in my role for a day, I can already see what an amazing group of people work here. The team has been welcoming, engaging and candid, and that was consistent throughout the entire interview process from the Executive Committee to the Board members. The commitment to patients and passion for winning has been universal, and that was one of the many reasons I wanted to join. Put simply, I can't begin to convey how excited I am

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opportunity that's ahead of us. The strong foundation that's been laid by Mike, David, Steve Meyers, and the rest of the commercial leadership team is truly impressive, and I'm looking forward to working together to grow ZILRETTA's market share and help make it the leading IA treatment in this space. Over the next few months, I'll be working intensively with our commercial organization, and I look forward to representing our commercial effort on future calls. At this point, I'll turn it over to David.

--------------------------------------------------------------------------------

David A. Arkowitz, Flexion Therapeutics, Inc. - CFO [5]

--------------------------------------------------------------------------------

Thank you, Melissa. I'll start by walking through our commercial metrics which, as Scott mentioned, can be found on our website and in the 8-K we issued today.

If you look at Slide 2, you can see that we recorded ZILRETTA net sales of $23.7 million in the fourth quarter of 2019, bringing full year 2019 net sales to $73 million. As Mike mentioned, year-over-year growth topped 20%. As in previous quarters, we expanded our list of target accounts in the fourth quarter to 4,972, and by December 31, 2019, we've called on almost all of them. At the end of the fourth quarter, 3,488 accounts had purchased ZILRETTA, which is an increase of nearly 360 purchasing accounts compared to the end of the third quarter.

As of the end of December, we had 2,642 accounts or 76% of all purchasing accounts placed at least 1 reorder for ZILRETTA. Notably, we saw our reorder rate increase in each quarter throughout 2019, and this occurred on a successively growing customer base. Slide 3 charts our quarterly sales from launch through the fourth quarter of 2019, which provides a very compelling view of ZILRETTA's growth in the market, especially since the introduction of the permanent J Code on January 1, 2019. We do not provide quarterly guidance, but based on routine seasonality impacts, directionally, we anticipate first quarter net sales to be roughly flat versus the fourth quarter of last year.

Moving to Slide 4. This slide and the remaining 2 slides reflect purchases of ZILRETTA by accounts, which represent physician practices, clinics and hospitals of various sizes and purchasing potential. As we look at the distribution of accounts that have purchased the ZILRETTA since launch, we stayed with the same groupings that we've used in previous quarters, accounts that have purchased 1 to 10 units, purchased 11 to 50 units or purchased more than 50 units. We continue to see a significant number of accounts with purchases of 1 to 10 units. And as of December 31, 2019, roughly 1,670 accounts had made purchases in this range. While approximately 1,030 accounts had purchased 11 of 50 units. In addition, 794 accounts had purchased more than 50 units, which represents growth of 150% as compared to Q1 2019 when 313 accounts had purchased in this category.

Looking at Slide number 5. You can clearly see the distribution of purchases by accounts. Those 794 accounts that have each purchased more than 50 units are in total, responsible for approximately 143,000 units or roughly 81% of all units purchased since launch. As we have mentioned previously, accounts generally move along a utilization continuum from 1 to 10 units to 11 to 50 units and then to more than 50 units. Importantly, none of our purchasing accounts has fully incorporated ZILRETTA into their practice. And this holds true for even the highest utilizers. As a result, we believe there is tremendous opportunity for us to increase utilization across each of these groups. Before I leave this slide, I would like to point out that the total ZILRETTA purchases by accounts in the fourth quarter were approximately 37,500 units, which is lower than the 48,600 units purchased in the third quarter. We believe this quarter-over-quarter reduction was primarily the result of the broad-based rebate program that we introduced in the third quarter. There was a strong amount of enthusiasm and pent-up demand for this program, and we believe that some purchases that otherwise would have occurred in the fourth quarter instead occurred in the third quarter. To a much lesser extent, we believe that the holidays in the fourth quarter had an impact on the sequential quarter reduction as well. Nevertheless, we saw total ZILRETTA purchases by accounts increased by more than 70% in the second half of 2019 versus the first half of 2019.

Moving to Slide 6. Here, we break out ZILRETTA purchases by new and existing accounts. And in the fourth quarter, we added about 350 new purchasing accounts. While we expect to eventually see a slowing in the number of new accounts coming on board each quarter, we continue to be pleased with the progress we are making with new accounts as they typically work their way through the ZILRETTA utilization continuum.

So at this point, I will briefly walk through the fourth quarter and full year 2019 financial results, which we included in the press release issued this afternoon and in our 10-K. We reported a net loss of $149.8 million for full year 2019 as compared to a net loss of $169.7 million for full year 2018. Net sales of ZILRETTA were $23.7 million for the fourth quarter of 2019 and totaled $73 million for full-year 2019. The cost of sales for full-year 2019 was $10 million. The fourth quarter 2019 net sales reflect a gross to net reduction of 11%. A gross to net reduction is primarily comprised of distributor and service fees, returns reserve, health care provider rebates and mandatory government discounts and rebates, such as Medicaid, 340B institutions, and Veterans Administration, Department of Defense. As we previously mentioned, in the third quarter, we started offering rebates to eligible health care providers that are variable based on the volume of product purchased. These provider rebates contributed 4% of the fourth quarter total gross to net reduction of 11%. Research and development expenses were $69.6 million, and $53.1 million for the years ended December 31, 2019 and 2018, respectively. The increase in research and development expenses year-over-year of $16.5 million was primarily due to an increase in salary and other related costs for additional headcount and stock-based compensation expense, an increase in expenses related to portfolio expansion and other program costs, including an upfront payment to Xenon Pharmaceuticals related to FX301, and an increase in development expenses for ZILRETTA. Selling, general and administrative expenses were $129.7 million and $121.3 million for the year ended December 31, 2019, and 2018, respectively. Selling expenses were $96.3 million and $87.3 million for the years ended December 31, 2019 and 2018, respectively. The year-over-year increase in selling expenses of $9 million was primarily due to salary and other employee-related costs and external costs related to marketing and reimbursement support activities.

General and administrative expenses were $33.4 million and $34 million for the years ended December 31, 2019 and 2018, respectively, which represents a decrease of $0.6 million year-over-year. Interest expense was $17.1 million and $15.7 million for the years ended December 31, 2019 and 2018, respectively. We expect that while our operating expenses will continue to increase in the near term, primarily driven by commercial activities in support of ZILRETTA, line extension clinical trials for ZILRETTA, continued development of FX201 and FX301 and development activities associated with future additions to the pipeline. We believe we will be able to increasingly leverage our infrastructure in support of these efforts. As of December 31, 2019, and we had approximately $136.7 million in cash, cash equivalents and marketable securities compared with $258.8 million as of December 31, 2018. In addition, earlier this quarter, we fully drew down $20 million from our revolving credit facility, which is secured by our accounts receivables. We believe that our current cash balance with the expected future sales

[Aduio Gap]

and the ongoing prudent management of (inaudible) will enable us to reach profitability. However, our projections are based on certain market assumptions, which may or may not be affected by the coronavirus [pandemic]. As a result, we will continue to review and reassess our

(technical difficulty)

in light of those factors. In addition, we will remain opportunistic as it relates to potential funding decisions, and we will do what we believe is in the best long-term interest of Flexion and our shareholders.

At this point, I would ask the operator to please open the line for questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions)

Our first question comes from Randall Stanicky with RBC Capital Markets.

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Daniel James Busby, RBC Capital Markets, Research Division - Senior Associate [2]

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This is Dan Busby on for Randall. A couple of questions. First, among the high-prescribing accounts, in particular, can you give us a sense of how much more room there is to grow within those practices? I think I heard you mentioned that you haven't fully penetrated any of those accounts yet. And of the physicians in those accounts who aren't using it, what's the pushback you're hearing?

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David A. Arkowitz, Flexion Therapeutics, Inc. - CFO [3]

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Yes, Dan, this is David. So as we shared in our prepared remarks and in the deck, we've got almost 800 accounts that have purchased 50 units, more than 50 units of ZILRETTA launch to date. The vast, vast majority of those accounts, ZILRETTA has not been fully adopted, incorporated into their practices (inaudible) even within those accounts of almost 50 accounts that have purchased more than 500 of units of ZILRETTA launch to date. So there's room to run with those almost 800 accounts as well as the other accounts that are less than 50 units of ZILRETTA purchased launch to date. And in terms of the other part of your question, in terms of why have they not fully incorporated ZILRETTA at this juncture. The way this will typically work is take a practice with 5 or 6 physicians

(technical difficulty)

2 of those physicians have started to use ZILRETTA and are using ZILRETTA for their patient -- appropriate patient population. But there just hasn't been awareness and experience with ZILRETTA for the other 3 or 4 docs in the practice. So it's a process. It's a process for the docs that are treating the existing patients to talk to their colleagues or our representatives to be making those physicians that are not yet using ZILRETTA, aware of ZILRETTA, getting them comfortable with reimbursement. So that's really what is going on.

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Daniel James Busby, RBC Capital Markets, Research Division - Senior Associate [4]

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Okay, that's helpful. And I guess, as a follow-up to that, if 1 practice, if 1 or 2 physicians within a practice, have secured reimbursement? Is it typically the case that other doctors who aren't using it, but may in the future, they would have reimbursement set up already? Or is that more kind of doctor by doctor?

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David A. Arkowitz, Flexion Therapeutics, Inc. - CFO [5]

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No, it's typically at the office level, at the clinical level. But the -- it's an issue of just getting familiar and comfortable with reimbursement, experiencing reimbursement, and that takes those docs that have not achieved that to just go through the process.

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Operator [6]

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Our next question comes from Elliot Wilbur with Raymond James.

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Elliot Henry Wilbur, Raymond James & Associates, Inc., Research Division - Senior Research Analyst [7]

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A couple of questions. First, Mike, I believe you mentioned in your commentary that you expect 1Q 2020 ZILRETTA sales to be essentially flat versus 4Q '19. Just want to get maybe a little bit more color behind that? How much of that you think is attributable to high deductible plans, perhaps influencing utilization versus other factors such as seasonality or just sort of overall company conservatism kind of in light of potentially increased macro uncertainty here in the short term?

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Michael D. Clayman, Flexion Therapeutics, Inc. - Co-Founder, President, CEO & Director [8]

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Yes. So I think there are a few things, Elliot. So it's a good and important question. First, just to recognize, historically, in this space, the hyaluronic acids are typically down 10% in the first quarter, with the driver being, as you point out, deductible. That is a key driver. I think that you also have to look at an older population may be less active in the winter months. And as a result, have less need to go to their physicians. So there are a couple of reasons why there is a basis for, relatively speaking, the first quarter being softer than other quarters and why we have guided to flat in the first quarter.

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Elliot Henry Wilbur, Raymond James & Associates, Inc., Research Division - Senior Research Analyst [9]

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Okay. And then with respect to your commentary around some of the data points that emerge from the claims data that you're referring to in terms of the payer mix with Medicare representing now a higher proportion. How should we think about the relative opportunity with respect to additional growth levers such as repeat administration, bilateral administration within the context of a greater Medicare book of business versus commercial?

Read more from the original source:
Edited Transcript of FLXN earnings conference call or presentation 12-Mar-20 8:30pm GMT - Yahoo Finance

CRANBURY, N.J. and PHILADELPHIA, March 05, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics, Inc. (Nasdaq: FOLD) today announced the official opening of the companys Global Research and Gene Therapy Center of Excellence in uCity SquareinPhiladelphia to advance its industry leading portfolio of rare disease gene therapy programs. In 2019, Amicus and the University of Pennsylvania (Penn) announced a major expansion of their Gene Therapy Collaboration which provides Amicus with disease-specific worldwide rights to Penns Next Generation Gene Therapy Technologies from the Wilson Lab for the majority of lysosomal storage disorders, as well as twelve additional more prevalent rare diseases including Rett Syndrome, Angelman Syndrome and select other muscular dystrophies.

John F. Crowley, Chairman and Chief Executive Officer ofAmicus Therapeutics, Inc., stated, This is a remarkable advancement in the history of Amicus and further strengthens our great collaboration with Dr. Jim Wilson and the Gene Therapy Center at Penn. Philadelphia is a magnet for talent in gene therapy and an engine for innovation. This new global research center located in the cradle of liberty will become part of the cradle of cures as we move many gene therapy programs forward toward patients in need. With exclusive global rights to 50 rare diseases in collaboration with Dr. Wilsons team we hope to be able to alleviate an enormous amount of human suffering with the great science work that will be done in this new facility.

The 75,000 sq. ft. Center is located on the top three floors of the new building at 3675 Market Street and consists of office and state-of-the-art laboratories. It will ultimately house approximately 200 researchers and drug developers focused exclusively on gene therapies.

A by invitation only ribbon cutting event takes place today to celebrate the opening with special guests to include Dr. Jim Wilson, government officials and patients living with rare diseases and their families.

About Amicus TherapeuticsAmicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com and follow on Twitter and LinkedIn.

CONTACTS:

Media:Christopher ByrneExecutive Director, Corporate Communicationscbyrne@amicusrx.com(609) 662-2798

Originally posted here:
Amicus Opens New Global Research and Gene Therapy Center of Excellence in Philadelphia - BioSpace

Scientists at the Casey Eye Institute, in Portland, Ore., have have injected a harmless virus containing CRISPR gene-editing instructions inside the retinal cells of a patient with a rare form of genetic blindness. KTSDesign/Science Photo Library/Getty Images hide caption

Scientists at the Casey Eye Institute, in Portland, Ore., have have injected a harmless virus containing CRISPR gene-editing instructions inside the retinal cells of a patient with a rare form of genetic blindness.

For the first time, scientists have used the gene-editing technique CRISPR to try to edit a gene while the DNA is still inside a person's body.

The groundbreaking procedure involved injecting the microscopic gene-editing tool into the eye of a patient blinded by a rare genetic disorder, in hopes of enabling the volunteer to see. They hope to know within weeks whether the approach is working and, if so, to know within two or three months how much vision will be restored.

"We're really excited about this," says Dr. Eric Pierce, a professor of ophthalmology at Harvard Medical School and director of the Inherited Retinal Disorders Service at Massachusetts Eye and Ear. Pierce is leading a study that the procedure launched.

"We're helping open, potentially, an era of gene-editing for therapeutic use that could have impact in many aspects of medicine," Pierce tells NPR.

The CRISPR gene-editing technique has been revolutionizing scientific research by making it much easier to rewrite the genetic code. It's also raising high hopes of curing many diseases.

Before this step, doctors had only used CRISPR to try to treat a small number of patients who have cancer, or the rare blood disorders sickle cell anemia or beta-thalassemia. While some of the initial results have been promising, it's still too soon to know whether the strategy is working.

In those other cases, doctors removed cells from patients' bodies, edited genes in the cells with CRISPR in the lab and then infused the modified cells back into the volunteers' bodies to either attack their cancer or produce a protein their bodies are missing.

In this new experiment, doctors at the Casey Eye Institute in Portland, Ore., injected (into the eye of a patient who is nearly blind from a condition called Leber congenital amaurosis) microscopic droplets carrying a harmless virus that had been engineered to deliver the instructions to manufacture the CRISPR gene-editing machinery.

Beginning in infancy, the rare genetic condition progressively destroys light-sensing cells in the retina that are necessary for vision. Vision impairment with LCA varies widely, but most patients are legally blind and are only able to differentiate between light and dark or perhaps to detect movement.

"The majority of people affected by this disease have the most severe end of the spectrum, in terms of how poor their vision is," Pierce says. "They're functionally blind."

The goal is that once the virus carrying the CRISPR instructions has been infused into the eye, the gene-editing tool will slice out the genetic defect that caused the blindness. That would, the researchers hope, restore production of a crucial protein and prevent the death of cells in the retina, as well as revive other cells enabling patients to regain at least some vision.

"It's the first time the CRISPR gene-editing is used directly in a patient," Pierce says. "We're really optimistic that this has a good chance of being effective."

The study is being sponsored by Editas Medicine, of Cambridge, Mass., and Allergan, based in Dublin. It will eventually involve a total of 18 patients, including some as young as ages 3 to 17, who will receive three different doses.

"We're very excited about this. This is the first time we're doing editing inside the body," says Charles Albright, the chief scientific officer at Editas.

"We believe that the ability to edit inside the body is going to open entire new areas of medicine and lead to a whole new class of therapies for diseases that are not treatable any other way," Albright says.

Francis Collins, director of the National Institutes of Health, calls the advance "a significant moment."

"All of us dream that a time might be coming where we could apply this approach for thousands of diseases," Collins tells NPR. "This is the first time that's being tried in a human being. And it gives us hope that we could extend that to lots of other diseases if it works and if it's safe."

Pierce, Albright and others stressed that only one patient has been treated so far and that the study, still at a very early stage, is designed primarily to determine whether injecting the gene-editing tool directly into the eye is safe.

To that end, the researchers are starting with lowest dose and the oldest patients, who have already suffered extensive damage to their vision. And doctors are only treating one eye in each patient. All of those steps are being taken in case the treatment somehow backfires, causing more damage instead of being helpful.

"CRISPR has never been used directly inside a patient before," Pierce says. "We want to make sure we're doing it right."

Still, he says, if the underlying defect can be repaired in this patient and others with advanced damage, "we have the potential to restore vision to people who never had normal vision before. It would indeed be amazing."

The study involves a form of Leber congenital amaurosis known as Type 10, which is caused by a defect in the CEP290 gene.

If the approach appears to be safe and effective, the researchers will start treating younger patients.

"We believe children have the potential to have the most benefit from their therapy, because we know their visual pathways are still intact," Albright explains.

The procedure, which takes about an hour to perform, involves making tiny incisions that enable access to the back of the eye. That allows a surgeon to inject three droplets of fluid containing billions of copies of the virus that has been engineered to carry the CRISPR gene-editing instructions under the retina.

The idea is that once there, the CRISPR editing elements would snip out the mutation that causes a defect in CEP290. The hope is that this would be a one-time treatment that would correct vision for a lifetime.

If it works, the volunteers in the study might be able to have the procedure repeated on the other eye later.

"If we can do this safely, that opens the possibility to treat many other diseases where it's not possible to remove the cells from the body and do the treatment outside," Pierce says.

The list of such conditions might include some brain disorders, such Huntington's disease and inherited forms of dementia, as well as muscle diseases, such as muscular dystrophy and myotonic dystrophy, according to Pierce and Albright.

"Inherited retinal diseases are a good choice in terms of gene-based therapies," says Artur Cideciyan, a professor of ophthalmology at the University of Pennsylvania, given that the retina is easily accessible.

But Cideciyan cautions that other approaches for these conditions are also showing promise, and it remains unclear which will turn out to be the best.

"The gene-editing approach is hypothesized to be a 'forever fix,' " he says. "However, that's not known. And the data will have to be evaluated to see the durability of that. We'll have to see what happens."

Original post:
CRISPR Used To Edit Genes Inside A Patient With A Rare Form Of Blindness : Shots - Health News - NPR

GRENOBLE, France and RESEARCH TRIANGLE PARK, N.C., March 02, 2020 (GLOBE NEWSWIRE) -- CYTOO, a leading drug discovery company on muscle disorders, today announced that it has entered into a research collaboration aimed at selecting a gene therapy candidate for a rare muscle disorder withAsklepios BioPharmaceutical, Inc.(AskBio), a clinical-stage, fully integrated adeno-associated virus (AAV) gene therapy company. Under the terms of the agreement, AskBio and CYTOO will work together to develop an AAV-based screening platform derived from patient cells. The goal of the collaboration is to screen and select a preclinical candidate capable of restoring a healthy phenotype from patient cells culturedin vitro.

CYTOO has developed a muscle-on-a-plate platform using patients primary cells, called MyoScreen. MyoScreen is anin vitrosystem in which skeletal muscle cells mimic the physiology, contractile and metabolic functions of human musclein vivoand allow infection by AAV-based gene therapy vectors targeting muscle.

Dr. Philippe Moullier, Chief Scientific Officer, AskBio Europe, said, As a leader in the gene therapy space, the ability to quickly and efficiently screen potential therapeutic candidates will be invaluable. The expertise provided by CYTOO and the MyoScreen platform will potentially give us a better understanding of how those candidates perform in patient cells and improve efficiency throughout the R&D process.

Luc Selig, CYTOOs CEO, added, Gene therapy for muscle disorders is becoming a reality for patients and their families, and we are proud that AskBio has chosen our expertise to investigate a potential new treatment. We have developed MyoScreen as a laboratory model of patient-derived muscle that can be used to screen gene therapy candidates and QC clinical and commercial batches.

The financial terms of the agreement were not disclosed.

About CYTOOCYTOO is a preclinical-stage drug discovery company addressing muscular disorders (NMDs, muscle waste, muscle disuse, metabolic ageing). The company has developed MyoScreen, a versatile and high-throughput muscle-on-a-plate R&D platform, from patient-derived myotubes, that can be used to model any muscle disorder and screen any type of therapeutic candidate. The platform is open to partnering with biotech and pharmaceutical companies and has been the starting point of CYTOOs internal drug discovery program on Duchenne Muscular Dystrophy. Among partners of CYTOO: Daiichi Sankyo, Pfizer, Axcella. CYTOO has offices in Grenoble, France and Bethesda, MD, USA.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process called Pro10 and an extensive AAV capsid and promoter library. Based in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. AskBio maintains a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications with a current clinical pipeline that includes therapeutics for Pompe disease, limb-girdle muscular dystrophy type 2i/R9 and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). Learn more atwww.askbio.comor follow us onLinkedIn.

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CYTOO and AskBio Enter Research Agreement to Screen Gene Therapy Candidates for Rare Muscle Disorder - GlobeNewswire