After reeling back a gene therapy from Sanofi, a North Carolina upstart bags enough money to do something about it – Endpoints News

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After spending some time in Sanofis pipeline, a gene therapy for a genetic eye disease that causes blindness is now back in the hands of its creators at Atsena Therapeutics. And on Wednesday, the North Carolina-based startup landed a $55 million Series A to push it through a Phase I/II trial.

Atsenas founders, power couple Shannon and Sanford Boye, did some early work on the gene therapy at the University of Florida in the early 2000s, before it was licensed by Sanofi. The biotech launched this year with $8 million in funding, then licensed the candidate from Sanofi for an undisclosed amount in July.

Its kind of like a homecoming for them, Atsena CEO Patrick Ritschel said of Shannon and Sanford Boye, who now serve as CSO and chief technical officer, respectively.

The lead candidate is designed to treat GUCY2D-associated Leber congenital amaurosis, or LCA1 a disease that affects the retina and is a leading cause of blindness in children. The drug uses an AAV capsid to deliver the target GUCY2D gene, the idea being that if you provide a replacement gene, it will encode the correct form of the protein.

This is a very good target for gene therapy. Its monogenic. It has a well-defined single gene characteristic. Importantly, the structure of the retina is well-preserved, so we think that the likelihood of success in gene therapy should also be quite high because the cells are there, theyre just not functional, Ritschel said.

The Phase I/II is enrolling 15 participants total over five cohorts, with completion of enrollment expected in late 2021 or early 2022. Then its a 52-week readout, Ritschel said, with a one-year follow-up. The Series A should carry the candidate to an efficacy readout, according to Atsena.

In addition to supporting that trial, the financing will also be used to build out the team, complete manufacturing development for Phase III, and advance the biotechs preclinical programs toward an IND. Atsena has a preclinical candidate for MYO7A-associated Usher syndrome (USH1B) that was also developed in the Boyes labs, and another mystery candidate that it has yet to release details on. And Ritschel is looking to add at least another 20 staffers by 2021.

Spark Therapeutics won approval for their own ophthalmic gene therapy back in 2017. The drug, Luxturna, treats patients with inherited retinal disease due to mutations in both copies of theRPE65 gene. In some cases, a defect in that gene results in LCA.

The field gained a Big Pharma player in October, when the Novartis Institutes for BioMedical Research bought out Cambridge, MA-based startup Vedere Bio, which Cyrus Mozayeni built around academic work from Ehud Isacoff and John Flannery at Cal-Berkeley to find a gene therapy approach to pan-genotypic vision restoration in patients with photoreceptor-based blindness. Novartis paid $150 million, with another $130 million on the table in milestones.

Were really excited, Ritschel said. I think that this is a company that has got a lot of programs, very solid science behind it. Its got great founders, strong support from the University of Florida where a lot of the technology originated from. And I think were looking forward to really building things out.

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After reeling back a gene therapy from Sanofi, a North Carolina upstart bags enough money to do something about it - Endpoints News

After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic – Endpoints News

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When former analyst Rachel McMinn started Neurogene from her apartment around three years ago, she would joke that theyd get office space as soon as her living room table was no longer big enough to hold company meetings.

We lasted about a year before my living room couldnt take it anymore, she said.

With several gene therapies for Batten disease and other lysosomal storage disorders in the preclinical and discovery stage, Neurogene is now bound for the clinic. And on Wednesday, they announced a $115 million Series B to get them there.

Gene therapy has generated so much enthusiasm for patients and families with these devastating disorders, but theres still a lot of science and innovation left on the table, McMinn said.

The CEO said Neurogene will split the Series B funds into four buckets, the first of which is advancing multiple gene therapy programs into the clinic. She anticipates filing the first IND in 2021 for CLN5, a rapidly progressive subtype of Batten disease caused by a variant in the CLN5 gene.

The second so-called bucket for the Series B funds will be expanding the companys portfolio, followed by another bucket for augmenting our resources for our novel technology platform, the CEO said. Then comes manufacturing.

Weve got the ability to make virus in-house, and the money from the financing will allow us to take that vector to the next stage and make GMP quality vector for use in dosing and clinical trials, McMinn said.

Because Neurogene manufactures products in-house, the biotech has gotten around the massive gene therapy manufacturing bottleneck, which has Big Pharma and big biotech spending billions on retrofitted plants and gene therapy factories.

The concept of gene therapy is simple: A viral particle is used to deliver a healthy copy of a gene to a patient with a dysfunctional gene. In the case of Neurogenes CLN5 candidate, viral vectors shuttle a payload into the body designed to make the CLN5 gene.

Over the next year, key milestones will be filing our first IND, completing the refurbishment of our GMP manufacturing facility, (and) advancing our programs towards the clinic, McMinn said. After CLN5, the goal is to file one to two INDs a year, she added.

The CEO previously served as an analyst at Piper Jaffray, Cowen and Bank of America Merrill Lynch, and as chief business and strategy officer at Intercept. During her time as an analyst, McMinn said most people would stay away from investing in neurology companies because drugs inevitably fail.

Theres really been nothing, very little innovation in devastating neurological disorders, for quite a long time, she said, adding that she was inspired to jump into R&D by an older brother who is neurologically impaired.

Neurogene attracted a slate of new and old investors, including EcoR1 Capital which led the round, and Redmile Group, Samsara BioCapital, Cormorant Asset Management, BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, Alexandria Venture Investments, and an undisclosed leading healthcare investment fund.

For me, I really want to make a difference, McMinn said, adding later, Im personally driven by developing something that is life-altering for people that really have no other option.

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After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic - Endpoints News

Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,…

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DUBLIN, Dec. 17, 2020 /PRNewswire/ -- The "Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change provides the strategists, marketers and senior management with the critical information they need to assess the global gene therapy market market.

Major players in the gene therapy market are Novartis AG, Bluebird Bio, Inc., Spark Therapeutics, Inc., Audentes Therapeutics, Voyager Therapeutics, Applied Genetic Technologies Corporation, UniQure N.V., Celgene Corporation, Cellectis S.A. and Sangamo Therapeutics.

The global gene therapy market is expected to decline from $3.22 billion in 2019 to $3.18 billion in 2020 at a compound annual growth rate (CAGR) of -1.30%. The decline is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The market is then expected to recover and reach $6.84 billion in 2023 at a CAGR of 29.09%.

The gene therapy market consists of sales of gene therapy related services by entities (organizations, sole traders and partnerships) that manufacture gene therapy drugs. Gene therapy is used to replace faulty genes or add new genes to cure disease or improve the body's ability to fight disease. Only goods and services traded between entities or sold to end consumers are included.

North America was the largest region in the gene therapy market in 2019.

The gene therapy market covered in this report is segmented by gene type into antigen; cytokine; suicide gene; others. It is also segmented by vector into viral vector; non-viral vector; others, by application into oncological disorders; rare diseases; cardiovascular diseases; neurological disorders; infectious diseases; others, and by end users into hospitals; homecare; specialty clinics; others.

In December 2019, Roche, a Switzerland-based company, completed its acquisition of Spark Therapeutics for $4.3 billion. With this deal, Roche is expected to strengthen its presence in the gene therapy segment, support transformational therapies and increase its product portfolio. Spark Therapeutics is a US-based company involved in gene therapy.

The high prices of gene therapy medicines are expected to limit the growth of the gene therapy market. The pressure to contain costs and demonstrate value is widespread. Political uncertainty and persistent economic stress in numerous countries are calling into question the sustainability of public health care funding. In less wealthy countries, the lack of cost-effective therapies for cancer and other diseases has influenced the health conditions of the population and has led to a low average life expectancy.

Luxturna, a one-time treatment for acquired retinal eye disease, costs $850,000 in the US and 613,410 in the UK, despite a markdown that is applied through Britain's National Health Service. Zolgensma, for spinal muscular atrophy, is valued at $2.1 million in the US and Zynteglo, which focuses on a rare genetic blood disorder, costs $1.78 million, thus restraining the growth of the market.

The use of machine learning and artificial intelligence is gradually gaining popularity in the gene therapy market. Artificial intelligence (AI) is the simulation of human intelligence in machines, which are programmed to display their natural intelligence. Machine learning is a part of AI.

Machine learning and AI help companies in the gene therapy market to conduct a detailed analysis of all relevant data, provide insights between tumor and immune cell interactions, and offer a more accurate evaluation of tissue samples often conflicted between different evaluators. For instance, since January 2020, GlaxoSmithKline, a pharmaceutical company, has been investing in AI to optimize gene therapy and develop off-the-shelf solutions for patients. It is also expected to reduce turnaround time and also the cost of gene therapies.

Key Topics Covered:

1. Executive Summary

2. Gene Therapy Market Characteristics

3. Gene Therapy Market Size And Growth 3.1. Global Gene Therapy Historic Market, 2015 - 2019, $ Billion 3.1.1. Drivers Of The Market 3.1.2. Restraints On The Market 3.2. Global Gene Therapy Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers Of The Market 3.2.2. Restraints On the Market

4. Gene Therapy Market Segmentation 4.1. Global Gene Therapy Market, Segmentation By Gene Type, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global Gene Therapy Market, Segmentation By Vector, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.3. Global Gene Therapy Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.4. Global Gene Therapy Market, Segmentation By End Users, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. Gene Therapy Market Regional And Country Analysis 5.1. Global Gene Therapy Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global Gene Therapy Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Companies Mentioned

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Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,...

Gene Therapy in One Eye Improves Vision in Both Eyes – The Scientist

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I

n a Phase 3 gene therapy trial intended to improve vision among patients with Leber hereditary optic neuropathy, recipients gained somewhat better sight in both eyes even though only one was treated. The results and an investigation into possible explanations for the findings were published December 9 in Science Translational Medicine.

The paper has very strong clinical implications that a single injection maybe is enough for bilateral effects, says Thomas Corydon, who studies ocular gene therapy at Aarhaus University in Denmark and was not involved in the work.

The onset of Leber hereditary optic neuropathy (LHON) is sudden. Patientsusually young menstart losing vision at the center of one eye. Within months, the other eye follows, leaving them legally blind. The disease is caused by a point mutation in the mitochondrial genome that leads to dysfunction and death of retinal ganglion cells, the axons of which make up the optic nerve. About 70 percent of patients have the same mutation, known as MT-ND4.

If you're going to start somewhere, it makes sense to tackle this variant, says Patrick Yu-Wai-Man, an ophthalmologist at the University of Cambridge in the United Kingdom. He and his collaborators, including teams from GenSight Biologics and a group led by University of Pittsburg Medical Center ophthalmologist Jos-Alain Sahel, as well as other groups, previously showed that the point mutation could be corrected in animal models and in cell culture using gene therapy.

Its difficult to get genetic material into the mitochondrial genome because mitochondria have two membranes, an outer and inner membrane, Yu-Wai-Man explains. In the clinical trial, he, Sahel, and colleagues overcame this hurdle by injecting an AAV vector containing a wildtype copy of the ND4gene with an added mitochondrial-targeting sequencea strategy that had already been shown to correctly direct the protein product of ND4 and other mitochondrial genes to the organelle.

Each of 37 patients received the therapeutic virus via a single injection into the vitreous fluid within one eye six to 12 months after the onset of vision loss. They also got a sham treatment in the other eye: a surgeon pressed the eye with a blunt cannula to simulate an injection.

We thought that, if there was going to be an effect, it would be isolated to that eye and then the other one would be the perfect internal control, Yu-Wai-Man tells The Scientist. But as it turns out, that wasnt the case.

With a slight delay in the sham-treated eye, both eyes started to improve. By 96 weeks after treatment, 29 of the patients had gained visual acuity in both eyes and reported increases in their quality of life.

Patients do improve, but, even with the treatment, they still function at a very low level, says Byron Lam, an ophthalmologist at the University of Miami who was not involved in the study. Most of the subjects were still near legal blindness at the end of the study.

To determine how the bilateral effect might be happening, Yu-Wai-Man and colleagues injected the therapeutic virus into one eye of three monkeys. Three months later, they found viral DNA in the noninjected eye and optic nerve. This raises the possibility that the viral vector supplies the wildtype protein in the untreated eye, but its not firm proof.

Finding viral DNA in the untreated eye in primates is a little short of being definitive because DNA expression alone doesnt prove that youre getting a therapeutic effect. Detecting DNA doesnt mean there is mRNA expression or protein production, says Mark Pennesi, an ophthalmologist at Oregon Health & Science University who did not participate in the work.

Previous work has shown that there could be transneuronal spread of the vector, but we also need to keep a critical mind and think that there might be other explanations, agrees Yu-Wai-Man. It could be that injecting the vector in one eye leads to some form of localized inflammation that induces mitochondrial biogenesis, thus making the mitochondria work better, he adds. Another option is that improvement in one eye leads to reorganization in the part of the brain that interprets signals from the eye, which could enhance vision overall.

Clearly, further investigations are needed to understand the underlying mechanisms of how the interocular diffusion of viral DNA vector occurs and whether there are other mechanisms by which the optic nerves directly communicate, Bin Li, an ophthalmologist at Tongji Hospital in China who was not involved in the study, writes in an email to The Scientist.Li explains that his group has also reported that material injected in one eye can reach the other optic nerve.

These findings have implications for how this type of research should be performed in the future, he writes. Theyve shown that contralateral sham-treated eyes cannot serve as true internal control for clinical studies.

When you read this paper, you get a little excited, and then in some ways, you get a little disappointed, because it does look like theres some kind of positive effect with this treatmentthat it does do something more than what would happen with just the natural history of the disease. Unfortunately, the results are confounded by the fact that you treat one eye, but then there is improvement in the untreated control eye, Pennisi tells The Scientist. The question then really becomes . . . why did you get that result?

Along with academic collaborators, Yu-Wai-Man, who consults for GenSight Biologics, will continue to explore this question as they focus on ongoing clinical trials of this therapeutic.

P. Yu-Wai-Man et al., Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy,Science Translational Medicine,doi:10.1126/scitranslmed.aaz7423, 2020.

Correction (December 14): The story has been updated to remove mention of a company that was not involved in the work and to specify which fluid compartment in the eye was injected.The Scientist regrets the error.

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Gene Therapy in One Eye Improves Vision in Both Eyes - The Scientist

Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change – GlobeNewswire

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New York, Dec. 15, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" - https://www.reportlinker.com/p05996809/?utm_source=GNW

The global cell and gene therapy market is expected to decline from $6.68 billion in 2019 to $6.92 billion in 2020 at a compound annual growth rate (CAGR) of 3.61%. The slow growth is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The entire supply chain has been disrupted, impacting the market negatively. The market is then expected to recover and reach $13.23 billion in 2023 at a CAGR of 24.10%.

The cell and gene therapy market consists of sales of cell and gene therapies by entities (organizations, sole traders and partnerships) that develop cell and gene therapies. Cell therapy refers to the transfer of intact, live cells that are originated from autologous or allogenic sources and gene therapy refers to the introduction, removal, or change in the genome for treating diseases. The market consists of revenue generated by the companies developing cell and gene therapy products by the sales of these products.

North America was the largest region in the cell and gene therapy market in 2019. It is also expected to be the fastest-growing region in the forecast period.

In December 2019, Roche, a Swiss multinational healthcare company, acquired Spark Therapeutics for $4.3 billion. The acquisition supports the commitment of Roche to bring transformational therapies and innovative approaches to people with serious illnesses. Spark Therapeutics will continue to work within the Roche Group as an independent company. Spark Therapeutics, headquartered in Philadelphia, is a fully integrated commercial company involved in the discovery, production, and distribution of gene therapies for genetic disorders including blindness, hemophilia, lysosomal storage, and neurodegenerative diseases.

The cell and gene therapy market covered in this report is segmented by product into cell therapy; gene therapy and by application into oncology; dermatology; musculoskeletal; others.

Limited reimbursements preventing patients from receiving treatments are expected to limit the growth of cell and gene therapy (CGT market. In 2019, Trinity Life Sciences, a life sciences solution provider, researched national and large regional commercial health insurance plans in the US. It found that the confluence of increasing price, patient volume and number of CGTs on the market is likely to change the reimbursement model for CGTs and impact payer budgets by 5-10%. Payers realize that financing needs to be generated for cost management due to the uncertainty surrounding reimbursement of ancillary costs. Limited reimbursements and uncertain insurance plans are preventing patients from receiving high-cost CGT, which is expected to limit market growth.

Chimeric antigen receptor (CAR) T-cell therapy is shaping the cell and gene therapy (CGT) market. (CAR) T-cell therapy is a combination of cell and gene therapy in which T cells are collected from the patients blood and are genetically engineered to produce modified receptors at their surface, known as chimeric antigen receptors (CARs). These modified T cells with special structures (receptors) are reinfused into the patient. Then, the modified receptors of T cell help in targeting the surface antigen of the cancer cell that ultimately results in the killing of tumor cells in patients. In 2020, the US-FDA approved Bristol-Myers Squibbs two CAR-T cell therapies to treat lymphoma and multiple myeloma and is set to be launched. Currently, FDA approved CAR-T cell therapy treatments like Tisagenlecleucel for the treatment of B-cell precursor acute lymphoblastic leukemia (ALL) in children and Axicabtagene ciloleucel for the treatment of adult patients with relapsed or refractory large B-cell lymphoma.

Steady investment and consolidation in cell and gene therapies contributed to the growth of the cell and gene therapy (CGT) market. After recognizing the potential of the CGT market, 16 out of the 20 largest biopharma companies by revenue, added CGT products to their portfolio. For instance, Merck invested $109 million in viral vector and gene therapy manufacturing in April 2020. Moreover, 12% of industrial clinical pipeline products and at least 16% of preclinical pipeline products consist of CGT. Steady investment and consolidation in CGT production capacity led to an increase in production capacity and also contributed to the growth of the market.Read the full report: https://www.reportlinker.com/p05996809/?utm_source=GNW

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Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change - GlobeNewswire

Passage Bio Invests In Gene Therapy Manufacturing R&D Site – Contract Pharma

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Passage Bio, Inc., a genetic medicines company focused on therapies for rare, monogenic central nervous system (CNS) disorders, has entered into a long-term lease to support Chemistry, Manufacturing and Controls (CMC) lab operations for the companys gene therapy programs. The new lab, scheduled to open in 2Q21 at the Princeton West Innovation Campus in Hopewell, NJ, will initially focus on state-of-the-art analytical capabilities, clinical assay development and validation, biomarker assay validation and clinical product testing to support both viral vector manufacturing and clinical development.The opening of the CMC lab is part of Passage Bios strategy to expand its internal manufacturing capabilities to support its lead gene therapy programs as they move into the clinic and advance toward commercialization. The CMC lab complements the recent opening of Passage Bios dedicated CGMP manufacturing suite at Catalent. These investments provide the company with the foundation for an integrated manufacturing supply chain with capabilities to advance multiple gene therapy programs to support clinical trials worldwide.The 62,000 sq.-ft. lab space is intended to support analytics, process development, quality control and pilot manufacturing. The 1.2 million-sq.-ft., multi-purpose research and development and biologic/pharmaceutical manufacturing campus also provides Passage Bio with expansion opportunities for additional lab space and CGMP manufacturing operations. Passage Bio plans to add more than 20 new positions in 2021 at the new lab.

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Passage Bio Invests In Gene Therapy Manufacturing R&D Site - Contract Pharma

Gene Therapy Market Worth USD 35.67 Billion at 33.6% CAGR; Rising Prevalence of Spinal Muscular Atrophy to Augment Growth: Fortune Business Insights -…

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Pune, India, Dec. 14, 2020 (GLOBE NEWSWIRE) -- The report mentions that the Gene Therapy Market size was USD 3.61 billion in 2019 and is projected to reach USD 35.67 billion by 2027, exhibiting a CAGR of 33.6% during the forecast period. The global gene therapy market is set to gain momentum from the rising incidence of different types of cancer. The field of this therapy is undergoing several technological advancements that would help in treating cancer in those patients who are at high risks of getting affected by this disease through genetic mutations. In 2019, the U.S. generated USD 2.16 billion in terms of revenue. The country is expected to dominate throughout the coming years stoked by the increasing usage of advanced gene therapies for the treatment of rare conditions.

KEY INDUSTRY DEVELOPMENTS:

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Increasing Innovations & Research Activities to Boost Growth

The U.S Food and Drug Administration (FDA) stated that it is expecting to receive more than 200 applications of this therapy by the end of 2020. This showcases that the rising number of research studies and innovations in this field would affect the gene therapy market growth positively in the near future. In North America, almost 208 companies are currently operating in this market. In addition to this, the Alliance for Regenerative Medicine declared that as of 2018, approximately 259 potential drug candidates are under Phase I clinical trials across the globe.

However, the outbreak of the COVID-19 pandemic is presently impacting the field of research. According to the director of the Office of Tissues and Advanced Therapy (FDA) named Wilson Brayan, nowadays the officials are prioritizing only those drugs that are associated with coronavirus.

To get to know more about the short-term & long-term impact of COVID-19 on this market, please click here: https://www.fortunebusinessinsights.com/industry-reports/gene-therapy-market-100243

The U.S. to Dominate Owing to Presence of Favorable Policies

In 2019, the U.S. generated USD 2.16 billion in terms of revenue. The country is expected to dominate throughout the coming years stoked by the increasing usage of advanced gene therapies for the treatment of rare conditions.

Besides, the presence of favorable reimbursement policies and guidelines would also help in propelling the market growth here. As this type of treatment is not legal in several developing nations, industry giants are emphasizing on the U.S. for launching their products.

Europe, on the other hand, is anticipated to grow significantly backed by the adoption of unique treatment options. Asia Pacific is set to hold a comparatively lower share on account of the decreasing usage of gene therapy because of its expensive nature.

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List of Key Players operating in Gene Therapy Market:

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Global Gene Therapy Market Segmentations:

By Application

Oncology

Neurology

Others

By Vector Type

Viral

Non-viral

By Distribution Channel

Hospitals

Clinics

Others

By Geography

U.S.

Europe (U.K., Germany, France, Italy, Spain, and Rest of Europe)

Asia-Pacific (Japan, China, and Rest of Asia- Pacific)

Rest of World

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Gene Therapy Market Worth USD 35.67 Billion at 33.6% CAGR; Rising Prevalence of Spinal Muscular Atrophy to Augment Growth: Fortune Business Insights -...

Though Promising, Gene Therapies Face Durability And Reimbursement Headwinds – Forbes

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WALPOLE , MA - AUGUST 6: Estelle Lemieux, a 21-month-old with spinal muscular atrophy, practices ... [+] using her new wheelchair outside of her home in Walpole, MA on Aug. 6, 2019. Estelle will be getting a treatment of Zolgensma after her insurer, Aetna, decided to cover the $2.1 million drug. (Photo by Jessica Rinaldi/The Boston Globe via Getty Images)

The promise of gene therapy is to cure diseases associated with faulty or missing genes. Theres enormous potential. Just this month, at the annual American Society of Hematology meeting, it was shown that gene therapy stops bleeding in hemophilia. Researchers reported that a single injection of a viral-mediated gene therapy vector decreases the bleeding rate among patients with Factor IX-related hemophilia B by 91% over a 6 month period.

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Ideally, gene therapies address the root causes of disease with a single curative dose. If they can replace a lifetime of expensive maintenance treatments this may lead to cost savings in the long run. Yet, the high upfront costs, uncertainty surrounding long-term durability, and adverse events have led to some concerns among payers and regulators.

Pharmaceutical firms deploy multiple approaches to pursuing curative gene therapy, including:

These approaches build on advances in basic science. Companies involved in gene therapy research and development include mid-size and large firms. Among other large pharmaceutical firms, Bayer is establishing a cell and gene therapy platform within its pharmaceuticals division. The company aims to deploy the platform in as many indications as possible.

Novel drug development is invariably a risky venture. The issue of risk is further amplified in gene therapy. Promising therapies face unexpected challenges. For example, in a surprise decision this fall, the Food and Drug Administration (FDA) rejected BioMarins license application for its gene therapy to treat severe hemophilia A. According to the FDA, valoctocogene roxaparvovec gene therapy, is not ready for approval in its present form. The FDA changed its data requirements for the application. The agency is now requesting that the sponsor BioMarin provide two years of data from the companys ongoing Phase 3 study of the therapy.

While development challenges will persist, payment hurdles may be equally difficult to overcome. Should many of the gene therapies in the pipeline be approved in the coming decade the budgetary impact burden on payers could become overwhelming. Payer concerns stem in part from there being hundreds of gene therapies in clinical development,across a wide range of therapeutic categories, including among others, cardiovascular disease, Parkinsons, various inherited disorders, different types of cancer, viruses such as HIV, and blood diseases like sickle cell anemia.

The churn or turnover at U.S. insurers - as beneficiaries frequently switch plans - lowers the potential return on investment for payers. So, being saddled with high upfront costs without necessarily experiencing the downstream long-term benefits of gene therapies is a considerable problem for which a structural solution has not yet been found.

The payer assumes all the risk with fixed, static pricing. And, the payer isnt able to properly assess that risk, given that clinical development of gene therapies has, thus far, mostly included only very small numbers of patients. Therefore, the real-world benefits and risks remain unclear at the time of approval. Clearly, given the uncertainties regarding long-term durability of gene therapies as well as the potential for toxicity and other adverse effects to patients, a dynamic pricing structure is not only desirable but essentially required for these treatments.

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Value-based contracts

In what appear to be harbingers of new ways to finance gene therapies and potentially turn fortunes around of therapies lagging in uptake, drug manufacturers are offering - and in some cases payers have been amenable to - indication-specific pricing arrangements, value-based contracts, and installment plans.

For example, in 2018, the FDA approved the gene therapy Luxturna. This treatment holds the promise to restore functional vision to the blind. The sponsor, Spark Therapeutics, set its products price at $425,000 per eye. Harvard Pilgrim entered into a unique outcomes-based contract with Spark Therapeutics. In the deal, Harvard Pilgrim pays for Luxturna, but gets certain refunds if the treatment wears off after a defined period of time. The full details of the contract are confidential. What is known, however, is that because of federal regulations, known as Medicaid best price rules, the maximum refund cannot exceed 23.1%, or the amount Spark Therapeutics is required to offer Medicaid programs. Spark Therapeutics did request that the Centers for Medicare and Medicaid Services (CMS) offer ways to work around the Medicaid best price requirement, in order for it to be able to accept installment payments and provide insurers deeper refunds or rebates in case the product doesnt meet certain targets.

Novartis Gene Therapies has been working closely with payers to create five-year outcomes-based agreements and novel pay-over-time options for the Zolgensma therapy, indicated for spinal muscular atrophy. The sponsor asserts that the treatment is cost-effective, even when priced at $2.125 million per patient. The installment plans would spread out payments over five years. In addition, the sponsor would offer a refund if a patient dies or the treatment otherwise fails within that period.The current alternative to Zolgensma is BiogensSpinraza, which patients take for the duration of their lifetime. The costs of Spinraza are approximately $4 million over a 10-year span.

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In 2019, BluebirdBio told investors it was seeking value-based installment plan contracts to reimburse its sickle cell anemia product LentiGlobin for transfusion-dependent beta-thalassemia. The installments would be paid over a period of up to five years.

After an initial charge, Bluebird Bio would only get reimbursed if the one-time infusion benefits patients. This implies that up to 80% of the cost of LentiGlobin would only be made if there is treatment success. And this success would then be measured and tracked in patient registries maintained by payers.

As part of its contracting preparations, Bluebird Bio has sought ways to bypass Medicaid best price rules; for example, waivers to establish an exemption. The company has also pursued a resolution to the issue of portability - when patients change insurers - by way of a mutual recognition strategy across payers.

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But, now the FDA wants Bluebird Bio to provide additional information on the manufacturing process it will use as it transitions the product, LentiGlobin, from clinical trials to commercial production. This will push back the timing of execution of contracts until LentiGlobin gets approved by FDA, which may not be until 2022 or later.

Across the various contract constructs described, payments can be administered in different ways that are not mutually exclusive:

Reimbursement of pharmaceutical products generally happens on a per-unit basis, which spreads out costs over years. But, the cost of a gene therapy is much more concentrated, possibly all upfront in a single payment. Such high upfront one-time costs make it harder for payers to underwrite the risk of full payment for one therapy, let alone the entire range of gene therapies that may be coming to market shortly. Therefore, a combination of installment plans and value-based contracting arrangements will likely be the wave of the future for gene therapy reimbursement.

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Though Promising, Gene Therapies Face Durability And Reimbursement Headwinds - Forbes

What is gene therapy? – The Star Online

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I have heard about people using genes to treat diseases nowadays, but I am not sure what this gene therapy means.

Gene therapy involves trying to alter or modify the genes inside your bodys cells in order to treat or stop a disease.

Since 2017, the US Food and Drug Administration (FDA) has approved three different types of gene therapy.

Maybe we can start at the beginning: what are genes?

Genes are the basic physical and functional unit of heredity.

Our genes are made out of DNA (deoxyribonucleic acid).

Each person has two copies of each gene one inherited from your mother and the other inherited from your father.

Each human being has around 20,000 to 25,000 genes.

These genes code for the way your body and mind are structured.

Some genes act as instructions (a blueprint) for your body to make various proteins, which in turn form your cells and organs, and the enzymes and hormones that regulate your body.

Other genes do not code for proteins.

Most genes are the same for all human beings, which is why we all look like human beings (and not a kangaroo, fish, bird or an alien)!

However, just under 1% of our genes vary slightly between each person.

That is why we have different races, heights, propensity for different diseases, curly or straight hair, etc.

These small differences also contribute to why we all look different from one another.

Genes that dont work as they should also cause diseases in the human body.

What types of diseases are caused by faulty genes?

These are what we call genetic disorders.

A genetic disease is any type of disease caused by an abnormality in our genetic blueprint.

This abnormality can range from very minor to significantly major.

What we consider minor is, for example, a small mutation in the DNA of a single gene resulting in the change of a single base protein.

What we consider major is a gross chromosomal abnormality, such as the addition of a whole chromosome or the subtraction of one.

Some genetic disorders are inherited from our parents.

Others are caused by mutations due to our environment.

Examples of single gene disorders, which are caused by the alteration of just one gene in our bodies, are:

Examples of multifactorial inheritance, which are caused by a combination of environmental factors and mutations in many of our genes, are:

If we inherited these genes from our parents, then how can we possibly modify or alter them? This sounds terribly like science fiction.

We are rapidly approaching that era where what used to be science fiction could become part of our everyday life.

In gene therapy, scientists can:

How do they do this? Do they have to harvest my cells? Im scared just thinking about it!

Many of the vectors are viruses, especially adenoviruses (not coronaviruses!).

Viruses have a natural ability to deliver genetic material into our cells.

After all, their main purpose is to attach themselves to cells and reproduce themselves.

Sometimes, the vector or virus is injected straight into our bodies, where they will deliver the gene that will modify our cells.

They are injected straight into the part of our body that has those defective cells.

Other times, we have to harvest healthy tissue from our body that needs to be modified.

These are usually tissues containing immune cells or stem cells, e.g. blood or bone marrow.

These tissue samples are then taken to the lab and specific cells are separated out.

The viral vector containing the corrective gene is then introduced to the harvested cells in the lab.

The modified cells are left to multiply, and then injected back into us.

Once inside our bodies, they will continue to multiply and eventually treat the disease or correct the defect within us.

Learn more about gene therapy in the next Tell Me About column on Dec 31 (2020).

Dr YLM graduated as a medical doctor, and has been writing for many years on various subjects such as medicine, health, computers and entertainment. For further information, email starhealth@thestar.com.my. The information contained in this column is for general educational purposes only. Neither The Star nor the author gives any warranty on accuracy, completeness, functionality, usefulness or other assurances as to such information. The Star and the author disclaim all responsibility for any losses, damage to property or personal injury suffered directly or indirectly from reliance on such information.

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What is gene therapy? - The Star Online

They thought their gene therapy failed. Instead, it spawned a medical mystery – Endpoints News

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Jos-Alain Sahel was on a rare vacation in Portugal in the spring of 2018 when his phone rang with grim news: The gene therapy he had worked on for a decade, a potential cure for a rare form of blindness, had failed in a pivotal trial.

In the first minute, I was very disappointed, Sahel says. I said, well OK, its not working.

A failed trial in drug development is crushing but not unexpected, a tradeoff of doing business in biology. You examine the full data, go back to the drawing board and either abandon the effort or tweak and try again. Sahel, founder of four companies and the longtime head of the Vision Institute of Paris, was used to the process. But this time, when the full data came, he was bewildered.

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They thought their gene therapy failed. Instead, it spawned a medical mystery - Endpoints News

Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) – Canada NewsWire

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Zolgensma is a gene therapy designed to address the genetic root cause of SMA by replacing the missing or defectiveSMN1gene1.It is administered during an intravenous (IV) infusion, delivering a new working copy of the SMN1 gene into a patient's cells, halting disease progression and restoring production of SMN protein1.

"SMA can be a devastating diagnosis for families to receive. Without treatment, many children would not be able to meet important developmental milestones like lifting their head, sitting or walking.Even breathing and swallowing can become difficult in the severe, infant-onset form of this disease," said Dr. Hugh McMillan, Pediatric Neurologist at the Children's Hospital of Eastern Ontario in Ottawa."The approval of Zolgensma in Canada offers children an opportunity to maximize their developmental potential from this one-time therapy.The decision to treat based upon weight may allow children diagnosed slightly later to also benefit from this therapy."

"When I first started diagnosing SMA, I couldn't have imagined that we would see such scientific advancements," said Dr. Nicolas Chrestian, Chief of Paediatric Neurology, specialized in neuromuscular disorders at Centre Hospitalier Mre Enfant Soleil, Universit Laval in Qubec City. "Zolgensma offers, in a single dose, the possibility of halting the progression of this degenerative condition that can rob children of regular developmental milestones."

In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3. Without a functionalSMN1gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking2. Left untreated, muscles become progressively weaker2,3. In the most severe form, this eventually leads to paralysis and ultimately permanent ventilation or death by age 2 in more than 90%of cases4.

"The SMA community is thrilled to have another treatment option to offer hope to families grappling with an SMA diagnosis. The approval of Zolgensma couldn't come soon enough. We will continue to advocate until everyone who needs access to treatment can benefit from innovations like this," said Susi Vander Wyk, Executive Director, CureSMA Canada.

"Today's announcement about the Canadian approval of Zolgensma is a significant milestone in our journey to reimagine medicine by changing the treatment paradigm for children with SMA." said Andrea Marazzi, Country Head, Novartis Pharmaceuticals Canada. "Our commitment to the SMA community truly comes to life when those that could benefit most from Zolgensma can access it. This is why we continue to work collaboratively with the pan-Canadian Pharmaceutical Alliance, provinces and territories to make this happen as quickly as possible."

The efficacy and safety data supporting the approval of Zolgensma in treating pediatric patients with SMA are derived from completed and ongoing open-label, single-arm, clinical trials in patients with infantile-onset SMA and 2 copies of SMN2 gene; and presymptomatic genetically diagnosed SMA and 2 or 3 copies of SMN2 gene1.

Zolgensma is the only gene therapy approved by Health Canada for the treatment of SMA1. Thirteen treatment sites have been identified in leading healthcare institutions with SMA expertise. The sites are located in: Vancouver, BC; Edmonton, AB; Calgary, AB; Saskatoon, SK; Winnipeg, MB; London, ON; Hamilton, ON; Toronto, ON; Ottawa, ON; Montreal, QC; Quebec City, QC; Halifax, NS.

About Spinal Muscular AtrophySMA is the leading cause of genetic infant death2. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities5.This is why it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression6.Early diagnosis is especially critical in the most severe form, where motor neuron degeneration starts before birth and escalates quickly5. Newborn screening for SMA is currently being implemented in Ontario and piloted in Alberta7,8.

About Novartis in Gene Therapy and Rare DiseaseNovartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

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

About Novartis globallyNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at https://www.novartis.com.

Zolgensma is a registered trademark of Novartis Gene Therapies.

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for thein vivogene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Gnthon forin vivodelivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

References

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Julie Schneiderman, +1 514 633 7873, E-mail: [emailprotected]

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Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire

Gene Therapy Injection in One Eye Improves Vision in Both – Technology Networks

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In a landmark phase 3 clinical trial, the international team, coordinated by Dr Patrick Yu-Wai-Man from the University of Cambridge and Dr Jos-Alain Sahel from the University of Pittsburgh and Institut de la Vision, Paris, successfully treated 37 patients suffering from Leber hereditary optic neuropathy (LHON). Subject to further trials, the treatment could help thousands of people across the world to regain and retain some of their sight.

The study, published today in the journal Science Translational Medicine, indicates that 78% of treated patients experienced significant visual improvement in both eyes. It suggests that the improvement in vision in untreated eyes could be due to the transfer of viral vector DNA from the injected eye.

LHON affects a specific type of retinal cells, known as retinal ganglion cells, causing optic nerve degeneration and rapidly worsening vision in both eyes. Within a few weeks of disease onset, the vision of most people affected deteriorates to levels at which they are considered legally blind. Visual recovery occurs in less than 20% of cases and few achieve vision better than 20/200 (largest letter on a standard eye chart). LHON affects approximately 1 in 30,000 people, mostly men, with symptoms usually emerging in their 20s and 30s. The majority of patients carry the m.11778G>A mutation in the MT-ND4 gene. Existing treatment for this blinding optic neuropathy remains limited.

As someone who treats these young patients, I get very frustrated about the lack of effective therapies, said senior investigator Dr Sahel, a professor of ophthalmology at the University of Pittsburgh. These patients rapidly lose vision in the course of a few weeks to a couple of months. Our study provides a big hope for treating this blinding disease in young adults.

The researchers injected rAAV2/2-ND4 a viral vector containing modified cDNA into the vitreous cavity at the back of one eye of 37 patients who had suffered vision loss for between 6 to 12 months. Their other eye received a sham injection. The technology, called mitochondrial targeting, was developed by the Institut de la Vision in Paris, France, and licensed to GenSight Biologics.

International coordinating investigator and neuro-ophthalmologist Dr Yu-Wai-Man, from Cambridges Department of Clinical Neurosciences and Moorfields Eye Hospital, London, said: We expected vision to improve in the eyes treated with the gene therapy vector only. Rather unexpectedly, both eyes improved for 78% of patients in the trial following the same trajectory over 2 years of follow-up.

Treated eyes showed a mean improvement in best-corrected visual acuity (BCVA) of 15 letters on an ETDRS chart, representing three lines of vision, while a mean improvement of 13 letters was observed in the sham-treated eyes. As some patients were still in the dynamic phase of the disease process upon enrolment, the visual gain from the nadir (worst BCVA for each eye) was even larger, reaching 28.5 letters for the treated eyes and 24.5 letters for sham-treated eyes.

Dr Yu-Wai-Man said: By replacing the defective MT-ND4 gene, this treatment rescues the retinal ganglion cells from the destructive effects of the m.11778G>A mutation, preserving function and improving the patients visual prognosis. The outcomes can be life-changing.

The researchers found that treated eyes were around three times more likely to achieve vision better than or equal to 20/200. Patient-reported outcome measures evaluated using the National Eye Institute Visual Function Questionnaire-25 (NEI VFQ-25) also confirmed the positive impact of treatment on quality of life and psychosocial well-being.

The researchers then conducted a study in cynomolgus macaques to investigate how the treatment of one eye could bring about improvement in the other. Macaques have a visual system similar to that of humans, which allows the distribution and effects of the gene therapy vector to be studied in much greater detail. Unilateral injection of rAAV2/2-ND4 was administered and after three months, tissues from various parts of the eye and the brain were analyzed to detect and quantify the presence of viral vector DNA using a transgene-specific quantitative PCR assay.

Viral vector DNA was detected in the anterior segment, retina and optic nerve of the untreated eye. The unexpected visual improvement observed in the untreated eyes could therefore reflect the interocular diffusion of rAAV2/2-ND4. Further investigations are needed to confirm these findings and whether other mechanisms are contributing to this bilateral improvement.

Dr Yu-Wai-Man said: Saving sight with gene therapy is now a reality. The treatment has been shown to be safe and we are currently exploring the optimal therapeutic window.

Our approach isnt just limited to vision restoration, added Dr Sahel. Other mitochondrial diseases could be treated using the same technology.

Reference: Yu-Wai-Man P, Newman NJ, Carelli V, et al.Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy.Sci. Transl. Med. 2020;12(573). doi:10.1126/scitranslmed.aaz7423

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 Injection in One Eye Improves Vision in Both - Technology Networks

Worldwide Gene Therapy Industry to 2025 – Cancer is Expected to Hold Significant Market Share in the Indication Segment – ResearchAndMarkets.com -…

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TipRanks

After a year that most of us want to forget, 2021 is shaping up to start with stability and an even keel. The election is safely behind us, the new Biden Administration promises a no drama approach, a closely divided and hyper-partisan Congress is unlikely to enact any sweeping legislation, reform or otherwise, and COVID vaccines are ready for distribution. Its a recipe for a calm news cycle.Which makes it a perfect time to buy into the stock market. Investors can read the tea leaves, or study the data whatever their preferred mode of stock analysis and use this period of calm to make rational choices on the stock moves. Using the TipRanks database, weve pulled up three stocks that present a bullish case. All three meet a profile that should interest value investors. They hold unanimous Strong Buy consensus ratings, along with a perfect 10 from the Smart Score. That score, a unique measure, evaluates a stock based on 8 factors with a proven high correlation to future overperformance. A 10 score indicates a strong likelihood that the stock will rise in the coming year. And finally, all three of these stocks present with double-digit upside potentials, indicating that they are still undervalued.UMH Properties (UMH)Well start in the real estate investment trust (REIT) sector, with UMH Properties. This company, which started out after WWII in the mobile home industry, later become the premier builder of manufactured housing. Today, UMH owns and manages a portfolio of 124 manufactured housing communities, spread across 8 states in the Northeast and Midwest, and totaling well over 23,000 units. As a REIT, UMH has benefitted from the nature of manufactured houses as affordable options in the housing market. UMH both sells the manufactured homes to residents, while leasing the plots on which the properties stand, and leases homes to residents. The companys same-property income, a key metric, showed 8.6% year-over-year increase in the third quarter.Also in the third quarter, UMH reported a 16% yoy increase in top line revenue, showing $43.1 million compared to $37.3 million in the year-ago quarter. Funds from Operations, another key metric in the REIT sector, came in at 11 cents per share, down from 14 cents in 3Q19. The decrease came as the company redeemed $2.9 million in Series B Preferred Stock.REITs are required to return income to shareholders, and UMH accomplishes this with a reliable dividend and a high yield of 4.7%. The payment, at 18 cents per common share, is paid quarterly and has been held stable for over a decade.Compass Point analyst Merrill Ross believes the company is in a sound position to create value for both households and shareholders."We believe that UMH has proven that it can bring attractive, affordable housing to either renters or homeowners more efficiently than has been possible with vertical rental housing. As UMH improves its cost of funds, it can compete more effectively with other MH community owners in the public and private realms, and because it has a successful formula to turn around undermanaged communities, we think that UMH can consolidate privately-owned properties over the next few years to build on its potential for value creation," Ross opined.To this end, Ross rates UMH a Buy, and her $20 price target implies a 25% one-year upside. (To watch Rosss track record, click here)Overall, the unanimous Strong Buy on UMH is based on 5 recent reviews. The stock is selling for $15.92, and the $18.40 average price target suggests it has room for 15% growth from that level. (See UMH stock analysis on TipRanks)Laird Superfood (LSF)Laird Superfood is a newcomer to the stock markets, having gone public just this past September. The company manufactures and markets a range of plant-based, nutrient-dense food additives and snacks, and is most known for its line of specialized non-dairy coffee creamers. Laird targets customers looking to add nutrition and an energy boost to their diet.Since its September IPO, the company has reported Q3 earnings. Revenue was strong, at $7.6 million, beating the forecast by over 26% and coming in 118% above the year-ago figure. The company also reported a 115% yoy growth in online sales. Ecommerce now makes up 49% of the companys net sales no surprise during the corona year.The review on the stock comes from Robert Burleson, a 5-star analyst from Canaccord. Burleson reiterates his bullish position, saying, We continue to view LSF as an attractive platform play on strong demand trends for plant-based, functional foods, noting LSFs competitively differentiated omni-channel approach and ingredients ethos. Over time, we expect LSF to be able to leverage its brand and vertically integrated operation into success in a broad range of plant-based categories, driving outsized top-line growth and healthy margin expansion.Burleson rates LSF shares a Buy alongside a $70 price target. This figure indicates his confidence in ~63% growth on the one-year horizon. (To watch Burlesons track record, click here)Laird has not attracted a lot of analyst attention, but those who have reviewed the stock agree with Burlesons assessment. LSF has a unanimous Strong Buy analyst consensus rating, based on 3 recent reviews. The stocks $62.33 average price target suggests room for ~39% upside in the coming year. (See LSF stock analysis on TipRanks)TravelCenters of America (TA)Last but not least is TravelCenters of America, a major name in the transportation sector. TravelCenters owns, operates, and franchises full-service highway rest stops across the US an important niche in a country that relies heavily on long-haul trucking, and in which private car ownership has long encouraged the road trip mystique. TAs network of rest stops offers travelers convenience stores and fast-food restaurants in addition to gasoline and diesel fuel and the expected amenities.The corona crisis has been hard time for TA, as lockdown regulations put a damper on travel. The companys revenues bottomed out in Q2, falling to $986 million, but rose 28% sequentially to hit $1.27 billion in Q3. EPS, at 61 cents, was also strong, and showed impressive 165% year-over-year growth. These gains came as the economy started reopening and with air travel still restricted, automobiles become the default for long distance, a circumstance that benefits TravelCenters. Covering TravelCenters for BTIG is analyst James Sullivan, who rates the stock a Buy, and his $40 price target suggests a 22% upside over the coming year. (To watch Sullivans track record, click here)Backing his stance, Sullivan noted, "TA is in the process of moving on from a series of unsuccessful initiatives under the prior management team. The current new management team has strengthened the balance sheet and intends to improve operations through both expense cuts and revenue-generating measures which should boost margins [...] While we expect the 2020 spend to be focused on non-revenue generating maintenance and repair items, we expect in 2021 and beyond that higher spending should generate good ROI All in all, TravelCenters shares get a unanimous thumbs up, with 3 Buys backing the stocks Strong Buy consensus rating. Shares sell for $32.87, and the average price target of $38.33 suggests an upside potential of ~17%. (See TA stock analysis on TipRanks)To find good ideas for stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Worldwide Gene Therapy Industry to 2025 - Cancer is Expected to Hold Significant Market Share in the Indication Segment - ResearchAndMarkets.com -...

Locanabio Raises $100 Million to Advance RNA-Targeted Gene Therapies – BioSpace

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San Diego-based Locanabio secured $100 million in a Series B financing round that will be used to advance the companys portfolio of novel RNA-targeted gene therapies for neurodegenerative, neuromuscular and retinal diseases.

The funding will support pre-clinical and clinical development of its gene therapy treatments for diseases such as Huntington's disease, myotonic dystrophy type 1, genetic forms of amyotrophic lateral sclerosis and retinal disease, the company said.

Locanabio has a unique approach to gene therapy. The company has combined two validated gene therapy and RNA modifications to treat diseases. Locanabio uses a gene therapy vector to deliver an RNA-targeting protein tipped with an RNA-modifying enzyme. Through targeting RNA, the company said its approach avoids the risk of off-target effects in DNA and is suited to address many diseases linked to dysfunctional processing of RNA.

The $100 Million Series B builds on $55 million the company secured in a Series A last year. Chief Executive Officer Jim Burns, who joined Locanabio one year ago, said the financing round will allow the company to advance several of the companys most promising programs into IND-enabling studies in 2021. The financing will also allow the company to continue to advance its RNA-targeting platform, which has the potential to be a major new advance in medicine that can bring hope to patients with many devastating genetic diseases, Burns said.

While all of Locanabios assets are still in the research phase, its most advanced is a therapy for myotonic dystrophy type 1 (DM1), a genetic neuromuscular disorder caused by a mutation in the DMPK gene that results in trinucleotide (CUG) repeat expansion in the expressed RNA. Locanabios DM1 program targets and destroys the toxic CUG repeats, according to company information. Earlier this year, as BioSpace previously reported, Locanabio published a paper demonstrating the benefits of its technology as a potential one-time treatment of DM1.

The financing round was led by Vida Ventures LLC with participation from RA Capital Management, Invus, Acuta Capital Partners, an investment fund associated with SVB Leerink Prior Locanabio investors ARCH Venture Partners, Temasek, Lightstone Ventures, UCB Ventures and GV, also participated in the financing round.

"We are pleased that a team of highly sophisticated investors led by Vida Ventures has joined in this financing round, further validating our progress in research and the significant potential of our unique RNA-targeting platform, Burns said in a statement.

With the Series B, Rajul Jain, director of Vida Ventures, joined Locanabio's board of directors. Before Vida Ventures, Jain served on the executive team and headed development for Kite Pharma and was previously global development lead for Amgen.

The unique approach in RNA targeting using gene therapy to deliver RNA binding proteins developed by Locanabio represents the next frontier of genetic medicine with the ability to target the root cause of a range of genetic diseases, Jain said in a statement. They have built a strong management team to execute this bold vision and we are proud to support them.

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Locanabio Raises $100 Million to Advance RNA-Targeted Gene Therapies - BioSpace

Taysha Gene Therapies Set to Join Russell 2000 Index on December 21, 2020 – Business Wire

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DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it is set to join the Russell 2000 Index, effective Monday, December 21, 2020, as part of the indexs quarterly initial public offering (IPO) additions.

The Russell 2000 Index measures the performance of the small-cap segment of the U.S. equity market. The index is a subset of the Russell 3000 Index and represents approximately 10 percent of the total market capitalization of that index. Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Approximately $9 trillion in assets are benchmarked against Russells U.S. indexes. Russell indexes are part of FTSE Russell, a leading global index provider.

For more information on the Russell 2000 Index and the Russell indexes IPO additions, please visit the Russell U.S. Index IPO Additions section on the FTSE Russell website.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

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FTSE Russell is a leading global index provider creating and managing a wide range of indexes, data and analytic solutions to meet client needs across asset classes, style and strategies. Covering 98% of the investable market, FTSE Russell indexes offer a true picture of global markets, combined with the specialist knowledge gained from developing local benchmarks around the world.

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Taysha Gene Therapies Set to Join Russell 2000 Index on December 21, 2020 - Business Wire

Rare Disease Gene Therapy Market: Increasing cases of genetic diseases to drive the market – BioSpace

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Rare Disease Gene Therapy Market: Snapshot

The global rare disease gene therapy is expected to witness a significant growth in the forecast period, 2020-2030 on account of the increasing cases of genetic diseases worldwide. Gene therapy is relevant to rare disease patients and has improved the wellbeing and personal satisfaction of more seasoned kids and youthful grown-ups with X-SCID. These kids are expected to experience complex clinical issues in the wake of getting live-sparing bone marrow transfers to treat the condition.

The report offers insights into the market emphasizing on factors boosting, repelling, challenging, and creating opportunities for the market in the forecast period. It also discusses the impact of the COVID-19 pandemic on the overall market. It also discusses the table of segmentation in details and lists the names of the prominent players functioning in the market.

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The global rare disease gene therapy market is categorized on the basis of type, indication, administration, distribution channel, and region. In terms of type, the market is bifurcated into non-viral vector gene therapy, and viral vector gene therapy. In terms of indication, the market is grouped into inflammatory diseases, neurological diseases, infectious diseases, hematology diseases, oncology diseases, genetic diseases, and others. With respect to segmentation by distribution channel, the market is categorized into online pharmacies, drugstores, retail pharmacies, and hospital pharmacies.

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Rare Disease Gene Therapy Market: Competitive Landscape

Expanding collective innovative work exercises among driving biopharmaceutical imaginative players is required to fuel the development of uncommon sickness quality treatment market. What's more, clinical-stage improvement organizations are allotting critical assets towards the advancement of the uncommon illness quality treatment market.

For example, in August 2020, the U.S. based Ovid Therapeutics teamed up with Italian firm Angelini Pharma to create, make and popularize drug gaboxadol for treatment of Angelman disorder (uncommon hereditary sickness) in European market.

Some of the players of the global rare disease gene therapy market include Amgen Inc., AbbVie Inc., F.Hoffmann-La Roche Ltd., Merck & Co., Johnson & Johnson, Baxter, Novo Nordisk A/S, Alexion, Pfizer Inc., Bristol-Myers Squibb Company, Novartis AG, Sanofi S.A., Teva Pharmaceutical Industries Ltd., Bayer AG, Allergen plc., Vertex Pharmaceuticals, and others.

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Rare Disease Gene Therapy Market: Interesting Insights

There has been an intense competition among biopharmaceutical players to create Covid treatment that is postponing on account of innovative work exercises in uncommon infection quality treatment market. Stop of assembling exercises and flexibly tie disturbance because of exchanging across outskirts will have sway on uncommon illness quality treatment market development rate. The decrease in treatment selection rate attributable to diminished patient visits to medical services offices will lessen development pace of uncommon sickness quality treatment market. The increasing number of uncommon infections is relied upon to drive the development of uncommon illness quality treatment market.

Furthermore, the expanding synergistic innovative work exercises among driving biopharmaceutical inventive players is relied upon to fuel the development of uncommon infection quality treatment market. Also, clinical-stage advancement organizations are allotting critical assets towards the improvement of the uncommon infection quality treatment market.

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Rare Disease Gene Therapy Market: Increasing cases of genetic diseases to drive the market - BioSpace

Single gene therapy injection surprisingly boosts vision in both eyes – New Atlas

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One of the ways scientists hope to offer better treatments for vision loss is through gene therapy, where carefully selected genetic material is injected into the eyes to address mutations. Researchers have been left surprised by the effectiveness of an experimental form of this treatment, which involved an injection into one eyeball yet improved vision across both.

Gene therapies have the potential to treat all kinds of health conditions, ranging from cancer, to diabetes in dogs, to obesity and damaged spinal cords. One area where we're seeing some really exciting progress is in hereditary vision loss, with studies demonstrating the potential of gene therapy to treat color blindness, progressive retinal diseases and glaucoma, with some recently receiving approval from the FDA.

This latest study was conducted by scientists at the University of Cambridge, the University of Pittsburgh and Paris Institut de la Vision, and focuses on a form of inherited vision loss called Leber hereditary optic neuropathy (LHON). This affects around one in 30,000 people and usually occurs in young folks aged in their 20s and 30s, destroying their retinal ganglion cells and in turn the optic nerve. Once the condition takes hold, vision can deteriorate to the point where the subject is considered legally blind in just a matter of weeks, with recovery occurring in less than 20 percent of cases.

The majority of patients suffer from the same mutation affecting the MT-ND4 gene, so the researchers were hopeful of targeting this mutation as a way of improving treatment outcomes for sufferers of LHON. They trialed their gene therapy as part of a study involving 37 patients who had suffered vision loss in the preceding six to 12 months. This meant injecting a viral vector packed with a modified complementary DNA called rAAV2/2-ND4 into the vitreous cavity at the back of just one eye, with a sham treatment injected into the other eye.

We expected vision to improve in the eyes treated with the gene therapy vector only, says study author Dr Yu-Wai-Man. Rather unexpectedly, both eyes improved for 78 percent of patients in the trial following the same trajectory over two years of follow-up.

To investigate the reasons behind this unexpected outcome, the team studied the gene therapys effects in macaques, which have a similar vision system to humans. This enabled them to analyze the tissues from different parts of the eye to see how the viral vector DNA had spread. This provided evidence of interocular diffusion, with the viral vector DNA turning up in the retina, optic nerve and anterior segment of the untreated eye.

As someone who treats these young patients, I get very frustrated about the lack of effective therapies, says senior investigator Dr Sahel, from the University of Pittsburgh. These patients rapidly lose vision in the course of a few weeks to a couple of months. Our study provides a big hope for treating this blinding disease in young adults.

The research was published in the journal Science Translational Medicine.

Source: University of Cambridge

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Single gene therapy injection surprisingly boosts vision in both eyes - New Atlas

Freeline takes the haemophilia B gene therapy fight to Uniqure – Vantage

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Bearing the usual perils of cross-trial comparisons in mind, the much larger Hope-B pivotal trial of Uniqures etranacogene dezaparvovec (AMT-061) found mean FIX activity of 37% of normal at 26 weeks, taking severe haemophilia B patients into the range seen in mild disease (Ash 2020 hints of durability emerge for Uniqure's gene therapy, December 8, 2020).

Although this has been heralded as a huge advance, around 30% of patients in Hope-B did not reach 30% FIX activity, and some participants still experienced bleeds.

Freeline hopes to go one step further and eliminate serious bleeds, returning haemophilia B patients to a normal life. The company noted that in B-Amaze there were no bleeds that needed FIX supplementation.

The increased FIX activity seen with verbrinacogene setparvov is down to Freelines novel synthetic capsid, called AAVS3, the companys founder, Professor Amit Nathwani of University College London, explained during yesterdays call. He said the capsid leads to three to sixfold higher levels of gene transfer compared with other, wild-type capsids, which in turn should spur high levels of protein expression using relatively low doses of vector.

However, Uniqures capsid might have an edge in one respect: Hope-B did not exclude patients with pre-existing neutralising antibodies to AAV.

Freelines trial did include some patients with neutralising antibodies, Ms Krop told Evaluate Vantage, although there was a cut-off over which patients with these antibodies were excluded. This was intentionally low, Ms Heggie added, without giving details.

Although the data from B-Amaze look promising, the patient numbers involved are small, and Freeline now needs to show that its claims hold up in a bigger population. The pivotal trial will enrol 20 patients initially, and the phase IIb portion will be used to confirm the dose before moving into the phase III portion.

Freeline hopes it will be able to file for accelerated approval based on 26-week FIX data along with 52-week annualised bleeding rate data in a subset of these patients. The study will then enrol up to 30 more patients to evaluate 52-week ABR, which is designed to support full approval.

It is too early to call a winner in haemophilia B gene therapy, according to Evercore ISIs Josh Schimmer, who added: Uniqure has a bird in the hand with its dataset while Freeline has a couple of promising ones in the bush.

Ms Heggie is not concerned about not being first to market. We know we're probably two years behind Uniqure, but we know patients will wait for the best treatment.

She also told Evaluate Vantage that, while Freeline is not yet discussing pricing, we expect to have really strong value proposition if, as we expect, itll be a functional cure.

Freeline still has a lot of work to do, but Uniqure might soon have some real competition.

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Freeline takes the haemophilia B gene therapy fight to Uniqure - Vantage

New York Auto Insurance Fraud Ring Busted Following Two-Year Investigation – Insurance Journal

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An enterprise insurance fraud scheme operating in lower Westchester and the Bronx has been taken down following a nearly two-year long investigation.

Westchester County District Attorney Anthony A. Scarpino Jr. announced the takedown of Operation Sledgehammer, in which five registered businesses and nine defendants were charged with enterprise corruption for defrauding insurance companies by enhancing motor vehicle damage and falsifying insurance claims.

Additionally, three other individuals were charged on separate felony complaints with other crimes relating to this scheme.

Auto insurance theft affects every one of us, said Scarpino in a press release issued by his office. Insurance fraud is not a victimless crime. When false claims are made and money is stolen from insurance companies, it forces all of our insurance rates to increase and costs each of us hard-earned money.

Between around May 31, 2019, and March 3, 2020, the defendants intentionally conducted and participated in a pattern of criminal activity in the form of auto insurance fraud via auto repair shops. This happened in the city of New Rochelle, in other parts of Westchester County, in Bronx County and elsewhere in the state of New York.

The defendants and uncharged and unnamed others were members of the Jawahir Enterprise, which operated and furthered an enhanced motor vehicle damage and automobile insurance fraud scheme. The Jawahir Enterprise focused on the defrauding of insurance companies by the submission of claims that evidenced enhancing damage to a vehicle either with or without knowledge of the vehicle owner and/or intentionally inflicting damage to vehicles that were not previously damaged.

Several schemes were used to defraud insurers. In some cases, defendants strategically enhanced vehicle damage so the body shop could increase the billed repair cost while keeping the actual cost of repair to a minimum. The insurer would be charged the higher amount. Another scheme involved staging accidents between two or more vehicles, at least one of which was owned by a person who had insurance.

An additional scheme involved members of the enterprise strategically striking insured vehicles with heavy objects such as sledgehammers or rubbing light assemblies alongside a vehicle. The damage was created to simulate collisions with stationary vehicles. At times, members of the Jawahir Enterprise created no new damage to their vehicles, but instead submitted claims to multiple insurance companies under different policies in an attempt to receive money.

The insurance companies primarily issued claim payments in the form of physical checks. In some instances, the proceeds of the fraudulently obtained insurance claim checks were laundered through a series of orchestrated deposits, transfers and cash withdrawals throughout bank accounts controlled by members of the Jawahir Enterprise.

During the early part of 2019, the National Insurance Crime Bureau began noting a disproportional number of high-end claims being submitted by Lees Auto Body located in New Rochelle for repairs to customers vehicles, said New Rochelle Police Commissioner Joseph F. Schaller in the release.

Suspecting that the claims had a fraudulent aspect, they then forwarded their initial findings to the Westchester County District Attorneys Office. A year-long investigation was launched by the Westchester County D.A.s Investigators in conjunction with the City of New Rochelle, City of Yonkers, New York State Police Special Investigations-Auto Theft Unit, New York State Department of Motor Vehicles Division of Field Investigations, New York State Department of Financial Services, New York State Environmental Conservation Police and the National Insurance Crime Bureau.

Through numerous investigative techniques, and unparalleled cooperation amongst members of all agencies, Operation Sledgehammer is being brought to a successful conclusion, Schaller said in the release. It is the largest Enterprise Corruption case ever prosecuted in Westchester County.

Four of the defendants Joseph S. Cermele, Rangee Jawahir, Georgette S. Sloley and Wayne M. Scott were arrested on Tuesday, December 1, 2020, and arraigned in New Rochelle City Court. The remaining individuals Ronnie Lee-Andros Jawahir, Allan Ramdeo Ramiah, Ronald Jawahir, Danesh Singh and Andrew Arjune were arrested Tuesday, December 8, 2020, and also arraigned in New Rochelle City Court. All individuals were released without bail and are to return to court on different dates throughout December and January.

All named individual defendants and companies have been charged with enterprise corruption, a class B felony, and other felony offenses listed in the felony complaint including grand larceny in the second degree, a class C felony, money laundering in the fourth degree, a class E felony, insurance fraud in the third degree, a class D felony, and grand larceny in the third degree, a class D felony.

These arrests send two messages, Scarpino said in the release. To all fraudsters: know that this type of illegal conduct will not be tolerated in this County and will be fully investigated and prosecuted. And to the people of Westchester: know that we actively seek out and bring to justice those enterprises which seek to gain illegal monies to the detriment of all insurance policy holders.

The case is being prosecuted by Investigations Division Assistant District Attorneys Laura Forbes and Emily Rowe-Smith. The investigation is continuing and more arrests are expected, the release said.

Source: Westchester County, New York, Office of the District Attorney

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New York Auto Insurance Fraud Ring Busted Following Two-Year Investigation - Insurance Journal

Innovation Needs Honesty to Thrive – KKOH

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We will never know the true cost of the lost innovation and economic growth inflicted by nefarious actors who fluff their valuation figures or just outright lie. News travels fast, fake news travels faster.

But investors deserve reliable information.

Phoenix-based Nikola Corp. has been making headlines in recent months as the company undergoes investigation from the SEC regarding claims that the electric-vehicle designer may have deceived investors about its business prospects.

Promised to rival Elon Musks industry disrupter Tesla, Nikola founder and, now former executive chairman Trevor Milton promised hydrogen power and battery innovations created through proprietary technologies.

Predictably, investor enthusiasm was rampant and the company was even able to secure a $2 billion procurement and manufacturing partnership with General Motors in September that pushed share prices to $79.

Nikolas outlook was rosy.

But a report from Hindenburg Research called out Milton and his company alleged fraudulent activities over nearly 15 years to trick investors with false promises about its prototypes sending shockwaves across investors who were misled by Nikolas claims to manipulate the stock price.

The exaggerated or false claims that Nikola Corp. is accused of making if found to be factual are not victimless crimes. Investors on all levels have lost big. The companys stock (NASDAQ: NKLA) has lost more than 50 percent of its value since the September report was published.

The hordes of new, often younger investors without such deep pockets who are known to seek visionary green-tech companies, just like Nikola claimed to be, have fallen victim. For example, investing app Robinhood, with an average user age of 33, reported 3 million new users in just four months this year.

These harms are compounded when fraud on this (alleged) scale results in far more wearily disbursed venture capital, especially among the innovators responsible for real disruptive technologies.

These actions also dissuade collaboration between companies out of fear of being misled. Nikola Corp. relied heavily on partnerships; however, they could not hold up their end of the bargain.

As Navigant Research Sam Abuelsamid told The New York Times,It certainly seems like Nikola really inherently doesnt have any technology of note. They have a vision and have been going around setting up partnerships with companies that do have the technology and are trying to pull it all together.

Alas, this is not the first high-profile case in which a company did not live up to its promises.

In 2018, tech startup HouseCanary, which offers real estate valuation technology, won a staggering $740 million judgment in a Bexar County, Texas, countersuit filed over the misappropriation of alleged trade secrets.

Title Source, now known as Amrock, originally sued HouseCanary after it neglected to deliver the innovative appraisal-oriented mobile app that HouseCanary was being paid $5 million annually to build.

Soon after the trial, former HouseCanary employees came forward with additional revelations. Among them, Anthony Roveda, a former HouseCanary employee, offered sworn testimony that, It was evident to me that during the time I worked at HouseCanary there was never a working version of the app . . .There wasnt anything to steal.

He also testified,In my experience, HouseCanary leadership, and the CEO Jeremy Sicklick in particular, was focused more on enhancing the image of the company and on fundraising than on creating a working product.

The whistleblowers also testified that there was collusion between HouseCanary executives and someone at Title Source, which led some to suggest that the dispute had been a HouseCanary set up from the onset.

One cannot help but draw parallels between the allegations facing Nikola and those made of HouseCanary.

Fortunately, due to legal errors made in the original trial, the Texas Court of Appeals for the Fourth Circuit in San Antonio reversed HouseCanarys $740 million award, ruling that the various alleged breaches occurred after HouseCanary had voluntarily disclosed its claimed trade secrets.

This was a pivotal step in the right direction and sets practical precedent for future intellectual property disputeswhich seem to be getting more contentious as technology advances.

What happens now?

A three-justice panel updated its June opinion two months later amending its reversal and requiring HouseCanary to either request that the trial court enter a judgment on the contract claim jury verdict or retry all claims against Amrock.

The tech sector itself and the many more who invest in it will be watching these cases and others like it closely. American innovation and the broader economy relies on partnerships between companies for success and growth; this helps to create jobs, develop better products for consumers and the environment, and bolsters the U.S. competitive position in the global market.

Jared Whitley is a long-time politico who has worked in the U.S. Congress, White House and defense industry. He is an award-winning writer, having won best blogger in the state from the Utah Society of Professional Journalists (2018) and best columnist from Best of the West (2016). He earned his MBA from Hult International Business School in Dubai. Read Jared Whitleys reports More Here.

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Innovation Needs Honesty to Thrive - KKOH