Archive for the ‘Gene Therapy Research’ Category

Its been a record year for biotech IPOs, and the execs at Nasdaq would like nothing better than to see that momentum continue into the first half of next year.

Since January, 72 biotech and biopharma companies have hit Wall Street, according to Nasdaq head of healthcare listings Jordan Saxe, together raising $13.2 billion.

The latest is Flagships Foghorn Therapeutics, which priced its shares last night at $16 apiece, the midpoint of a $15 to $17 range. The Cambridge, MA-based biotech which initially filed for a $100 million raise on Oct. 2 is netting $120 million from a 7.5 million-share offering. The proceeds will go right into its gene traffic control platform, including two lead preclinical oncology candidates.

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Bo Cumbo jumps from the top commercial post at Sarepta to the helm of a gene therapy startup with some influential backers, big plans and $107M -...

The cause of fragile X syndrome (FXS), the most common inherited intellectual disability, is easy to see in the lab. Under electron microscopy, an affected X chromosome exhibits a deformed tip that gives the disorder its name and pinpoints the causative gene malfunction. Theres no cure for the disease, whose symptoms include learning deficits and hyperactivity and which has been linked with autism. FXS occurs in 1 in 4,000 to 7,000 males and 1 in 8,000 to 11,000 females in the United States.

Most research on FXS has focused on the brains neurons, the cells that transmit electrical and chemical impulses. But for a decade Yongjie Yang, associate professor of neuroscience at Tufts University School of Medicine, has pursued a different path, investigating the involvement of glia cells, particularly astroglia, which support neuron function and make up more than half the brain. In the past month, hes published in the Proceedings of the National Academy of Sciences (PNAS) and Glia. Tufts Now spoke with Yang about his work.

Tufts Now: What do we know about FXS?

Yongjie Yang: Fragile X syndrome is caused by the mutation of a single gene, FMR1, that codes for the FMRP protein, which is found in all brain cells and is essential for normal cognitive development. The mutation doesnt actually change the genetic code. Instead it causes part of the gene, specifically the chemical bases CGG, to repeat. We all carry those repeats in different numbers. If you carry roughly 50 or fewer, your brain development will be normal, but if the repeats go beyond 200, you will have the full mutation and your brain will produce only 10 to 20 percent of the needed level of FMRP, especially if youre male. FXS was characterized in 1943 but the genetic mutation wasnt identified until 1991, almost half a century a later.

What is the relationship between autism spectrum disorder (ASD) and FXS?

The two are intermingled. ASD is much more common, occurring in 1 in 54 children according to new estimates. Its believed that 1 to 6 percent of people with ASD have the FXS mutation, and that mutation accounts for the largest genetic subset of those with ASD. Many people with FXS are also autistic. FXS is a learning and intellectual disability, while ASD includes a wide range of social and communications challenges.

What are the key findings of your most recent research?

The study in Glia shows that some physical symptoms of FXS can be induced in mice by eliminating FMRP from astroglia alone. So in thinking about gene therapy for FXS, we need to consider glia cells, not just neurons. Our PNAS paper is exciting because it defines a unique, distinct FMRP-dependent pathway in mouse and human astroglia that regulates communications from astroglia to neurons through mGluR5, an important receptor for glutamate, the neurotransmitter that triggers brain activity. Interestingly, this regulation pathway isnt found in neurons. Its also the first study to demonstrate how overall protein expression is changed in FMR1-deficient astroglia. Unveiling astroglia-specific molecular mechanisms involved in FXS development could give us new targets for potential therapeutics.

Whats next?

We want to better understand the pathophysiology of FXS and identify new avenues for drugs and other interventions to attenuate the effects of the disease. Of course gene therapy would be wonderful but it often takes a long, long time and carries a lot of risk. Most other studies have focused on the neuron side, and drug trials based on these studies have failed so far. Our glia/astroglia perspective gives a fresh view to search for new targets.

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Getting to the Roots of Fragile X Syndrome - Tufts Now

Carla said: The radiotherapy was really tough and Luca had to be sedated every day for six weeks so that he could receive the treatment. It was also hard to separate the boys for those six weeks as Rocco is non-verbal autistic and the two brothers are extremely close. We made sure we FaceTimed as often as possible so the boys could wave to each other.

I havent spoken to Luca about cancer as hes too young to understand it properly. But he knows that he needs to take medicine to get better, and thats enough for now.

Weve had our challenges, but he shows amazing bravery and courage and has a huge smile on his face every day. He is my strength and inspires me to keep on going. Even when his hair fell out from the chemotherapy, he was so happy not to have to go for haircuts! I'm just so proud of him.

Carla added: Luca is living proof of why research into cancer is so important as, thanks to treatment, he is still here today. But although his tumour has been successfully targeted with treatment, he has sadly been left with side effects which will need monitoring for many years to come. Radiotherapy for childrens cancers is very effective, but what many people dont realise is how harsh the treatment can be on youngsters, especially when they are blasting an area as sensitive as the brain.

Lucas short-term memory and attention span has definitely been affected by what hes been through. Sometimes when were chatting, hell just zone out as though he doesnt understand what Im saying. And hes also more forgetful, I can tell him something one minute thats totally forgotten the next. As he gets older, he will need to be monitored for any more effects of the treatments.

The Manchester-based scientists are benefitting from the new Stand Up To Cancer-Cancer Research UK Paediatric Cancer New Discoveries Challenge awards. Their work has been recognised because it reflects Stand Up To Cancer and Cancer Research UKs shared ambition to accelerate the development of new treatments for some of the rarest and hardest to treat cancers in children and young people.

Lead scientist Dr Martin McCabe, who is based at The Christie, said: Its great news for Manchester that we have been awarded this funding. Its an ambitious goal, but we hope this research could lead to safer radiotherapy treatments for childhood brain tumours treatments that arent as tough on young people as the ones we use now, and maybe new treatments that could help more young people to survive this type of cancer in the future. This research could be a real game-changer for generations to come as we develop ways to deliver radiotherapy accurately to tumours but avoid sensitive areas of the brain and ultimately give patients much better lives.

He continued: The award is also a proud recognition of Manchesters reputation as a world-leading centre in cancer research. Manchester remains an international leader in the fields of proton beam therapy, gene therapy and immunotherapy and is home to The Royal Manchester Childrens Hospital, the biggest childrens hospital in Europe and The Christie, the biggest single-site, dedicated cancer hospital in Europe. Were excited to have been awarded this funding and were looking forward to bringing our expertise to a global team to help more young people across the world with this devastating disease.

Stand Up To Cancer in the UK is a joint national fundraising initiative from Cancer Research UK and Channel 4.This Friday October 23 will see special editions of Celebrity Gogglebox, and The Last Leg to raise awareness of the cause

Anna Taylor, Cancer Research UK spokesperson for the North West, said: People in Manchester have every right to feel proud of the groundbreaking research being carried out on their doorstep, and of their fundraising efforts, which are helping to beat the disease.

Every year, over 40,000 people are diagnosed with cancer in the North West**. So, were working every day to get new cancer tests and treatments to people who need them the most. Cancer doesnt stop in the face of a pandemic. It can affect anyones life, at any time so we only have one option: speed up life-saving research.

Thats why now is the time to Stand Up To Cancer. Were asking everyone to donate or fundraise in any way they can, so we can keep funding incredible scientists like Dr McCabe and his team and help save more lives.

To get involved visit su2c.org.uk

More:
Mum's heartfelt reason for backing children's brain tumour research - The University of Manchester

As we enter 2020, one drug class that is set to take-off is gene therapy. With three approvals (Zolgensma (US), Zynteglo (Europe) and Beperminogene Perplasmid (Japan), 2019 was a break out year for gene therapy developers. The $16 billion-plus investment by various investors finally seems to be paying off as we enter 2020 with am.,

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While several positives make us all very optimistic about the future of gene therapies, there are a few challenges that must be addressed before these therapies can achieve their true potential. Challenges related to pipeline development, platform selection, manufacturing, and reimbursement are amongst the top hurdles that these companies are currently facing. Also, the challenges related to supply chain and logistics are beginning to arise as these therapies reach the market.

These logistical challenges are more pronounced when products are to be distributed to international markets. Given the lack of experience specific to gene therapies in the industry, the networks may not be as developed, and delay times at ports of entry may be ruinous to products with limited shelf lives. A few other industries that have faced such logistic issues in the past are the food delivery market, and the rental cabs or bookings market. Earlier, it had become so difficult to track the status of your pizza order. But now, with the rise of mobile applications, you can track the progress in real-time.

Let us imagine a scenario wherein you are celebrating the new years eve with your family. You have decided to order pizzas for the family from this amazing restaurant. You call the restaurant and place the order. The restaurant confirms your order and then the wait begins. 10 minutes.20 minutes30 minutes pass and you have no idea where your order is. The new year celebration excitement suddenly turns to anxiety and you are worried and unsure about when can you exactly expect your pizza.

Now let us think about what happens to a patient that is eligible for a gene therapy treatment. Given the exhaustive inclusion criteria, only a limited number of patients qualify for these therapies, and once they qualify, the wait begins. And it is not just the patient who is waiting, their entire family, along with the doctors are anxiously waiting for that personalized dose of cells to be delivered at the hospital bed that can finally cure the disease. If we are ready to stretch our imaginations, this wait and anxiety that the patient faces can easily be compared to the pizza hut craving that you were having on the new years eve.

The solution simple. Get an App!!

Do what every company seems to be doing these days. Make personalized therapies even more personalized. Get an app that tells the patient the whereabouts of their therapy across the supply chain. Give the patient some control and deliver not just a therapy, but an experience.

Some of the players, such as (illustrative list) Trakcel, Veeva, Be the Match BioTherapies, are already deploying solutions that can be used to efficiently manage and streamline various aspects of the complex supply chains of cell and advanced therapies. Presently, there are more than 160 innovative software-enabled systems for managing the cell and advanced therapies supply chain.

For further information, check out the report here

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Roots Analysis Leaders in Pharmaceutical & Biotechnology Market Research

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About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

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A Pizza Hut Prescription for Gene Therapy Companies | Roots Analysis - Eurowire

Heres your wrap of the latest technology, innovation, and finance news.

Alternative Foods

How far are cell-based meats from commercialisation? Verdict talked with Memphis Meats about where they are on commercialisation journey.

We need to find clever process solutions and cell feed formulations to ensure that were translating our raw materials into meat in the most cost-effective ways.

For the vegans who dont like soy milk, Impossible Foods is developing a plant-based alternative to cows milk.

As with Impossible Foods meat products, the goal of the milk alternative is to preserve the experience of consuming dairy products, including the texture, mouth feel, and flavour, while reducing the demand for raising animals like cattle.

Gaming

Alexandria Ocasio-Cortez played Among Us on Twitch and 430,000 people tuned in, making her the third most popular streamer on the site ever.

More important, the event offered a glimpse at the future of political campaigning. In the waning weeks of 2020s volatile American election campaign, Democrats are increasingly turning to Twitch streams of popular video games to reach out to young voters and urge every last one of them to show up and vote.

Video games are helping veterans with PTSD.

Amazons cloud-gaming service, Luna, is now available to select gamers in the United States.

Cyberpunk 2077 is using procedurally-generated facial animations to provide full lip-sync for every character in all 10 dubbed languages. See Judy speak English, French, German, and Japanese at the 17:05 point.

Artificial Intelligence

Facebook has developed a new AI thats capable of directly translating between any pair of 100 languages without first translating to English, as many systems do, and its performing pretty well.

The AI outperforms such systems by 10 points on a 100-point scale used by academics to automatically evaluate the quality of machine translations. Translations produced by the model were also assessed by humans, who scored it as around 90 per cent accurate.

A new paper outlines less than one-shot learning, a new technique designed to let AI learn with practically no data.

This is what most interests Tongzhou Wang, an MIT PhD student who led the earlier research on data distillation. The paper builds upon a really novel and important goal: learning powerful models from small data sets, he says of Sucholutskys contribution.

Landing AI is a company founded by Andrew Ng, one of the cofounders of Google Brain and the former chief scientist at Baidu. Theyve recently launched LandingLens, a computer vision platform that enables manufacturers to train AI models.

I feel like this is where the field of AI needs to go. Rather than highly skilled engineers at Landing AI or Google or wherever doing all the machine learning work to build verticalized platforms, someone in a platform [who] really understands what is a dent versus what is a sensible-minded blemish can do the customization. I think this is important for machine learning to reach its full potential, Ng said.

Biology

For the first time, scientists have improved the resolution of cryo-electron microscopes to the point where they can see individual atoms (see paper 1, paper 2).

Now, with the help of improvements in electron beam technology, detectors, and software, two groups of researchersfrom the United Kingdom and Germanyhave narrowed that to 1.25 angstroms or better,sharp enough to work out the position of individual atoms, they report today inNature.

Space

Microsoft is taking Azure cloud computing to outer space.

The goal is to serve as a bridge between satellites and new services from both the public and private sectors, spanning military, telecommunications, agriculture, energy, and more.

NASA has awarded Nokia a $14.1 million contract to build a 4G network on the moon.

Blue Origin has found a business niche with NASA and private science experiments.

Tucked under the collar at the top of the booster on Tuesdays launch were prototypes of sensors that could help NASA astronauts safely reach the lunar surface in a few years. It is part of NASAs Tipping Point program, which seeks to push innovative technologies.

Head of NASAs Pluto mission, Alan Stern, is going to space with Virgin Galactic.

Stern will oversee two different experiments while on board the flight, each meant to take advantage of the brief stay in the space environment.

OSIRIS-REx, a NASA spacecraft, has sampled material from the asteroid Bennu.

Surveillance

Activists around the world are building facial recognition tools to identify police officers that arent wearing identification.

I am involved with developing facial recognition to in fact use on Portland police officers, since they are not identifying themselves to the public, Mr. Howell said. Over the summer, with the city seized by demonstrations against police violence, leaders of the department had told uniformed officers that they couldtape over their name. Mr. Howell wanted to know: Would his use of facial recognition technology become illegal?

Mobility

Boom Supersonic unveiled XB-1, a subscale prototype of their planned supersonic passenger jet.

Dubbed XB-1, the 71-foot-long, single-seat test vehicle was built to validate the design and technology of the companys planned eventual final product, a $200 million airliner called Overture that will be three times XB-1s size and carry 55 passengers to Mach 2.2.

Health

Can gene therapy be used to treat some forms of autism?

Gene therapies are now moving into the autism space, and the Angelman trial is a sign of things to come. A success in this space will completely change the way that we think about genetic testing in autism, saysTimothy Yu, a neurologist and geneticist atBoston Childrens Hospital in Massachusetts. It will hold out the idea that if you can name the disease, you can actually do something to improve the quality of life for that child.

Researchers are using lab-grown tissue grafts for personalised joint replacements.

A team at the University of Leeds have developed a robotic arm to perform colonoscopies.

The researchers successfully tested this method in an artificial colon as well as in two pigs. They believe the magnetically controlled procedure may be lesspainfulthan conventional colonoscopies and could be used on patients without sedation.

Ecommerce

Alibaba is spending $3.6 billion to double its stake in Sun Art Retail Group, Chinas largest big-box retailer with more than 480 large supermarket-department stores.

Gavin Baker argues that leading brick-and-mortar retailers are likely to be the biggest long-term Covid beneficiaries.

Finance

China is experiencing a boom in share sales.

So far this year, exchanges in Shanghai and Shenzhen have hosted more than $47.5 billion of IPOs and listings for firms that have shares already trading elsewhere, Refinitiv data shows.

That is already the highest annual tally compared with any full year since 2010 and an unprecedented 27% of the global total, the data shows. If deals in Hong Kong by Chinese companies are added, the proportion rises to 43%.

Other Snippets

MIT Technology Review looked at Singapores huge bet on vertical farming.

Since then, food security has raced up the agenda. Now the governments stated policy is that it wants to produce enough food to supply 30% of its own nutritional needs by 2030, up from just 10% now. To get there, it says, Singapore will need to grow 50% of all fruits and vegetables consumed domestically, 25% of all proteins, and 25% of all staples, such as brown rice. The commitment effectively aims to triple production by volume in the next 10 years. And since the country is short of land, it has pinned its hopes on technology.

Zoom has begun rolling out end-to-end encryption.

All Zoom users free or paid can now host a meeting with up to 200 participants with end-to-end encryption on the platform, meaning the company cannot access any of the data it is hosting.

The New York Times discussed the problem of free speech in an age of disinformation, and how democracies in Europe and Canada balance free speech with other democratic values.

A Japanese politician is battling to vanquish the ink stamp, the printer, and the fax machine.

Why do we need to print out paper? Mr. Kono asked rhetorically at a news conference soon after taking charge of the issue in September. In many cases, it is simply because the hanko is required. So if we can put a stop to that culture, then it will naturally eliminate the need for printouts and faxes.

Fast Company outlined 25 moments in tech that defined the past 25 years.

Read more:
Innovation Wrap: Cell-Based Meat, AI Less-Than-One-Shot Learning, Gene Therapy & Autism - ShareCafe

NEW DELHI: Convalescent plasma therapy, which uses the blood of recovered Covid-19 patients as a potential treatment, has shown limited effect in reducing the progression to severe disease or death in a trial conducted in India, scientists say. The study, published in the British Medical Journal (BMJ) involved 464 adults with moderate Covid-19 who were admitted to hospitals in India between April and July.

As many as 239 adult patients received two transfusions of convalescent plasma, 24 hours apart, alongside standard care, while the control group comprising of 229 patients received standard care only.

One month later, 44 patients or 19 per cent of those who received the plasma had progressed to severe disease or had died of any cause, compared with 41 patients or 18 per cent in the control group.

"Convalescent plasma was not associated with a reduction in progression to severe Covid-19 or all cause mortality," the researchers wrote in the journal.

"This trial has high generalisability and approximates convalescent plasma use in real life settings with limited laboratory capacity," they said.

The researchers noted that a prior measurement of neutralising antibody titres in donors and participants might further clarify the role of convalescent plasma in the management of Covid-19.

Patients in the study were aged at least 18 years who had confirmed Covid-19 based on a RT-PCR result for SARS-CoV-2, the virus that causes the disease.

Participants in the intervention arm received two doses of 200 millilitre (mL) of convalescent plasma, transfused 24 hours apart, in addition to the best standard of care.

Although the observational studies conducted previously suggested clinical benefits in recipients of convalescent plasma, the trials were stopped early and failed to ascertain any mortality benefit from plasma treatment in patients with Covid-19, the researchers said.

Although plasma treatment was associated with earlier resolution of shortness of breath and fatigue and higher negative conversion of SARS-CoV-2 RNA on day 7 of enrolment, as a potential treatment for patients with moderate Covid-19 it showed limited effectiveness.

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Covid-19 plasma therapy has shown little benefit in patients in India, study finds - ETHealthworld.com

The rise in the popularity of cell and Gene Therapy has been widely talked about in recent years. The rise in this popularity has come with its own challenges, a number of which were identified in a recent report by Root Analysis. The challenges vary from the high cost of manufacturing to low capacity. In fact, the Roots Analysis team interviewed several stakeholders to understand their perspective about the key challenges in the vector manufacturing market. The key points from the interviews have been highlighted in this article. In the figure, I have added some of these challenges:

For More Insights Click Here

Capacity constraints remain a key challenge and have been talked about quite actively. In fact, we have seen several players invest heavily in enhancing their vector manufacturing capacity. However, one other area that has silently emerged as a potentially hot topic is the novel vectors (beyond traditional AAV vectors).

Currently, the vector manufacturing market is dominated by viral vectors, such as those based on AAV, adenovirus, lentivirus, and retrovirus. However, certain non-viral vectors, such as plasmid DNA, also hold a considerable share. Roots Analysis, in their report, talked about the rise of these non-viral vectors and identified the key emerging vectors types. Some of the vectors highlighted in the report are alphavirus, Anc80 vector, B. longum, Listeria monocytogenes, minicircle DNA, myxoma virus, Sendai virus, self-complementary vectors (which are essentially improved versions of AAV vectors) Sleeping Beauty transposon-based non-viral vectors, and Vaccinia virus-based vectors.

In fact, several players in the biopharmaceutical industry have already begun using some of the novel vector types mentioned above for the development of their respective pipeline therapy candidates. A few of the players involved in the novel vectors space are highlighted below:

The stakeholders interviewed by Roots Analysis agreed to the rising demand for novel vector types. Here are the excerpts from some of the interviews:

We believe that a few novel vectors, having low immunogenicities and targeting different cell types, are likely to soon be introduced into the market. I am also aware of companies that are researching different (better) versions of adeno-associated viral vectors. Executive & Scientific Officer, A small-sized company based in Belgium

We have worked with a couple of transposons and the Vaccinia viruses as vectors, the latter cannot exactly be regarded as a novel approach. I believe, transposons and Sendai virus are the only novel vector systems that are likely to soon become popular. Managing Director, A management consulting firm for regenerative medicines based in Japan

To overcome the challenges associated with the production of contaminant free final product using conventional plasmids, we are developing a minicircle DNA vector, which are devoid of antibiotic resistance genes and prokaryotic plasmid components, which are crucial for the replication of constructs in bacteria. Project Manager and Marketing Manager, a small-sized company based in Germany

For further information, check out the report here

Read more insights at

Roots Analysis Leaders in Pharmaceutical & Biotechnology Market Research

You may also be interested in the following titles:

About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

Contact Information

Roots Analysis Private Limited

Gaurav Chaudhary

+1 (415) 800 3415

[emailprotected]

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Cell and Gene Therapy Manufacturing: Rising Demand Forces Companies to Look Beyond Viral Vectors | Roots Analysis - Eurowire

Shortly after Sirnaomics brought in a $47 million Series C for its small interfering RNA pipeline last year, Patrick Lu the founder, president and CEO was asked to outline the scientific advances that will be necessary to make better drugs out of RNA tech.

The next step in the evolution of RNAi as a leading therapeutic will be the ability to safely target organs outside the liver such as lung, brain, etc, he had offered. This will revolutionize disease treatments if the industry can demonstrate similar data sets for non-liver targets as we have seen in liver-based diseases.

Then in April, the trans-Pacific biotech did just that. In a Phase II open-label dose escalation study, Sirnaomics reported interim results suggesting that its lead drug, STP705, helped certain cancer patients clear their squamous cell carcinoma.

Investors now say its time for a Series D, pumping $105 million into the STP705 program as well as another lead drug named STP707. The clinical focus, Sirnaomics added, will be evaluating these dual-targeted siRNA inhibitors, which hit TGF-1 and COX-2 either locally or systemically, together with checkpoint inhibitors. But with almost 10 other programs in the pipeline, the company remains on track to explore not just RNAis application in cancer but also in fibrosis diseases, metabolic diseases and viral infections.

Rotating Boulder Fund, an existing investor, led the round alongside new backers Walvax Biotechnology and Sunshine Riverhead Capital. Others on the syndicate include Sangel Capital, Longmen Capital, HongTao Capital and Alpha Win Capital.

In addition to a potential collaboration with Walvax on technical transfer and commercialization, Lu is open about preparing for an IPO in near future.

The company is the only biopharma venture conducting innovative R&D and clinical development in the field of RNAi therapeutics in both the US and China, the two largest markets for cancer and fibrosis disease treatments, Donald (Xiaochang) Dai, managing partner of Rotating Boulder Fund, noted in a statement.

With offices in Gaithersburg, MD and Suzhou BioBay just west of Shanghai, Sirnaomics recognizes that it is traveling down a path blazed by the likes of Alnylam and Arrowhead. But it boasts of a platform comprising a new polypeptide nanoparticle delivery system and a way to hit two targets at once promising to push RNAi beyond rare diseases or even cardiovascular conditions.

At Sirnaomics specifically, we are forging a path to bring RNAi therapeutics to the mainstream as therapeutic modalities for treatment of many diseases, such as non-melanoma skin cancer, liver cancer, liver fibrosis and NASH, Lu said in his 2019 interview.

Read this article:
Chinese investors wager $105M on an IPO-bound biotech looking to push RNAi as mainstream cancer therapy - Endpoints News

This article was reviewed by Russell N. Van Gelder, MD, PhD

Neuronal cell replacement therapies remain a challenge in retinal diseases. Some fish and salamanders have the innate ability to regenerate retinal tissue after injuries and, as Russell N. Van Gelder, MD, PhD, pointed out, if researchers could harness this ability in humans, the possibilities would be great for repairing or replacing damaged tissue in a wide variety of retinal diseases. Stem cells are the key to cell replacement therapies.

Stem cells are cells that have not terminally differentiated and still have the potential to become many types of terminal cells, said Van Gelder, from the Department of Ophthalmology at the University of Washington in Seattle. We all started as embryonic stem cells in the earliest phases of development.

Related: Retinal pathologies challenging to image with current technologies

Van Gelder went on to explain that there are now methods to create equivalently totipotent stem cells from individual induced progenitor stem cells derived from an individuals blood or epithelial cells.

The overarching goal is to create a cell type that needs replacement from a stem cell precursor, he said.

A major achievement in this quest for regenerative ability occurred in 2014 when an entire eye cup was grown from progenitor stem cells.

Van Gelder also described a study1 in which green fluorescent proteinlabeled retinal precursors derived from embryonic stem cells were transplanted into the subretinal space of macaques. Three months after the procedure, the researchers demonstrated that the bolus of cells persisted and had outgrowth of axons that were seen going to the optic nerve and on to the brain.

This result establishes the validity of a stem cell-based approach for doing regenerative medicine in primates, he said.

Related: Persistent retinal detachment associated with retinoblastoma

Replacement therapy hurdlesAs of now, however, no stem cell-based replacement treatment has received FDA approval. The problems preventing establishment of a treatment have been technical in nature and include correct cellular differentiation as well as generating adequate numbers of cells for large transplantation experiments, establishing correct cell polarity and connectivity, and ensuring the safety of these approaches regarding tumor or hamartoma formation, Van Gelder explained.

Managing inflammatory responses is a problem after cell transplantation. He cited a Japanese study2 of individual progenitor cell-derived retinal progenitor cells transplanted subretinally in monkey models.

Even with an immune HLA-matched donor, there was still a marked inflammatory response at the site of the transplantation, Van Gelder said. This and other inflammatory responses will have to be managed for cell transplantation to be successful.Related: Intravitreally injected hRPCs improve vision in retinitis pigmentosa cases

There are regulatory hurdles to clear. The FDA Center for Biologics Evaluation and Research regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy.

Van Gelder recalled the well-publicized case of transplantation of fat-derived mesenchymal cells into patients eyes, resulting in loss of vision bilaterally. He pointed out that it is important to temper patient expectations regarding these therapies and to ensure that the work is being done with the highest degree of ethical integrity.

While great progress has been made in this field, significant barriers remain to the successful adoption in the clinical setting in the coming years, Van Gelder concluded. The barriers to cell replacement should be overcome.

Read more by Lynda Charters

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Russell N. Van Gelder, MD, PhDe: russvg@uw.edu Van Gelder has no financial interests in this subject matter. He serves on the advisory committee for the National Eye Institute Audacious Goals Initiative.

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References

1. Chao JR, Lamba DA, Kiesert TR, et al. Transl Vis Sci Technol. 2017;6:4; doi:10.1167/tvst/6/3/4

2. Fujii S, Sugita S, Futatsugi Y, et al. A strategy for personalized treatment of iPS-retinal immune rejections assessed in cynomolgus monkey models. Int J Mol Sci. 2020;21(9):3077. doi:10.3390/ijms21093077

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Harnessing regeneration of retinal tissues: An option almost within reach - Ophthalmology Times

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Oct. 21, 2020 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company focused on developing adeno-associated virus (AAV) based gene therapies for the treatment of rare inherited diseases, today announced that Sue Washer, President & Chief Executive Officer, and Dave Knop, PhD, Vice President, Process Development and Manufacturing, will participate in the Cantor Fitzgerald Key Considerations for Gene Therapy Manufacturing and Platforms virtual panel at 10:00 a.m. ET on Wednesday, October 28, 2020.

The panel will focus on the critical components and capabilities to gene therapy manufacturing, including a discussion on AGTCs proprietary manufacturing processes associated with AAV, and key company catalysts expected over the next 12 months. The call will be moderated by Kristen Kluska, Biotechnology Research Analyst of Cantor Fitzgerald.

Presentation Information:Panel: Key Considerations for Gene Therapy Manufacturing and PlatformsDate: Wednesday, October 28, 2020Time: 10:00 a.m. 11:30 a.m. ET

To register for the event, please click here.

About AGTCAGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The Companys most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Its pre-clinical programs build on the Companys industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders.

IR/PR CONTACTS: David Carey (IR) or Glenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) 871-8485david.carey@finnpartners.com or glenn.silver@finnpartners.com

Corporate Contact:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com

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AGTC to Participate in Cantor Fitzgerald Virtual Panel: Key Considerations for Gene Therapy Manufacturing and Platforms - BioSpace

A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

Published today in Nature Biomedical Engineering, the paper, titled, Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing, illustrates the use of a new generation CRISPR technology and lays the foundation for the development of a new therapeutic modality for a wide range of inherited ocular diseases caused by different gene mutations.

"In this proof-of-concept study, we provide evidence of the clinical potential of base editors for the correction of mutations causing inherited retinal diseases and for restoring visual function," said Krzysztof Palczewski, PhD, the Irving H. Leopold chair and a distinguished professor in the Gavin Herbert Eye Institute, Department of Ophthalmology at the UCI School of Medicine. Our results demonstrate the most successful rescue of blindness to date using genome editing.

Inherited retinal diseases (IRDs) are a group of blinding conditions caused by mutations in more than 250 different genes. Previously, there was no avenue available for treating these devastating blinding diseases. Recently, the FDA approved the first gene augmentation therapy for Leber congenital amaurosis (LCA), a common form of IRD which originates during childhood.

As an alternative to gene augmentation therapy, we applied a new generation of CRISPR technology, referred to as base editing as a treatment for inherited retinal diseases, said first author Susie Suh, assistant specialist in the UCI School of Medicine Department of Ophthalmology. We overcame some of the barriers to the CRISPR-Cas9 system, such as unpredictable off-target mutations and low editing efficiency, by utilizing cytosine and adenine base editors (CBE and ABE). Use of these editors enabled us to correct point mutations in a precise and predictable manner while minimizing unintended mutations that could potentially cause undesirable side effects, said co-first author Elliot Choi, also an assistant specialist in the UCI Department of Ophthalmology.

Using an LCA mouse model harboring a clinically relevant pathogenic mutation in the Rpe65 gene, the UCI team successfully demonstrated the therapeutic potential of base editing for the treatment of LCA and by extension other inherited blinding diseases. Among other results, the base editing treatment restored retinal and visual function in LCA mice to near-normal levels. Base editing was developed at the Broad Institute of MIT and Harvard in the lab of David Liu, PhD.

After receiving treatment, the mice in our study could discriminate visual changes in terms of direction, size, contrast and spatial and temporal frequency, said Palczewski. These results are extremely encouraging and represent a major advance towards the development of treatments for inherited retinal diseases."

Gene therapy approaches to treating inherited retinal diseases are of special interest given the accessibility of the eye, its immune-privileged status and the successful clinical trials of RPE65 gene augmentation therapy that led to the first US Food and Drug Administration-approved gene therapy. Now, as demonstrated in this study, base-editing technology can provide an alternative treatment model of gene augmentation therapy to permanently rescue the function of a key vision-related protein disabled by mutations.

This research was supported in part by grants from the National Institutes of Health, the Research to Prevent Blindness Stein Innovation Award, Fight for Sight, the Eye and Tissue Bank Foundation (Finland), The Finnish Cultural Foundation, the Orion Research Foundation, the Helen Hay Whitney Foundation, US Department of Veterans Affairs, and a Research to Prevent Blindness unrestricted grant to the Department of Ophthalmology, University of California, Irvine.

Reference: Suh S, Choi EH, Leinonen H, et al.Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing.Nat. Biomed. Eng. 2020. doi:10.1038/s41551-020-00632-6

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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CRISPR Therapy Restores Retinal and Visual Function in Mice - Technology Networks

MELBOURNE, Australia and KING OF PRUSSIA, Pa., Oct. 20, 2020 /PRNewswire/ -- In its annual R&D briefing to investors today, CSL Limited (ASX:CSL; USOTC:CSLLY) demonstrated how the company is advancing a novel research portfolio across four strategic scientific platforms (Plasma Fractionation, Recombinant Technology, Cell and Gene Therapy, Adjuvanted Cell and Egg-based Vaccines) across six therapeutic areas (Immunology, Hematology, Respiratory, Cardiovascular and Metabolic, Transplant, Influenza Vaccines) and two businesses (CSL Behring and Seqirus) to help patients lead full lives, protect public health and drive future business growth. It's this scientific capability and diverse portfolio that positioned the company to quickly respond to the COVID-19 public health crisis.

"CSL's acumen in vaccines, monoclonal antibodies, recombinant technologies, manufacturing capabilities and external partnerships, along with a therapeutic area focus and insight that includes Immunology and Respiratory, has supported the growth and progress of our pipeline and has also enabled us to respond quickly to the need for potential solutions in the world's fight against COVID-19," said Bill Mezzanotte, MD, MPH,Executive Vice President, Head of Research and Development and Chief Medical Officer. "Right across the spectrum of infection and disease, CSL has taken on projects we think make sense both scientifically and that fit our capabilities."

The company's efforts in the fight against COVID-19 include:

"The same collaborative mindset with which CSL has responded to the COVID-19 pandemic is also reflected in how we are advancing our pipeline," said Andrew Nash, PhD, Senior Vice President, Head of Research and Chief Scientific Officer. "Across therapeutic areas, strategic platforms and geographies we continue to grow our footprint and capabilities so that we can deliver a significant benefit to patients around the world."

Other advancements in CSL's R&D pipeline in the last year include:

Setting the pace in Plasma Product development:CSL is continuously improving current plasma fractionation processes and developing new therapeutic options, which has shown major relevance in the fight against COVID-19 and is also evident in other therapeutic areas. For example:

Advancing its Cell and Gene Therapy research by:

Building momentum in Recombinant Technology:CSL has established a highly differentiated preclinical and clinical stage pipeline of recombinant proteins and monoclonal antibodies from its proficiency in protein biology and innate cell immunity. In addition to using this technology against COVID-19, the company is studying:

About CSLCSL (ASX:CSL) is a leading global biotechnology company with a dynamic portfolio of life-saving medicines, including those that treat hemophilia and immune deficiencies, as well as vaccines to prevent influenza. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL including our two businesses, CSL Behring and Seqirus - provides life-saving products to more than 70 countries and employs 27,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For more information about CSL Limited, visitwww.csl.com.

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CSL's Broad Response to COVID-19 and Advancements across the Company's Strategic Scientific Platforms are the Focus of Annual R&D Day - PRNewswire

ReportsWeb delivers well-researched industry-wide information on the Gene Therapy market. It studies the markets essential aspects such as top participants, expansion strategies, business models, and other market features to gain improved market insights. Additionally, it focuses on the latest advancements in the sector and technological development, executive tools, and tactics that can enhance the performance of the sectors.

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The research on the Gene Therapy market concentrates on extracting valuable data on swelling investment pockets, significant growth opportunities, and major market vendors to help understand business owners what their competitors are doing best to stay ahead in the competition. The research also segments the Gene Therapy market on the basis of end user, product type, application, and demography for the forecast period 20192026. Detailed analysis of critical aspects such as impacting factors and competitive landscape are showcased with the help of vital resources, which include charts, tables, and infographics.

For more clarity on the real potential of the Gene Therapy market for the forecast period 20192026, the study provides vital intelligence on major opportunities, threats, and challenges posed by the industry. Additionally, a strong emphasis is laid on the weaknesses and strengths of a few prominent players operating in the same market. Quantitative assessment of the recent momentum brought about by events such as collaborations, acquisition and mergers, product launches and technology innovation empower product owners, as well as marketing professionals and business analysts make a profitable decision to reduce cost and increase their customer base.

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Geographically, this report focuses on product sales, value, market share, and growth opportunity in key regions such asUnited States, Europe, China, Japan, Southeast Asia, and India.

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GENE THERAPY MARKET KEY TRENDS, GROWTH, SHARE, SIZE, ANALYSIS AND FORECAST TO 2026 - PRnews Leader

SAN DIEGO and WALTHAM, Mass., Oct. 21, 2020 /PRNewswire/ --Poseida Therapeutics, Inc. (NASDAQ:PSTX), a clinical-stage biopharmaceutical company utilizing proprietary gene engineering platform technologies to create cell and gene therapeutics with the capacity to cure, and TScan Therapeutics, Inc., a biopharmaceutical company focused on the development of T cell receptor (TCR)-engineered T cell therapies in oncology, today announced a research collaboration and license agreement to explore developing TCR-T cell therapies for the treatment of COVID-19. Poseida's allogeneic T cell platform will be used in combination with TScan's discovered immunodominant epitopes and TCR sequences for the development and commercialization of allogeneic TCR-T cell therapies.

"The TScan relationship will allow us to expand our platform and T cell experience, utilizing one or more TCRs in allogeneic cell therapy applications," said Devon Shedlock, Ph.D., Senior Vice President of Research and Development for Poseida. "We are impressed by TScan's novel platform for the discovery of TCRs. This, coupled with the opportunity to explore our allogeneic T cell platform's potential for also treating diseases outside of oncology, including COVID-19 and eventually other infectious diseases, represents an exciting and differentiated approach."

"Therapeutic strategies to treat COVID-19 have largely focused on neutralizing antibodies that block viral infection or small molecule drugs that inhibit viral replication," said Gavin MacBeath, Ph.D., Chief Scientific Officer at TScan. "We are excited about the possibility of combining our SARS-CoV-2 TCR findings with Poseida's allogeneic T Cell approach to explore a potential cellular therapy for patients with COVID-19. We look forward to collaborating with the Poseida team on this approach."

Emerging studies suggest that patients who fail to mount effective T cell responses often end up with more severe and life-threatening disease. Administering virus-specific engineered T cells may provide a rapid and effective way to address this deficiency. Results from TScan demonstrate that generating a memory T cell response targeting specific proteins may be important for fighting the virus and for long-term immunity. Combining these findings with Poseida's off-the-shelf allogeneic T cell product approach, which has generated a very high percentage of stem cell memory T cells (Tscm) and has the potential to yield hundreds of doses from a single manufacturing run using proprietary booster molecule technology, may provide both an immediate therapeutic effect, as well as longer-term immunity.

TScan recently announced data generated by their TCR/Target Discovery platform identifying the precise targets of T cells isolated from COVID-19 convalescent patients, along with the TCRs that recognize them. A core set of targets were shared among patients with the same human leukocyte antigen (HLA) type and were primarily located outside the spike protein. A paper describing this work is currently in press at Immunity, a Cell Press journal. TScan worked with clinical partner Atlantic Health Systems to recruit recovering COVID-19 patients for this work. Combining these findings with Poseida's off-the-shelf allogeneic T cell product platform may provide an accessible cell-based therapy for patients with insufficient T cell responses.

Under the terms of the Agreement, Poseida will gain access to TScan's data and IP related to TCR sequences and targets potentially valuable in treating COVID-19, and TScan will provide TCR expertise and counsel to the research collaboration. Detailed financial terms of the collaboration were not disclosed.

About Poseida Therapeutics, Inc.Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing our proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure. We have discovered and are developing a broad portfolio of product candidates in a variety of indications based on our core proprietary platforms, including our non-viral piggyBac DNA Modification System, Cas-CLOVER site-specific gene editing system and nanoparticle- and AAV-based gene delivery technologies. Our core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable us to engineer our wholly-owned portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics.

About TScan TherapeuticsTScan discovers and develops transformative T cell therapies (TCR-T) to treat liquid cancers, solid tumors, and other serious diseases. Our proprietary, high-throughput platform identifies previously uncharacterized, clinically-derived shared T cell antigens and all off-target TCR interactions, to enable the development of highly efficacious TCR-Ts with minimal off-target effects. Lead program TSC-100 is expected to enter clinical development for liquid cancers in 2021, and the Company is advancing additional TCR-Ts for solid cancers. TScan was co-founded by Chair Christoph Westphal (Partner, Longwood Fund) based on pioneering research from the Elledge Lab at Brigham and Women's Hospital. The Company has raised over $80 million to date from leading strategic collaborators and investors including Longwood Fund, Novartis Institutes for Biomedical Research, Astellas Venture Management, Novartis Venture Fund, Bessemer Venture Partners, GV, 6 Dimensions Capital, and Pitango Venture Capital.

Forward-Looking StatementsStatements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These forward-looking statements are based upon Poseida's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with development and regulatory approval of novel product candidates in the biopharmaceutical industry and the other risks described in Poseida's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Poseida undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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Poseida Therapeutics and TScan Therapeutics Announce Research Collaboration and License Agreement to Explore Developing Allogeneic T Cell Receptor...

Facts & Factors (FnF),a leading market research company recently published a research report onResearch Report on Cancer Gene Therapy Market Size & Share 2020 Covering COVID-19 Impact Analysis and Forecast to 2026to its research database. This[190+] PDF pages Cancer Gene Therapy Marketreport analyzes the comprehensive overview of the market comprising an executive summary that covers core trends evolving in the market.

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Cancer Gene Therapy Market Size & Share Insights on Growing Applications by 2026 - re:Jerusalem

Report is a detailed study of the Cell and Gene Therapy market, which covers all the essential information required by a new market entrant as well as the existing players to gain a deeper understanding of the market. The primary objective of this research report named Cell and Gene Therapy market is to help making reliable strategic decisions regarding the opportunities in Cell and Gene Therapy market.

Major Market Players with an in-depth analysis:

Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

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The Global Cell and Gene Therapy Market segments and Market Data Break Down are illuminated below:

By Therapy Type:Cell TherapyStem CellsT CellsDendritic CellsNK CellsTumor CellsGene TherapyGlobal Cell and Gene Therapy Market, By Indication:Cardiovascular DiseaseCancerGenetic DisorderInfectious DiseaseNeurological DisordersOthers

The Cell and Gene Therapy market report offers the current state of the market around the world. The report starts with the market outline and key of the Cell and Gene Therapy market which assumes a significant job for clients to settle on the business choice. It additionally offers the key focuses to upgrade the development in the Cell and Gene Therapy market. Some fundamental ideas are likewise secured by reports, for example, item definition, its application, industry esteem chain structure and division which help the client to break down the market without any problem. Also, the report covers different factors, for example, arrangements, efficient and innovative which are affecting the Cell and Gene Therapy business and market elements.

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Competitive Analysis has been done to understand overall market which will be helpful to take decisions. Major players involved in the manufacture of Cell and Gene Therapy product has been completely profiled along with their SWOT. Some of the key players include Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST. It helps in understanding their strategy and activities. Business strategy described for every company helps to get idea about the current trends of company. The industry intelligence study of the Cell and Gene Therapy market covers the estimation size of the market each in phrases of value (Mn/Bn USD) and volume (tons). Report involves detailed chapter on COVID 19 and its impact on this market. Additionally, it involves changing consumer behavior due to outbreak of COVID 19.

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Chapters Define in TOC (Table of Content) of the Report:

Chapter 1: Market Overview, Drivers, Restraints and Opportunities, Segmentation

Overview

Chapter 2: COVID Impact

Chapter 3: Market Competition by Manufacturers

Chapter 4: Production by Regions

Chapter 5: Consumption by Regions

Chapter 6: Production, By Types, Revenue and Market share by Types

Chapter 7: Consumption, By Applications, Market share (%) and Growth Rate by

Applications

Chapter 8: Complete profiling and analysis of Manufacturers

Chapter 9: Manufacturing cost analysis, Raw materials analysis, Region-wise

Manufacturing expenses

Chapter 10: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 11: Marketing Strategy Analysis, Distributors/Traders

Chapter 12: Market Effect Factors Analysis

Chapter 13: Market Forecast

Chapter 14: Cell and Gene Therapy Research Findings and Conclusion, Appendix, methodology and data source

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The qualitative contents for geographical analysis will cover market trends in each region and country which includes highlights of the key players operating in the respective region/country, PEST analysis of each region which includes political, economic, social and technological factors influencing the growth of the market. The research report includes specific segments by Type and by Application. This study provides information about the sales and revenue during the historic and forecasted period of 2020 to 2027.

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Covid-19 Impact On Cell and Gene Therapy Market Rising Trends Forecast to 2027 | Leading Players Amgen, Biogen, BioMarin Pharmaceuticals,...

INCOG BioPharma Services, a biopharmaceutical manufacturing services startup, announced on Oct. 15 that it will be establishing its first manufacturing facility and global headquarters in Fishers, Indiana. In addition, it will develop 150 new positions by the end of 2024.

The new facility will be approximately 60,000 square feet in size, and the company is investing $60 million in the construction of the building. With this facility in place, INCOG BioPharma will be able to launch its manufacturing operations and support biopharmaceutical businesses seeking to advance and accelerate early phase clinical projects.

Indiana is globally recognized as a leader in life sciences, ranking second in the nation for total exports," said Governor Eric J. Holcomb. "As a state, were committed to continuing to fuel this sector by providing the business-friendly environment and skilled workforce that companies like INCOG BioPharma need to launch operations and create quality careers for Hoosiers. We're grateful INCOG BioPharma chose to establish its global headquarters in the Hoosier state and look forward to supporting their growth in central Indiana."

More than 65 team members will be added to the company over the course of the next two years. Hiring will begin in early 2021 for positions in engineering, manufacturing sciences, quality assurance and other sectors.

Over the past several months, we evaluated several states and cities to identify the optimal location for our new venture," said Cory Lewis, founder and CEO of INCOG BioPharma. "It became clear that the state of Indiana and the city of Fishers was the ideal location, given the vast amount of life science industry experience that exists in the state. By locating our business in Fishers, we will have access to a large talent pool of skilled and trained resources. INCOG BioPharma is excited to partner with the state of Indiana and the city of Fishers as we build a life science company with a passion for customer service and a long-term vision focused on sustainability for both our employees and customers."

INCOG BioPharma is not the only company expanding in the biopharmaceutical industry. Pfizer, for example, announced back in August that it would be making a half billion dollar investment in the construction of its new gene therapy manufacturing facility. The structure, which is currently located in Sanford, N.C., will continue to support Pfizers investment in gene therapy research and development, similar to its Chapel Hill and Kit Creek, N.C., facilities.

This investment will further strengthen Pfizers leadership in gene therapy manufacturing technology, said Mike McDermott, President, Pfizer Global Supply. The expansion of the Sanford site is expected to create hundreds of highly skilled jobs, which would increase Sanfords high-tech manufacturing environment and is part of our overall plan to invest approximately $5 billion in U.S.-based capital projects over the next several years.

Through this new investment, the company will expand its end-to-end capabilities in gene therapy by investing in all stages of research, development and manufacturing. Pfizer will also become better suited to produce and supply both clinical- and commercial-scale quantities of potentially life-altering gene therapy medicines to patients with rare diseases.

At Pfizer, our purpose is breakthroughs that change patients lives, said Angela Hwang, Group President, Pfizer Biopharmaceuticals Group. Were excited to build this new state-of-the-art facility in Sanford because it will have the potential to help us develop novel methods to deliver transformative treatments to patients.

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INCOG BioPharma Paves the Way for New Jobs with Expansion in Indiana - BioSpace

Global Gene Therapy for Inherited Genetic Disorders Market: Overview

Rapid advances in mammalian DNA sequencing technologies over the past several years have enabled the identification of the aberrant genes responsible for a vast spectrum of genetic disorders. Gene therapy as a novel approach inarguably holds profound potential in finding universal therapeutic alternatives to treating inherited genetic disorders. Gene therapy for inherited genetic disorders entails introducing a functional copy of the defective gene to make up for the missing function, and can be accomplished using in vivo or ex vivo gene transfer.

Gene therapy for inherited genetic disorders has generated groundswell of interest in the research fraternity in finding cure for or in treatment of Mendelian genetic error causing rare diseases. Particularly, gene therapy in recent years has held promising potential in the treatment of a range of recessive gene disorders most notably sickle cell anemia, hemophilia, muscular dystrophy, cystic fibrosis, and other monogenic disorders. The axes of developments in the gene therapy for inherited genetic disorders market have been in the U.S., Europe, China, and Australia.

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Global Gene Therapy for Inherited Genetic Disorders Market: Notable Developments

Growing body of clinical studies done on mice models have unrivalled troves of preclinical data, which bodes well for the effectiveness of gene therapy for inherited genetic disorders. New approaches in the gene therapy for inherited genetic disorders market are being adopted to bring progress in this direction. In this regard, Salmeterol, a medicine approved for asthma, has shone a new light. The vasodilator to be used along with gene therapy has shown potential in increasing the effectiveness of the therapy for Glycogen storage disease type II (Pompe disease).

A team of investigator led by the researcher at Duke University Medical School discussed the preclinical data recently at 2019 annual meeting of the American Society of Gene & Cell Therapy. The preclinical data showed that the Asthma medicine reduces the accumulation of toxic glycogen accumulated in lysosome. The researchers concluded that it holds potential as an adjunctive therapy, and building on that may pave way for novel approaches on gene therapy for inherited genetic disorders.

Efforts to translate the findings of clinical research on gene therapy for inherited disorders to make the therapy a part of standard treatment has caught momentum in recent times. In this regard, vectors containing non-viral vectors have attracted the attention of scientists. A team of researchers at Fred Hutchinson Cancer Research Center in 2019 found that gold nanoparticles enable them to deliver gene-editing tools to blood stem cells in lab models. This might, they opined, pave way for more practicaland accessiblegene therapies for inherited disorders, notably for treating life-threatening blood disorders. Gene therapies were mediated by CRISPR. In the coming years they hope to collaborate with companies with commercial interest to develop the therapy for patient populations.

Some of the bigplayerseyeing promising stakes in the gene therapy for inherited genetic disorders market areSpark Therapeutics Inc., Orchard Therapeutics, Novartis AG, bluebird bio Inc., and BioMarin Pharmaceutical.

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Global Gene Therapy for Inherited Genetic Disorders Market: Key Drivers

Since 2000, scores of clinical trials involving patients with inherited genetic disorders have raised hopes of the medical fraternity of the potential of gene therapies. Thus far, more than 5000 clinical trials on gene therapy have been conducted, especially for hard-to-treat diseases. Diseases such as inherited blindness and leukemia have seen the efficacy and safety of gene therapies. Advances in bioengineering are expected to invigorate pre-clinical pipelines. In the not-so-distant future, success of more protocols will catalyze the prospects of the gene therapy for inherited genetic disorders market.

Further, advances have been made in viral and non-viral vectors with the purpose of making gene transfer more efficient, thereby boosting the gene therapy for inherited genetic disorders market. Particularly, new approaches emerged with the aim of making vectors more powerful.

Global Gene Therapy for Inherited Genetic Disorders Market: Regional Assessment

On the regional front, Asia Pacific bears considerable potential in the gene therapy for inherited disorders market. Of note, numerous strategic alliances have shifted their focus on the region, particularly China. The North America market has also been rising at a promising pace, driven by several gene-therapy tools and related drugs in the final stages of clinical trials. Favorable reimbursement models has also encouraged research into the gene therapy for inherited disorders.

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Gene Therapy for Inherited Genetic Disorders Market Robust pace of Industry during 2018-2028 - The Think Curiouser

-- Dr. Carl Regillo to present OPTIC data at the Retina Subspecialty Day onFriday, November 13, 2020 at 2:15 pm PT --

-- Company to host webcast with key opinion leaders on Saturday, November 14, 2020 at 7:30 am PT --

REDWOOD CITY, Calif., Oct. 20, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the presentation of additional clinical data for Cohorts 1-4 in the OPTIC Phase 1 clinical trial of ADVM-022 intravitreal injection gene therapy in wet age-related macular degeneration (wet AMD). This will be in the form of a live broadcast during the American Academy of Ophthalmology (AAO) 2020 Virtual Meeting.

Presentation of New OPTIC Data:Event: Retina Subspecialty Day 2020 Virtual MeetingTitle: Intravitreal Gene Therapy with ADVM-022 for Neovascular AMD: OPTIC Phase 1 StudyDate: November 13, 2020Time: 2:15 pm PTPresenter: Carl Regillo, M.D., F.A.C.S, chief of retina services at Wills Eye HospitalPresentation: At the beginning of Dr. Regillos presentation, Adverum plans to issue a press release and post the new data presentation under Events and Presentations in the Investors section of the companys website.

Presentation of Existing OPTIC Data:Event: AAO 2020 Virtual MeetingTitle: Phase 1 Study of Intravitreal Gene Therapy with ADVM-022 for Neovascular AMD (OPTIC Trial Cohorts 1-4, from the August 10, 2020 data presentation)Date: November 14-17, 2020Presenter: Arshad M. Khanani, M.D., M.A., managing partner and director of clinical research, Sierra Eye Associates, clinical associate professor of ophthalmology, University of NevadaPresentation: On or before November 13, 2020 the virtual presentations will be available on demand to AAO participants, and Adverum plans to post the presentation under Events and Presentations in the Investors section of the companys website.

Adverum Webcast:Date: November 14, 2020Time: 7:30 9:00 am PT (10:30 am 12:00 pm ET)Presenters:

The live video webcast will be accessible under Events and Presentations in the Investors section of the company's website. The archived audio webcast will be available on the Adverum website following the call and will be available for 30 days.

On November 5, 2020 after market, Adverum plans to report recent business progress and financial results for the third quarter of 2020. The Company plans to host the KOL webcast on November 14, 2020 to review new OPTIC data instead of hosting a quarterly conference call this quarter.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is advancing the clinical development of its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with wet age-related macular degeneration and diabetic macular edema. For more information, please visit http://www.adverum.com.

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Adverum Biotechnologies to Present New Data from the OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD at AAO 2020 Virtual -...

NEW YORK, Oct. 21, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") today announced that the National Institutes of Health has awarded Actinium a Small Business Technology Transfer grant to support a clinical collaboration with Memorial Sloan Kettering Cancer Center ("MSK") to study Iomab-ACT, Actinium's CD45-targeting Antibody Radio-Conjugate, for targeted conditioning to achieve lymphodepletion prior to administration of a CD19-targeted CAR T-cell therapy developed at MSK. The CD19 CAR-T has been previously studied by MSK in a Phase 2 trial with chemotherapy conditioning in patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL) or diffuse large B-cell lymphoma (DLBCL). MSK will lead this first of its kind study to utilize targeted radiopharmaceutical ARC-based lymphodepletion to replace chemotherapy-based conditioning prior to CAR T-cell therapy. The study will assess the feasibility of using Iomab-ACT targeted lymphodepletion prior to MSK's 19-28z CAR-T and assess safety and efficacy outcomes relative to results with MSK's CAR-T 19-28z in patients who had received chemotherapy-based lymphodepletion prior to CAR-T administration.

Results published in the New England Journal of Medicine with MSK's 19-28z CD19 CAR-T in 53 patients with R/R B-ALL reported complete remissions in 83% (44/53) of patients. Median event-free survival (EFS) was 6.1 months and median overall survival (OS) was 12.9 months at a median follow up period of 29 months (range 1 65 months) for all patients. Patients with low disease burden, defined as less than 5% blasts in the bone marrow, had markedly enhanced outcomes with increased median EFS of 10.6 months and median OS of 20.1 months. There was a 26% (14/53) rate of Grade 3 of greater cytokine release syndrome (CRS), with 1 patient death as a result, and 42% of patients experienced Grade 3-4 immune effector cell-associated neurotoxicity syndrome (ICANS). In addition to improved duration of response and survival, patients with low disease burden prior to receiving CAR T-cell therapy had lower rates of CRS and neurotoxicity.1

"We are excited to be collaborating with MSK on this trial as they are aleader in the field of cellular therapies. We selected MSK's 19-28z CAR T-cell therapy for this NIH grant funded collaboration because ithas produced high response rates in patients with relapsed or refractory B-ALL who have previously undergone several lines of standard therapy. However, toxicities such as cytokine release syndrome and neurologic toxicity, as well as durability of response, remain a challenge as is the case with many other CAR T-cell therapies" commented Dr. Mark Berger, Actinium's Chief Medical Officer. "Iomab-ACT enables the delivery of targeted radiation that selectively and specifically targets immune cells, including those implicated in the CAR-T-associated toxicities of cytokine release syndrome and neurotoxicity. We are hopeful that this study will demonstrate improvements in safety and outcomes with MSK's CAR 19-28z as a result of Iomab-ACT targeted lymphodepletion and that this will allow clinicians to make important improvements in patients' ability to receive CAR T-cell therapies."

CAR-T is a type of cellular therapy in which a patient's own (autologous) T-cells are genetically engineered outside of the body to target the patient's cancer cells and which are then reinfused back into the patient to seek out and kill cancer cells. Currently there are 2 approved CD19 targeted CAR-T therapies, which both require chemotherapy-based conditioning to deplete the patient's lymphocytes, known as lymphodepletion, and many other CAR-T constructs in development that also use chemotherapy conditioning for lymphodepletion.

Iomab-ACT targets cells that express CD45, an antigen found on immune cells such as lymphocytes and macrophages as well as leukemia and lymphoma cancer cells and delivers the radioisotope warhead iodine-131 to achieve cell depletion. Iomab-ACT is intended to deplete CD45+ immune cells such as macrophages that are implicated in CAR-T related toxicities and may also have an anti-tumor effect on chemo-refractory cancers. Iomab-ACT is a low dose extension of Actinium's lead program, Iomab-B, which is being studied in a pivotal Phase 3 trial for targeted conditioning prior to a bone marrow transplant. Preclinical data supporting Iomab-ACT's application in targeted lymphodepletion prior to ACT such as CAR-T was recently published in the journal Oncotarget (https://www.oncotarget.com/archive/v11/i39/). In addition, clinical data with trace doses of Iomab-B has shown transient, reversible lymphodepletion in patients and drug clearance pharmacokinetics that fit within the vein to vein time of CAR-T manufacturing and administration.

Sandesh Seth, Actinium's Chairman and CEO, said, "This clinical trial collaboration with MSK is a strong step forward for Actinium and our targeted conditioning program. The 19-28z CAR-T has already produced promising data and we look forward to working with MSK to explore Iomab-ACT's potential to reduce toxicities and improve patient outcomes. As we advance towards the SIERRA interim analysis in the fourth quarter, we are focused on the continued expansion of our ARC-based targeted conditioning program for bone marrow transplant and cell and gene therapies with the goal of providing targeted conditioning regimens that are less toxic and more effective than current chemotherapy-based conditioning. With these therapies being administered in a select number of concentrated centers, we see a large and growing market opportunity where our ARC-based targeted conditioning can improve outcomes and increase access to these important curative treatment options."

Iomab-ACT Program Update Call DetailsWebcast link: https://ir.actiniumpharma.com/presentations-webinarsDate: October 22, 2020Time: 8:00 AM EDT

Source:

1) Park et al. Long-Term Follow-up of CD19 CAR Therapy in Acute Lymphoblastic Leukemia. N Engl J Med 2018;378:449-59. DOI: 10.1056/NEJMoa1709919

About Actinium Pharmaceuticals, Inc.

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively kill patient's cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, CAR-T and other cell therapies or gene therapy to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, Iomab-B is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and promising single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. Beyond Iomab-B, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 120 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc.

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update, including but not limited to, statements relating to the Company's expectations regarding the intended use of proceeds of the public offering. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the SEC, including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Investor Contact:

Clayton RobertsonActinium Pharmaceuticals, Inc. [emailprotected]

Hans VitzthumLifeSci Advisors, LLC[emailprotected] P: (617) 430-7578

SOURCE Actinium Pharmaceuticals, Inc.

http://www.actiniumpharma.com/

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Actinium Pharmaceuticals, Inc. Awarded Grant by National Institutes of Health to Study Novel Iomab-ACT Targeted Conditioning with a CD19 CAR T-Cell...

Paris, October 21, 2020 SparingVision (the Company), a genomic medicine company focused on ocular diseases, today announces a 44.5 million financing round. The round was led by 4BIO Capital (4BIO) and UPMC Enterprises, and included Jeito Capital (Jeito)and Ysios Capital (Ysios). In addition, current investors Bpifrance and Foundation Fighting Blindness (FFB) participated in the round.

Proceeds from the financing will be primarily used to advance the development of SparingVisions breakthrough treatment SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa (RP). Most notably the funding will support SparingVisions GMP activities (including the manufacturing of a first clinical batch of the product, currently ongoing), the IND/CTA regulatory activities and the conduct of a first-in-man study, scheduled to start in 2021. The Company also intends to further expand its management team and commence operations in the US.

SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting ofone neurotrophic factor and one oxidative stress reducing enzymewhich, acting synergistically, aim to slow or stop the degeneration of photoreceptors. Loss of photoreceptors leads to blindness in RP, one of the most common inherited retinal diseasesthat affects two million patients worldwide.There is currently no treatment approved for RP patients independently of their genetic background.

Torreya Capital, LLC served as exclusive placement agent for the offering.

SparingVision also announces today that the Companys Chairman of the Board, Stphane Boissel, has been appointed Chief Executive Officer of the Company.

Stphane Boissel, SparingVisions President and Chief Executive Officer, said,We are delighted to have closed this financing round, which demonstrates the excitement around SparingVisions lead compound, SPVN06. With its singular mutation-agnostic approach, SPVN06 could have a much broader commercial potential than most gene therapy products for RP currently in development and will be used as an anchor to build an economically-viable portfolio of therapies in the field of ophthalmology. Our shareholders, both new and existing, are all long-term, strategic and patient-centric investors that share our vision and we are excited to be working with them to achieve our goals.

Before joining SparingVision as President and CEO, Stphane Boissel was Executive Vice President, Corporate Strategy at Sangamo Therapeutics (Sangamo), a Nasdaq-listed gene-editing company based in San Francisco, California. Prior to Sangamo, he was CEO of TxCell, the first ever CAR-TReg company, that was sold to Sangamo in 2018. Prior to TxCell, Stphane served as CEO of Genclis, a molecular diagnostic company, and EVP and CFO of Innate Pharma, a Nasdaq-listed company, and Transgene.

Stphane has also been a Board member of several leading biotechnology companies, including Nasdaq-listed Erytech Pharma and Elsalys Biotech, where he served as Chairman. Earlier in his career, Stphane worked in investment banking for Lazard, where he focused on principal investment in France, Singapore and Hong Kong. He started his career at PWC. Stphane graduated in management and finance from the IAE Lyon, University of Lyon and PSL Paris-Dauphine University (France) and received his MBA from the University of Chicago Booth School of Business.

Dr. Jos-Alain Sahel, Chair of the Department of Ophthalmology at the University of Pittsburgh School of Medicine, Director of the Institut de la Vision (Sorbonne Universite, Inserm, CNRS, Paris) and co-founder ofSparingVision along with Dr. Thierry Lveillard, added,SparingVisions neuroprotective approach, which I started working on some 20 years agoat Institut de la Vision, has all the attributes of a winning gene therapy solution for retinitis pigmentosa patients in desperate need of a universal therapeutic option. I am looking forward to helping Stphane and the team best position SPVN06 for clinical development up to regulatory licensing.

The Board of Directors of SparingVision expresses its deepest gratitude to Florence Allouche, co-founder, who is leaving her position as President of the Company, for her hard work and support over the years which has enabled the Company to pass many milestones and successfully refinance itself.

**ENDS**

Contacts:

SparingVision

Stphane Boissel

President and CEO

Stanislas Piot

CFO

Consilium Strategic Communications

Amber Fennell, Olivia Manser, Lizzie Seeley

+44 (0)20 3709 5700

sparingvision@consilium-comms.com

NOTES TO EDITORS:

About SparingVision:

SparingVision is a biotechnology company focused on the discovery and development of innovative therapies for the treatment of blinding inherited retinal diseases. SparingVision is developing SPVN06, a gene-independent treatment for retinitis pigmentosa, the most common inherited retinal degeneration. There is currently no treatment to treat all genetic forms of this rare retinal disease that leads to blindness and affects nearly 2 million people worldwide. SparingVision is a spin-off of the Paris Vision Institute. SparingVisions investors include4BIO Capital,Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre,UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visitwww.sparingvision.com

About 4BIO Capital:

4BIO Capital isan international venture capital firm focused solely on the advanced therapies sector. 4BIOs objective is to invest in, support, and grow early stage companies developing treatments in areas of high unmet medical need, with the ultimate goal of ensuring access to these potentially curative therapies for all patients. Specifically, it looks for viable, high-quality opportunities in cell and gene therapy, RNA-based therapy, targeted therapies, and the microbiome.

The 4BIO team comprises leading advanced therapy scientists and experienced life science investors who have collectively published over 250 scientific articles in prestigious academic journals includingNature,The Lancet,Cell, and theNew England Journal of Medicine. 4BIO has both an unrivalled network within the advanced therapy sector and a unique understanding of the criteria that define a successful investment opportunity in this space.

For more information, please visitwww.4biocapital.com

About UPMC Enterprises:

UPMC Enterprises is the innovation, commercialization, and venture capital arm of UPMC, a $21 billion health care provider and insurer based in Pittsburgh. With an emphasis on translational science and digital solutions, UPMC Enterprises provides its portfolio companies and partners with capital, connections and resources to develop solutions to health cares most complex problems. Working in close collaboration with innovators from UPMC and the University of Pittsburgh Schools of the Health Sciences, as well as others worldwide, UPMC Enterprises strives to accelerate science from the bench to the bedside and has committed to investing $1 billion in novel drugs, diagnostics and devices by 2024.

For more information, please visitwww.enterprises.upmc.com

About Jeito Capital:

Jeito Capital is an international investment company with a patient benefit driven approach that focuses both on financing ground-breaking medical innovation and promoting positive societal impact. Jeito has a unique, long-term investment strategy, with the aim of providing continuity from clinical development to market access for breakthrough drugs with validated proofs of concept. This continuity is expressed both in the support provided to entrepreneurs by the Jeito Capital team and by the investment of significant capital to ensure the growth of companies, the acceleration towards commercialization and faster access to these major innovations. Jeito Capital is based inPariswith a presence inEuropeand the United States.

About Ysios Capital:

Ysios Capital is a leading Spanish independent venture capital firm that provides private equity financing to early and mid-stage life science companies that develop disruptive therapeutic products and platform technologies. Founded in 2008, Ysios Capital manages c.a. 350 M through three dedicated funds in life sciences and is led by a team of professionals experienced in building biotech companies.For more information, please visit:www.ysioscapital.com

About Bpifrance:

Bpifrance is the French national investment bank. It finances businesses at every stage of their development through loans, guarantees, equity investments and export insurances. Bpifrance also provides extra financial services (training, consultancy) to help entrepreneurs meet their challenges (innovation, export). For more information, please visit:www.bpifrance.comandpresse.bpifrance.fr. Follow us on Twitter: @Bpifrance @BpifrancePresse

About the Foundation Fighting Blindness:

Established in 1971, the Foundation Fighting Blindness is the worlds leading private funding source for retinal degenerative disease research. The Foundation has raised more than $800 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the entire spectrum of retinal degenerative diseases including: retinitis pigmentosa, age-related macular degeneration, Usher syndrome, and Stargardt disease. VisitFightingBlindness.orgfor more information.

AboutFondation Voir et Entendre:

Created in May 2007, the Foundation"Voir et Entendre"'s challenges are to defy sensory handicaps linked to vision and hearing, both of which have become public health stakes. As we can see throughout industrialized countries ocular and auditory diseases are gaining ground, mainly due to the ageing populations.

The Foundation therefore brings together the best-known French and international scientific teams, hospital services and industries around common research themes, with the aim to rapidly find new and adapted treatments.

Within the Foundation is the research center Vision Institute managed by Jos-Alain Sahel, the Clinical Investigation Centre of the National Ophthalmologic Hospital Centre (CHNO) of the Quinze-Vingts, reference centers for rare diseases and four clinical hospital departments.

The head office is located at the Vision Institute, itself within the CHNO of the Quinze-Vingts in Paris. The latter has supported this project from the outset along with the Pasteur Institute, National Institute of Health and Medical Research (INSERM), Pierre and Marie Curie University and the French Federation of the Blind and Visually Impaired.

https://www.institut-vision.org/fr/institute/2-uncategorised/8-the-foundation.html

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SparingVision Raises 44.5 Million and Appoints Stphane Boissel as Chief Executive Officer - BioSpace

Swiss pharma giant Roche has partnered with Cambridge, Massachusetts-based Dyno Therapeutics for the development of gene therapies for central nervous system (CNS) and liver diseases.

The companies will utilise Dynos CapsidMap platform to develop adeno-associated virus (AAV) vectors for gene therapies targeting a range of CNS and liver-directed diseases.

Dynos CapsidMap artificial intelligence platform will be used to identify novel AAV capsids it does this by optimising tissue targeting and immune-evading properties, as well as improving packaging capacity and manufacturability, according to Dyno.

Dynos technology uses artificial intelligence to design novel capsids, the cell-targeting protein shell of viral vectors. Current gene therapies are limited to small number of naturally occurring AAV vectors, which face challenges with delivery, immunity, packaging size and manufacturing.

As part of the collaboration, Dyno will be responsible for the design of the novel AAV capsids, while Roche and its Spark Therapeutics division will develop the capsids in preclinical studies, with the aim of taking them through clinical development and to commercialisation.

In return for access to its capsid identifying technology, Dyno will receive an undisclosed payment as well as further payments related to research, clinical and sales milestone payments.

In addition to potential royalties for any resulting products developed from the collaboration, Dyno could be eligible to receive payments over a total value of $1.8bn.

This new partnership represents Dynos largest collaboration to date, and we are excited to work with Roche and Spark Therapeutics to expand the frontier of gene therapies for the central nervous system and liver, said Eric Kelsic, chief executive officer and co-founder of Dyno.

"The Dyno, Roche and Spark teams share a bold vision for gene therapy and believe that enhancing vectors that deliver these therapies is key to developing new treatments for patients in need, he added.

The Roche deal is Dynos third pharma collaboration, in addition to its existing partnerships with Novartis and Sarepta Therapeutics.

In May, Dyno and Novartis announced that they had signed a deal focused on developing improved gene therapies with AAV vectors for research, development and commercialisation of gene therapies for ocular disease.

The Sarepta deal is focused on the development of new AAV vectors for gene therapies aimed at targeting muscle diseases.

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Roche to collaborate with Dyno on gene therapies for CNS/liver diseases - PMLiVE

MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News:

Sensorion (Paris:ALSEN) (FR0012596468 ALSEN), a pioneering clinical-stage and gene therapy biotech company which specializes in the development of novel therapies to treat, prevent and restore within the field of hearing loss disorders, today announces its interim annual results as of June 30, 2020 alongside its outlook for 2020.

Sensorions landmark gene therapy agreement with Institut Pasteur was a major inflection point in the Companys development. The positive preliminary preclinical data from the program targeting the OTOF gene reinforces the potential of this partnership, which expands our pipeline and supports our goal to become a leader in the field of hearing loss. The Phase 2 trial of SENS-401 in sudden sensorineural hearing loss is progressing and we expect results in mid-2021. We were delighted to successfully close an oversubscribed private placement in September, which raised 31 million. As part of this financing, we were pleased to see renewed support from existing shareholders such as Invus and Sofinnova Partners and to welcome new high-quality US and European investors. Following this financing, we estimate the cash in hand will carry us through to H2 2022 comments Nawal Ouzren, CEO of Sensorion.

First-half 2020 financial results

The half-year accounts as of June 30, 2020, drawn up according to IFRS standards and approved by the Board of Directors on October 20, 2020, have been duly reviewed by statutory auditors.

The simplified income statement as of June 30, 2020 is as follows:

In Euros IFRS standards

30.06.2020

30.06.2019

Operating income

902,203

1,042,407

Research and Development expenses

3,661,766

5,226,883

General and Administrative expenses

1,915,400

1,257,185

Total operating expenses

5,577,166

6,484,068

Operating profit/loss

-4,674,963

-5,441,662

Financial profit/loss

-44,031

-22,929

Net profit/loss

-4,718,994

-5,464,591

On June 30, 2020, Sensorions operating income, mainly the research tax credit, amounted to 0.9m, compared to 1.0m on June 30, 2019.

Operating expenses decreased by 14%, down from 6.5m on June 30, 2019 to 5.6m on June 30, 2020, mainly due to a 1.5m reduction in research costs partially offset by a 0.7m increase in G&A expenses.

The research and development expenses decreased by 30%, down from 5.2m on June 30, 2019 to 3.7m on June 30, 2020 following the halting of the SENS-111 program in December 2019 and the slowdown of expenditure on the SENS-401 clinical trial as a result of delays due to the COVID-19 pandemic.

G&A expenses are up 52%; they amounted to 1.9m on June 30, 2020, compared with 1.3m on June 30, 2019 mainly due to the increase in personnel expenses.

Operating losses on June 30, 2020 thus amounted to 4.7m, compared with a loss of 5.4m on June 30, 2019.

Net loss amounted to -4.7m on June 30, 2020, compared with -5.5m on June 30, 2019.

As of June 30, 2020, the company employed 24 people.

Financial structure

On February 10, 2020, Invus Public Equities LP converted all the 12,500,000 convertible bonds (CBs) it had subscribed for in June 2019 into ordinary shares in the Company. The conversion was undertaken on a price basis of 0.76 per share. Following this operation, Invus held 20,591,259 ordinary shares and 42.29% of the share capital and voting rights in Sensorion.

On February 13, 2020, Sofinnova Crossover I SLP converted all the 7,500,000 convertible bonds (CBs) it had subscribed for in June 2019 into ordinary shares in the Company. The conversion was undertaken on a price basis of 0.76 per share. Following this operation, Sofinnova Crossover I SLP held 11,822,258 ordinary shares and 20.19% of the share capital and voting rights in Sensorion.

Equity capital amounted to 28.7m on June 30, 2020, compared with 13.2m on June 30, 2019.

As of June 30, 2020, cash and cash equivalents amounted to 30.7m compared with 30.4m on December 31, 2019.

On September 18, 2020, Sensorion successfully raised 31m of gross proceeds before deducting underwriting commissions and estimated expenses payable by the Company.

Based on its forecasted expenses, the cash position of 30.7m at June 30, 2020 and the net proceeds from the offering, the Company believes it will be able to fund its operations until the second half of 2022.

Key developments: Science and research & development

In the second half of 2019, Sensorion launched two preclinical gene therapy programs targeting Usher Syndrome type 1 and Otoferlin deficiency, two monogenic forms of hereditary deafness. Under the framework agreement signed with Institut Pasteur in May 2019, other projects could also emerge in the area of genetic disorders of the inner ear. During the five years partnership agreement, Sensorion has preferred rights to the genetic disorders of the inner ear research pipeline of Institut Pasteur and the ability to implement collaborations leading to a license. These programs are conducted under the sponsorship of Professor Christine Petit, Director of the French Hearing Institute and Chair of our Scientific Advisory Board.

On June 9, 2020, Sensorion announced positive preliminary preclinical data from its gene therapy program targeting Otoferlin deficiency. In vivo experiments conducted in non-human primates (NHPs) show good safety and promising preliminary data on inner ear tissue tropism and the achievement of a high transduction rate efficiency.

The SENS-401 Phase 2 clinical trial in the treatment of sudden sensorineural hearing loss (SSNHL) in adults is a randomized, double-blind and placebo-controlled study, aiming to recruit ~260 patients. It is being conducted in 11 countries at approximately 30 sites in Europe and Canada.

On February 17, 2020, Sensorion received Ethics Committee approval to include new military sites in the SENS-401 Phase 2 study. The new centers will recruit volunteer military personnel exposed to extreme noise during their professional activities and suffering from hearing loss.

On March 13, 2020, Sensorion provided an update on the SENS-401 SSNHL Phase 2 AUDIBLE-S trial enrollment. Patient recruitment rates from this trial now indicate the data will be available by mid-year 2021, which is later than previously announced. An important factor resulting in delayed recruitment in the trial was the reallocation of emergency room resources due to the COVID-19 situation.

The independent Data Safety Monitoring Board (DSMB) undertook a review of the safety data for the patients included in the Phase 2 clinical trial on June 5, 2020. It confirmed the absence of any concern on the safety of SENS-401 and recommended continuing the trial as scheduled.

Following the agreement signed in December 2017, Sensorion and Cochlear (world leader in cochlear implants) have continued their collaboration. Thanks to its otoprotective properties demonstrated in several preclinical models, SENS-401 could potentially preserve residual hearing in patients with cochlear implants. Since 2018, we have successfully conducted additional safety studies to assess the feasibility of long-term treatment with SENS-401 that may be required in cochlear implant indications. Preclinical data from these studies are expected by the end of 2020.

Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases enabling it to select the best targets and modalities for drug candidates in the field of small molecules and gene therapy. This platform makes it possible to carry out a panel of investigations ranging from histology and cell culture (in vitro) to behavioral and electrophysiological tests (in vivo). The Company is also working on the identification of biomarkers to improve diagnosis and treatment of these illnesses with a high unmet medical need.

On January 30, 2020, Sensorion presented new SENS-401 preclinical data at the ARO (Association for Research in Otolaryngology) Mid-Winter Meeting 2020. A poster and oral presentation highlighted the potential to significantly reduce hearing loss from chronic noise exposure in a rat model. A second poster featured the age-related hearing loss with significant early decline in functional auditory measures in Wistar rats.

Capital breakdown after the September 2020 capital increase

Sensorions financial position strengthened further at the end of September 2020 following a 31m capital increase. The capital raise was achieved at a share price which was a 3.5% discount over the weighted average share price on the day preceding the date on which the issuance price was set (the Reserved Offering).

The completion of this capital increase was, amongst others, supported by existing shareholders, Invus Public Equities LP, Sofinnova Partners and WuXi AppTec.

To the best of the Companys knowledge the capital structure on a non-diluted basis before and after the private placement is as follows:

Shareholders

Number of shares before the Reserved Offering (1)

% of the share capital before the Reserved Offering

% of voting rights before the Reserved Offering

Number of shares after the Reserved Offering (1)

% of share capital after the Reserved Offering

% of voting rights after the Reserved Offering

Subscription

(in )

Inserm Transfert Inititiative

982,911

1.68%

1.68%

982,911

1.28%

1.28%

-

Innobio (Bpifrance)

3,499,874

5.98%

5.98%

3,499,874

4.56%

4.56%

-

Management, employees and directors

160,000

0.27%

0.27%

160,000

0.21%

0.21%

-

Cochlear

533,755

0.91%

0.91%

533,755

0.70 %

0.70%

-

Invus Public Equities LP

See the article here:
Sensorion reports 2020 first half results - Business Wire

Four years after a Phase I/II setback in spinal muscular atrophy (SMA), Novartis is hoping its drug branaplam will find more success in a new neurological indication: Huntingtons disease.

The decision was announced a year after the head of research, Jay Bradner, said he did not see a big opportunity in SMA, according to Reuters. Novartis says it has preclinical data showing that branaplam reduces levels of mutant huntingtin protein, and SMA data showing patients on the drug had reductions in huntingtin mRNA. The FDA gave branaplam their orphan drug designation, and Novartis plans to move forth with a Phase IIb trial next year.

Novartis intends to start a development program for branaplam to determine if it has the potential to be a transformative treatment for people living with this devastating condition, the company said in a statement.

Novartis isnt dropping the SMA indication altogether according to a company statement, branaplam is still under investigation for SMA. But a 2-year testing hiatus set Novartis behind its rival Roche, which won approval for its SMA therapy Evrysdi last year.

In 2016, Novartis halted enrollment in a Phase I/II study in SMA after signs of nerve injury appeared in some animal tests, according to a letter it released. The trial was resumed about 2 years later in Belgium, Germany, Denmark and Italy, with additional nerve tests added to the protocol.

It is still a long road, but we are pleased to be back on track, the letter read.

Evrysidi is priced by weight, beginning at less than $100,000 for infants under 15 pounds and capping at $340,000 per year. Analysts predicted that the drug would threaten Biogens SMA drug Spinraza, which comes in at $382,500 on the low-end. But Mani Foroohar of SVB Leerink predicted Novartis gene therapy Zolgensma would take the lead in young SMA patients. Given strong data, one-time therapy, and simple IV administration, we see Zolgensma as remaining the treatment of choice for very young SMA patients, he wrote, according to aFierce Pharma report.

View post:
Years after a major trial setback, Novartis switches gears with SMA drug. This time they're trying it for Huntington's - Endpoints News

COVID-19 impact will also be included and considered for forecast.

Global Gene Therapy Market research report provides detail information about Market Introduction, Market Summary, Global market Revenue (Revenue USD), Market Drivers, Market Restraints, Market Opportunities, Competitive Analysis, Regional and Country Level.

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Gene Therapy Market: 2020 Opportunities, Key Players, Competitive and Regional Analysis by Forecast 2025 - The Think Curiouser