StemCell Therapy

In March, Novartis was able to announce that Zolegensma (onasemnogene abeparvovec) that it had received a positive opinion on its application for marketing approval by the European Medicines Agency.

Yesterday this was followed up by the announcement that the European Commission (EC) had provided the gene therapy conditional approval for the treatment of patients with spinal muscular atrophy. The treatment will be available for those with a bi-allelic mutation in the SMN1 gene and a diagnosis of SMA type 1; or for patients with SMA with the mutation and up to three copies of the SMN2 gene.

The company added that the approval also covers babies and young children with SMA, up to a body weight of 21kg (46 pounds).

Novartis stated that the treatment will be made available immediately in France and that Germany would gain access shortly.

In terms of pricing, the company noted that it was working with stakeholder organizations across Europe, so that pricing would "work within existing, local pricing and reimbursement frameworks."

The pricing has proved a controversial aspect of the one-time gene therapy, which entered the US market at a cost of $2.1m (1.9m) per patient.

In its announcement, the company countered that the cost of caring for a child with SMA to the healthcare system is approximately 2.5m to 4m for 10 years of care.

In fourth quarter results, the treatment saw sales of $170m in the US.

Read more here:
Novartis' gene therapy approved in Europe, talks ongoing on price - BioPharma-Reporter.com

LONDON, May 26, 2020 /PRNewswire/ -- Beacon Targeted Therapies, a Hanson Wade Ltd company, is a leader in bespoke and tailored clinical trial and drug development database products in targeted therapies providing accurate, in-depth, and real-time information on the rapidly evolving drug development landscapes.

Today we announce the launch of our latest database solution Beacon Gene Therapy, which addresses the growing demands on accurately tracking the gene therapy space with the latest scientific and technological innovations and monitoring competitors.

Our sector-specific, modular solution brings together all publicly known available sources of data on gene therapies and houses them under one repository. It is manually curated and includes clinical trial and drug records for preclinical, active, approved, and discontinued assets for all types of therapies where the gene/genetic material is being transferred to the patient, either through cell-free or cell-based mechanisms, aimed at treating non-oncology indications.

Beacon Gene Therapy Includes:

Beacon Gene Therapy allows you to search the clinical trial and drug landscape by:

Allowing you to cut through the noise and focus in on the literature and insights you need for evidence decision-making on your drug development programs.

Our sources cover (but are not limited to):

"Beacon is in a unique position to provide drug developers in the gene therapy space a complete clinical trial and drug development pipeline overview. To keep up with the many technological innovations and challenges in the space such as immunogenicity." - Curtis Dingley Commercial Director, Beacon Targeted Therapies

For more information the Beacon Gene Therapy database solution, please visit: https://beacon-intelligence.com/gene-therapy.

About Beacon Targeted Therapies:

Beacon Targeted therapies is a Hanson Wade Ltd company a world leader in conference and data products in high growth industries. Beacon Targeted Therapies is a clinical trial and pipeline database solution, designed in partnership with pharmaceutical professionals, tracking targeted therapies to provide accurate, in-depth, and real-time information in the rapidly evolving landscape of drug development.

For Media Enquiries: J-Ney AntiqueBeacon Targeted Therapies Hanson Wade+44-(0)203-141-8700Beacon@hansonwade.comwww.beacon-intelligence.com

View original content:http://www.prnewswire.com/news-releases/beacon-targeted-therapies-launches-its-latest-clinical-trial-and-drug-database-solution---beacon-gene-therapy-301060198.html

SOURCE Beacon Targeted Therapies

Continued here:
Beacon Targeted Therapies Launches Its Latest Clinical Trial and Drug Database Solution - Beacon Gene Therapy - BioSpace

By Jonny Lupsha, News Writer

According to the Fierce Biotech article, the mice who underwent the new gene therapy were injected with a gene that makes the protein follistatin, which in turn blocks a protein called myostatin. Myostatin regulates muscle growth. The therapy caused a significant buildup of muscle mass in the mice while also preventing obesity, the article said. The mice didnt just build muscle; they also nearly doubled their strength without exercising any more than they usually did. Despite being fed a high-fat diet, they had fewer metabolic issues and stronger hearts than did animals that did not receive the follistatin gene.

Scientists have been developing gene therapy for many years. It can change our bodies in many ways, and has potential serving as a treatment for cancer and muscular dystrophy.

The procedure that the mice underwent encapsulates what gene therapy isalthough scientists generally focus on people.

I define [gene therapy] as the addition of genes to humans for medical purposes, said Dr. David Sadava, Adjunct Professor of Cancer Cell Biology at the City of Hope Medical Center.

Dr. Sadava said gene therapy is based on four assumptions. First, whoever is doing the gene therapy has to know the gene thats involved in whichever problem needs to be treated. Second, they must have a normal, healthy copy of that gene available in the lab. Third, they must know where and when the gene is normally expressed. Finally, they have to be fairly certain what will happen when the gene is expressed normally.

Additionally, gene therapy must do several things in order to be considered successful.

First, gene therapy must get the gene into the appropriate cells, Dr. Sadava said. Second, gene therapy must get the gene expressed in those cells. Third, we have to get the gene integrated into the genome of the target cells so itll be there permanently. And fourth, you better not have any bad side effects to gene therapy, like any therapy in medicine.

According to Dr. Sadava, one kind of gene therapy is referred to as gene augmentation, and it comes into play when the functional product of a gene has been lost and no longer gets produced normally. By injecting a gene into someone, healthy copies of a protein product will be made and function restored.

We could hypothetically think of muscular dystrophy as a good target for gene therapy, he said. We know that muscles lack the protein dystrophinits an organizing proteinso well put in the good gene for good dystrophin.

Another kind of gene therapy is called target cell killing. Dr. Sadava said it uses a gene that either produces a poison that kills certain types of cells or it stimulates the immune system to do so. Target cell killing can be applied to cancer.

A gene is put into cancer cells that allows them to produce a protein that will make a toxic drug from a harmless chemical, Dr. Sadava said. So the idea is we inject a harmless chemical into the body, it goes all over the body and when it enters a tumor cell, its converted into a poison by the gene product of the gene that weve put in for gene therapy. So we might put in a gene that will cause a protein to be made that attracts killer T cells so the tumor will stick up its hand and say Come kill me now.'

Gene therapy is an exciting field in science and medicine with a lot of potential for humans. For now, it may seem like its just helping some overweight mice get a confidence boost, but the practical applications should shore up within our lifetime.

Dr. David Sadava contributed to this article. Dr. Sadava is Adjunct Professor of Cancer Cell Biology at the City of Hope Medical Center in Duarte, CA, and the Pritzker Family Foundation Professor of Biology, Emeritus, at The Claremont Colleges. Professor Sadava graduated from Carleton University with a B.S. with first-class honors in biology and chemistry. He earned a Ph.D. in Biology from the University of California, San Diego.

Read the rest here:
Lab Mice Shed Fat and Build Muscle with Gene Therapy - The Great Courses Daily News

BOSTON and LONDON, May 27, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that presentations by the management team will be made at the following investor conferences in June:

Live webcasts of the presentations will be available under "News & Events" in the Investors & Media section of the company's website at http://www.orchard-tx.com. Webcast replays will be archived on the Orchard website following the presentation.

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

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us onTwitterandLinkedIn.

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

Contacts

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

Continued here:
Orchard Therapeutics to Present at Virtual Investor Conferences in June - GlobeNewswire

OXFORD, England, May 27, 2020 /PRNewswire/ -- Evox Therapeutics Ltd ('Evox' or the 'Company'), a leading exosome therapeutics company, is pleased to announce the appointment of Martin Andrews as a Non-Executive Director. Martin is a highly experienced senior pharmaceutical executive with broad R&D, commercial and operational experience, and has deep specialist expertise in rare diseases, gene therapy and vaccines.

Martin is an experienced Non-Executive Director and commercial leader, with a strong track record of strategy development and operational delivery. He has had a long and successful career at GlaxoSmithKline, where he has held many senior positions. Most recently, Martin was Senior Vice President, Rare Diseases. Here, he led the global rare disease business and oversaw the development of a portfolio of ex vivo gene therapies, and the launch of Strimvelis, the world's first life-saving gene therapy for children. Furthermore, under his leadership, GlaxoSmithKline transferred its gene therapy portfolio to Orchard Therapeutics. Prior to that, Martin was Senior Vice President, Global Vaccines Commercial, where he led the development of the growth strategy and transformation of the commercial capability in GlaxoSmithKline's Vaccines division.

Martin has previously held Board positions at Orchard Therapeutics and the Alliance for Regenerative Medicine. He is currently a Non-Executive Director of Freeline Therapeutics, where he brings his commercial expertise to their gene therapy portfolio of drugs.

Dr Antonin de Fougerolles, Chief Executive Officer of Evox, commented:

"We're very pleased to welcome Martin as a Non-Executive Director. With his rare disease drug development experience and strong track-record of commercial success, Martin will be a great asset to the company. His expertise will play an important role in helping guide our business growth."

Commenting on his appointment, Martin Andrews said:

"I'm delighted to be joining the Board of Evox.This is an exciting period for the Company and I believe its technology has the potential to transform how medicines are developed and delivered for patients with conditions that are not possible to treat adequately today, especially those with rare diseases. I am thrilled to be part of the team and look forward to contributing."

About Evox Therapeutics

Evox Therapeutics is a privately held, Oxford-based biotechnology company focused on harnessing and engineering the natural delivery capabilities of extracellular vesicles, known as exosomes, to develop an entirely new class of therapeutics. Backed by leading life sciences venture capital groups and supported by a comprehensive intellectual property portfolio, Evox's mission is to positively impact human health by creating novel exosome-based therapeutics for the treatment of various severe diseases with limited options for patients and their families. Evox has created substantial proprietary technology to modify exosomes using various molecular engineering, drug loading, and targeting strategies to facilitate targeted drug delivery to organs of interest, including the brain and the central nervous system. Exosome-based drugs have the potential to address some of the limitations of protein, antibody and nucleic acid-based therapies by enabling delivery to cells and tissues that are currently out of reach using other drug delivery technologies, and Evox is leading the development within this emerging therapeutic space.

For further information visit: http://www.evoxtherapeutics.com

View original post here:
Evox Therapeutics Appoints Martin Andrews as Non-Executive Director - WFMZ Allentown

CAMBRIDGE, Mass.--(BUSINESS WIRE)--ElevateBio, LLC, a Cambridge-based creator and operator of a portfolio of innovative cell and gene therapy companies, announced that the company will present at the Jefferies Virtual Healthcare Conference on Thursday, June 4, 2020 at 8:30 a.m. ET.

About ElevateBio

ElevateBio, LLC, is a Cambridge-based creator and operator of a portfolio of innovative cell and gene therapy companies. It begins with an environment where scientific inventors can transform their visions for cell and gene therapies into reality for patients with devastating and life-threatening diseases. Working with leading academic researchers, medical centers, and corporate partners, ElevateBios team of scientists, drug developers, and company builders are creating a portfolio of therapeutics companies that are changing the face of cell and gene therapy and regenerative medicine. Core to ElevateBios vision is BaseCamp, a centralized state-of-the-art innovation and manufacturing center, providing fully integrated capabilities, including basic and transitional research, process development, clinical development, cGMP manufacturing, and regulatory affairs across multiple cell and gene therapy and regenerative medicine technology platforms. ElevateBio portfolio companies, as well as select strategic partners are supported by ElevateBio BaseCamp in the advancement of novel cell and gene therapies.

ElevateBios investors include F2 Ventures, MPM Capital, EcoR1 Capital, Redmile Group, Samsara BioCapital, The Invus Group, Surveyor Capital (A Citadel company), EDBI, and Vertex Ventures HC.

ElevateBio is headquartered in Cambridge, Mass, with ElevateBio BaseCamp located in Waltham, Mass. For more information, please visit http://www.elevate.bio.

Read the rest here:
ElevateBio to Present at the Jefferies Virtual Healthcare Conference - Business Wire

Gilead CEO Dan ODay has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosens Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. ODay also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. Theres $500 million in potential TIGIT milestones on US regulatory events likely capped by an approval if Gilead partners on it and the stars align on the data. And theres another $150 million opt-in payments for the rest of the Arcus pipeline.

Unlock this story instantly and join 82,300+ biopharma pros reading Endpoints daily and it's free.

SUBSCRIBE SIGN IN

Read more here:
European regulators accept FibroGen's anemia drug for review; Passage Bio's lead gene therapy gets more love from the FDA - Endpoints News

NewsEnvironmentResearchers at Queen Mary University of London and the Royal Botanic Gardens, Kew, discovered genes that create chemicals harmful to insects

Monday, 25th May 2020, 11:22 pm

A set of genes has been identified that could protect ash trees from a deadly beetle, which attacks by burrowing into their stems.

Named the Emerald Ash Borer (EAB), the killer pest is expected to destroy hundreds of millions of trees worldwide in the years to come without any intervention.

Researchers at Queen Mary University of London and the Royal Botanic Gardens, Kew, discovered genes that create chemicals likely to be harmful to the insects.

They sequenced the genomes of 22 types of ash tree and used this information to analyse how the different species are related to each other.

Help trees fight deadly beetles

Meanwhile, the US Department of Agriculture Forest Service, in Ohio, tested the resistance of more than 20 ash species to EAB by hatching eggs on the bark of trees, and following the fate of the beetle larvae.

Resistant ash trees killed the larvae when they burrowed into their stems, but susceptible ones did not.

The scientists discovered 53 candidate resistance genes, several of which are involved in making chemicals that are likely to be harmful to insects.

The findings suggest that breeding or gene editing could be used to place these resistance genes into ash species currently affected by EAB.

Dr Laura Kelly, an academic visitor at Queen Mary and lead author of the study, published in the journal Nature Ecology & Evolution, said: Knowledge of genes involved in resistance will...help efforts to identify trees that are able to survive the ongoing threat from EAB, and in turn, could facilitate restoration of ash woodlands in areas which have already been invaded."

Originally posted here:
Gene therapy could help millions of ash trees fight deadly beetle Emerald Ash Borer - inews

PENNINGTON, N.J. and SAN DIEGO, May 27, 2020 /PRNewswire/ --OncoSec Medical Inc.(NASDAQ:ONCS) (the "Company" or "OncoSec"), a company developing late-stage intratumoral cancer immunotherapies, today announced Notices of Allowance for three new patent applications covering its interleukin-12 (IL-12) based immunotherapy platform, including its lead product candidate TAVO and next-generation product candidate TAVOPLUS, and electroporation gene delivery system.

"OncoSec continues to prioritize the expansion of its intellectual property portfolio across multiple facets of its technology," said Keir Loiacono, Vice President, Corporate Development at OncoSec Medical Incorporated. "These three new allowances demonstrate OncoSec's strong patent position in three critical areas: device development, new treatment alternatives and novel adjuvant therapies with previously undescribed combinations. As we look to the future, we believe this will not only help to secure our competitive position in the intratumoral oncology space, but potentially create new opportunities for licensing revenue."

About OncoSec Medical IncorporatedOncoSec Medical Incorporated (the "Company," "OncoSec," "we" or "our") is a late-stage biotechnology company focused on developing cytokine-based intratumoral immunotherapies to stimulate the body's immune system to target and attack cancer.OncoSec's lead immunotherapy investigational product candidate TAVO (tavokinogenetelseplasmid) enables the intratumoral delivery of DNA-based interleukin-12 (IL-12), a naturally occurring protein with immune-stimulating functions.The technology, which employs electroporation, is designed to produce a controlled, localized expression of IL-12 in the tumor microenvironment, enabling the immune system to target and attack tumors throughout the body. OncoSec has built a deep and diverse clinical pipeline utilizing TAVO as a potential treatment for multiple cancer indications either as a monotherapy or in combination with leading checkpoint inhibitors; with the latter potentially enabling OncoSec to address a great unmet medical need in oncology: anti-PD-1 non-responders.Results from recently completed clinical studies of TAVO have demonstrated a local immune response, and subsequently, a systemic effect as either a monotherapy or combination treatment approach along with an acceptable safety profile, warranting further development. In addition to TAVO, OncoSec is identifying and developing new DNA-encoded therapeutic candidates and tumor indications for use with its new Visceral Lesion Applicator (VLA), to target deep visceral lesions, such as liver, lung or pancreatic lesions. For more information, please visitwww.oncosec.com.

TAVO is a trademark of OncoSec Medical Incorporated.

Risk Factors and Forward-Looking StatementsThis release, as well as other information provided from time to time by the Company or its employees, may contain forward-looking statements that involve a number of risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements. Forward-looking statements provide the Company's current beliefs, expectations and intentions regarding future events and involve risks, uncertainties (some of which are beyond the Company's control) and assumptions. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. You can identify forward-looking statements by the fact that they do not relate strictly to historical or current facts. These statements may include words such as "anticipate," "believe," "could," "estimate," "expect," "intend," "may," "plan," "potential," "should," "will" and "would" and similar expressions (including the negative of these terms). Although we believe that expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements. The Company intends these forward-looking statements to speak only at the time they are published on or as otherwise specified, and does not undertake to update or revise these statements as more information becomes available, except as required under federal securities laws and the rules and regulations of the Securities Exchange Commission ("SEC"). In particular, you should be aware that the success and timing of our clinical trials, including safety and efficacy of our product candidates, patient accrual, unexpected or expected safety events, the impact of COVID-19 on the supply of our candidates or the initiation or completion of clinical trials and the usability of data generated from our trials may differ and may not meet our estimated timelines. Please refer to the risk factors and other cautionary statements provided in the Company's Annual Report on Form 10-K for the fiscal year ended July 31, 2019 and subsequent periodic and current reports filed with the SEC (each of which can be found at the SEC's websitewww.sec.gov), as well as other factors described from time to time in the Company's filings with the SEC.

Company Contact:Gem HopkinsHead of Corporate Communications858-210-7334ghopkins@Oncosec.com

View original content to download multimedia:http://www.prnewswire.com/news-releases/oncosec-strengthens-ip-portfolio-with-three-new-patents-covering-tavo-and-its-electroporation-gene-delivery-system-301066037.html

SOURCE OncoSec Medical Incorporated

Read more from the original source:
OncoSec Strengthens IP Portfolio with Three New Patents Covering TAVO and Its Electroporation Gene Delivery System - BioSpace

R.A. Session II, president, CEO, and founder of Taysha Gene Therapies [Background image: Olena Yepifanova via iStock]

Dallas-based biotech Taysha Gene Therapies has emerged out of stealth with a $30 million seed round and a new strategic partnership with UT Southwestern. The company aims to end severe and life-threatening diseases of the central nervous system caused by variation in a single gene, also known as monogenic CNS diseases.

Hitting the ground running, the company launches with a pipeline of 15 adeno-associated virus gene therapy programs, along with options to four others.

Taysha plans human testing this year and expects to file four Initial New Drug applications in 2021. In addition, the biotech is developing an improved treatment delivery platform that uses machine learning, DNA shuffling, and something it calls directed evolution.

The gene therapy startup closed its round of seed financing in early March just as the Dow dropped a couple of thousand points, according to a report in EndpointsNews.

If you had to ask me, was I worried? Absolutely. I think I wouldnt be human if I wasnt, RA Session II, president, CEO, and founder of Taysha, told the healthcare publication.

Session had a lot of certainty to balance out all the uncertainty of launching a new biotech during a pandemic, noted Endpoints Amber Tong.

The experience of a proven management team, including some who previously led the development and commercialization of the first FDA-approved gene therapy for CNS disease, Zolgensma, is part of the equation.

The startup intends to combine the speed, scale, and expertise of its partners UT Southwestern Gene Therapy Program with the experience of the Taysha team.

Session says the launch reunites former investors and executives from AveXis. Tayshas funding round was co-led by PBM Capital, the first institutional investor in AveXis, and Nolan Capital, the investment fund of former AveXis CEO Sean Nolan.

Tayshas Board of Directors played a key role in the formation of the company, it said. The board is comprised of Sean Nolan, Paul Manning of PBM Capital, Claire Aldridge, Ph.D., of UT Southwestern, and RA Session II, President, CEO, and Founder of Taysha.

Nolan, who serves as Chairman of the Board, sees promise in Tayshas approach for patients suffering from many devastating CNS diseases.

This is an exciting time for gene therapy, he said.

The strategic partnership with UT Southwestern could help Taysha to rapidly and efficiently translate novel AAV gene therapies from bench to bedside, the company said.

UTSW is home to some of the brightest minds in gene therapy, Sessions says. By joining forces with UT Southwestern, Taysha can advance its potential cures with both speed and scale.

The collaboration is ultimately creating an engine for new cures, said Claire Aldridge, Ph.D., a Taysha board member and associate vice president of commercialization and business development at UT Southwestern Medical Center.

Together, Taysha and UTSW will merge cutting-edge translational research, hands-on clinical care, and expertiseboth regulatory and commercial, she said.

Aldridge has already witnessed how quickly and efficiently the partners can leverage their collective expertise and resources. That means getting new gene therapies to the patients who so desperately need them, she said.

The UT Southwestern Gene Therapy Program, led by Steven Gray, Ph.D., Director of the Viral Vector Core and Assistant Professor in the Department of Pediatrics, and Berge Minassian, M.D., Division Chief of Child Neurology, has the capacity to support Tayshas wide range of preclinical and clinical development programs, the company said.

Under the partnership, UT Southwestern, which has developed a state-of-the-art viral vector manufacturing facility, will run discovery and preclinical research, as well as lead studies, provide manufacturing, and execute natural history studies.

For its part, Taysha will lead all clinical development, regulatory strategy, commercial manufacturing, and commercialization activities, according to the company.

A joint steering committee of key leadership members from Taysha and UT Southwestern will govern the collaboration.

Sign up to keep your eye on whats new and next in Dallas-Fort Worth, every day.

The free, five-day celebration of all things startup is officially underway.

Browse our curated selection of events to plan your next week and beyond.

Friday morning's Startup Week activities led off with a women's networking breakfast and a Creative Mornings event. Then, sessions have covered equity in real estate, corporate innovation, fundraising, and the future of philanthropy.

Thursday led off with a session delving into blockchain's use in health care and events giving tips in SEO, influencer marketing, and legal issues.RevTech also hosted its signature Tech Trends in Retail event.

Wednesday's panels have tackled disruptive technologies such as blockchain, the corporate-startup relationship, and more.

Read the original here:
Dallas Gene Therapy Startup Launches with $30M Seed Round and a UT Southwestern Partnership - dallasinnovates.com

The team that developed Zolgensma is back for round two. A group of former AveXis executives and investors unveiled a new gene therapy company, and theyre wasting no time. With 15 programs, $30 million in seed funding and an unrivaled partnership with UT Southwestern Medical Center, Taysha Gene Therapies plans to be in the clinic by the end of the year.

Its working on adeno-associated vector (AAV) gene therapies for monogenic diseasesthat is, diseases caused by a defect in a single geneof the central nervous system. Tayshas lead program targets GM2 gangliosidosis, a very rare disorder that progressively destroys nerve cells in the brain and spinal cord, but the companys portfolio includes conditions that are more prevalent.

The company plans to start clinical trials for three more programs by the end of 2021: treatments for Rett syndrome, a neurodevelopmental disorder; SURF1 deficiency, the most frequent cause of Leigh syndrome; and a SLC6A1 genetic epilepsy, which is similar to Dravet syndrome, Taysha CEO and co-founder R. A. Session II told FierceBiotech.

FiercePharma Presents the Virtual eRegulatory Operations Summit

Join industry experts and learn how to build and manage cohesive, compliant, and timely electronic submissions through innovative technologies and seamless clinical documentation processes.

RELATED: Passage Bio pulls off $216M IPO, blowing past original goal

And thats not allTaysha has the option to pick up four more prospects from UT Southwestern.

The reason why we can tackle 15 programs with the option to four additional programs is were able to focus on what we do best and were able to allow our collaborators to focus on what they do best, Session said.

Under their partnership, UT Southwestern is working on discovery and preclinical work all the way through IND-enabling studies. It has its own GMP viral manufacturing site to support that work, as well as clinical development, which Taysha will pick up. The company will also take care of regulatory strategy, commercial manufacturing and commercialization. Its a skill set that Tayshas management team honed at AveXis as itdeveloped the gene therapy that eventually became Novartis spinal muscular atrophy treatment Zolgensma.

We essentially flew the plane and built it at the same time when we were developing AveXis We have people with the experience of being able to develop, manufacture and commercialize a gene therapy program and were marrying that with a best-in-class academic research institution, Session said.

RELATED: FDA lets Novartis off the hook in Zolgensma data manipulation

Dividing the labor creates this engine for innovation that allows the partners to advance many programs in parallel, Session added. With about 50 people in its gene therapy unit, UT Southwestern can develop a capsidthe protein shell of a virusfor treatment delivery or get a candidate into animal models much more quickly than a biotech going it alone, he said. The same goes for late-stage development and commercialization on Tayshas side.

For its initial programs, Taysha is focusing on AAV gene therapies because the team knows they work.

AAV9 is the best way we have to treat monogenic CNS disease. If its not broke, dont fix it, Session said. We know how to effectively dose AAV9; we know its safe, effective and efficient. And we know its scalable. These are problems we had to solve at our previous company.

But, moving forward, Taysha and UT Southwestern are working on new technologies, including an AAV delivery platform that would allow for the redosing of gene therapies as well as an AAV capsid platform aimed at improving target delivery.

The rest is here:
Taysha Gene Therapies hits the ground running with $30M, 15 programs - FierceBiotech

Biotech executive, Julia Berretta, Ph.D., is named Chief Executive Officer

Genespire, a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, announced today the successful close of a 16M Series A financing from Sofinnova Partners, a leading European life sciences venture capital firm based in Paris, London and Milan. The company also announced the appointment of Julia Berretta, Ph.D., as Chief Executive Officer and member of the Board of Directors. Graziano Seghezzi, Managing Partner at Sofinnova Partners, and Lucia Faccio, Ph.D., Partner at Sofinnova Partners, will also join the Board.

Genespire was founded in March 2020 as a spin-off of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), one of the worlds leading cell and gene therapy research institutes spearheaded by gene therapy pioneer Prof. Luigi Naldini. The Company was co-founded by Fondazione Telethon and the San Raffaele Hospital, along with Prof. Naldini and Dr. Alessio Cantore.

The funds will be used to advance Genespires leading-edge platform technologies towards the development of novel gene therapies in two main areas: primary immunodeficiencies and metabolic genetic diseases.

"Our mission has always been to develop breakthrough solutions for genetic diseases," said Prof. Naldini, Genespires co-founder and Director of SR-Tiget. "This financing enables the company to translate our innovative science and early stage programs into clinical development. The appointment of Dr. Berretta as CEO is a major reinforcement of our team."

Sofinnova Partners Dr. Faccio added, "Genespire is an exciting investment with all the key ingredients for success: Outstanding scientists that developed the first ex-vivo gene therapy to market, experienced executives brought in through Sofinnova Partners network and game changing technologies that have the potential to impact the lives of patients with genetic diseases."

"I am thrilled to be joining Genespire and such exceptional scientific founders," said Dr. Berretta. "Genespire was born of decades of experience in the gene therapy field, and is optimally positioned to advance transformative therapies for patients affected by severe inherited diseases."

Dr. Berretta was part of the Executive Committee of Cellectis S.A., a Nasdaq-listed clinical stage gene editing company developing CAR-T cell therapies for cancer, where she led business development as well as strategic planning. She is also an independent Board member of Treefrog Therapeutics, an innovative stem cell company.

About Genespire

Genespire is a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini, Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners. http://www.genespire.com

About Sofinnova Partners

Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, with offices in London and Milan, the firm brings together a team of 40 professionals from all over Europe, the U.S. and Asia. The firm focuses on paradigm-shifting technologies alongside visionary entrepreneurs. Sofinnova Partners invests across the Life Sciences value chain as a lead or cornerstone investor, from very early-stage opportunities to late-stage/public companies. It has backed nearly 500 companies over more than 48 years, creating market leaders around the globe. Today, Sofinnova Partners has over 2 billion under management.

For more information, please visit: http://www.sofinnovapartners.com

About Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit: http://www.telethon.it

Story continues

About Ospedale San Raffaele

Ospedale San Raffaele (OSR) is a clinical-research-university hospital established in 1971 to provide international-level specialised care for the most complex and difficult health conditions. OSR is part of Gruppo San Donato, the leading hospital group in Italy. The hospital is a multi-specialty center with over 60 clinical specialties; it is accredited by the Italian National Health System to provide care to both public and private, national and international patients. Research at OSR focuses on integrating basic, translational and clinical activities to provide the most advanced care to our patients. The institute is recognized as a global authority in molecular medicine and gene therapy, and is at the forefront of research in many other fields. Ospedale San Raffaele is a first-class institute which treats many diseases and stands out for the deep interaction between clinical and scientific area. This makes the transfer of scientific results from the laboratories to the patients bed easier. Its mission is to improve knowledge of diseases, identify new therapies and encourage young scientists and doctor to grow professionally. For more information, please visit: http://www.hsr.it

About the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200429005417/en/

Contacts

Julia BerrettaCEO, Genespire S.r.linfo@genespire.com +39 02 83991300

Bommy LeeHead of Communications, Sofinnova Partnersblee@sofinnovapartners.com +33 (0) 6 47 71 38 11

North AmericaRooneyPartners LLCKate Barrettekbarrette@rooneyco.com +1 212 223 0561

FranceStrategiesImage (S&I)Anne Reinanne.rein@strategiesimage.com +33 6 03 35 92 05

Originally posted here:
Genespire Secures 16 Million Series A Financing from Sofinnova Partners to Advance Transformative Gene Therapies - Yahoo Finance

Hours after Gilead announced that an NIH trial testing their antiviral drug remdesivir in Covid-19 patients had succeeded, NIAID director Anthony Fauci sat on a couch in the Oval Office and gave the world the top-line readout.

The drug induced a 31% improvement on the primary endpoint of time to recovery: 11 days in the drug arm compared to 15 days in the placebo arm, he said, adding that patients taking the drug appeared less likely to die, with an 8% mortality rate in the drug arm compared to 11% in patients given the placebo.

The mortality data were not yet statistically significant, he cautioned but were trending in the right direction. Fauci, surrounded by President Trump, Vice President Mike Pence and several other advisors, said the news was a very optimistic sign in the hunt for treatments to fight the virus.

Although a 31% improvement doesnt seem like a knockout 100%, it is a very important proof of concept, he said. Because what it has proven, is that a drug has blocked this virus.

Fauci said more details would come and that the study would be submitted to a peer-reviewed journal. Trump, who deferred to Fauci in giving the readout, echoed Faucis commentary.

Its a beginning, that means you build on it, Trump said. But its a very positive event.

Shortly after the briefing, the New York Times reported that the FDA was preparing to issue an emergency use authorization for the drugs use in Covid-19. In an email to Endpoints News, the FDA did not confirm or deny the Times report, but a spokesperson said the agency has been engaged in sustained and ongoing discussions with Gilead Sciences regarding making remdesivir available to patients as quickly as possible, as appropriate.

Unlock this story instantly and join 80,400+ biopharma pros reading Endpoints daily and it's free.

SUBSCRIBE SIGN IN

Read more:
In the heart of Texas, the AveXis crew gathers for another go at gene therapy with $30M and a powerhouse academic pact to start - Endpoints News

The Paris-based BrainVectis, a biotech developing gene therapies for Huntingtons disease and Alzheimers disease, has been acquired by the US clinical-stage gene therapy company AskBio.

AskBios gene therapy experience and manufacturing capacity will help to accelerate BrainVectis lead candidate for Huntingtons into phase I testing. In return, AskBio gets to expand its list of target indications.

AskBio recognized the scientific merit of the work at BrainVectis as an opportunity to strengthen our central nervous system clinical pipeline, Robin Fastenau, VP of Communications for AskBio, told me. No financial details about the acquisition were disclosed.

BrainVectis lead candidate focuses on increasing the expression of a protein called CYP46A1. This enzyme is key for turning excess cholesterol into a derivative that can be cleared from the brain into the blood. It is also reduced in Huntingtons and Alzheimers patients, allowing toxic levels of cholesterol to build up in the brain. By increasing the levels of this enzyme, BrainVectis aims to restore normal cholesterol metabolism and improve the clinical outcome.

So far, BrainVectis lead gene therapy candidate has shown proof-of-concept in animal models of Huntingtons. It also received orphan drug designation from the European Commission last year.

According to Nathalie Cartier-Lacave, CEO and Founder of BrainVectis, AskBio offers a strong cell-line manufacturing process. In particular, it can manufacture a range of viral vectors, including BrainVectis vector of choice: the adeno-associated virus.

This powerful adeno-associated virus technology and Askbios expertise in clinical applications will allow us to rapidly go to clinical application in patients, Cartier-Lacave told me.

There are currently no approved treatments able to slow down the progress of Huntingtons and Alzheimers. Combined with aging populations in developed countries, these debilitating diseases are creating a healthcare challenge. Many companies are trying and sometimes failing to develop drugs able to stop the progression of Alzheimers, for example, as such a drug could make a huge impact on society.

According to Cartier-Lacave, Huntingtons disease is the first target for the company going forward. The disease is caused by a mutation in a gene called HTT that is important for the function of nerve cells. Its a very severe disease for which we think the treatment may not only decrease the toxic mutated protein, but also preserve neurons from death, she added.

Gene therapies are becoming ever more popular in the biotech industry for their potential to tackle previously incurable conditions. BrainVectis is one of several companies aiming gene therapies at the brain; another is the French company Lysogene. However, there are major challenges with developing gene therapies for the brain, for example, getting the therapy past the blood-brain barrier and into the brain tissue.

AskBio might be up to the challenge, as it has taken several gene therapies to the clinic. Its proprietary treatment for the neuromuscular indication Pompe disease is currently in phase I/II, and it has licensed its technology to several big pharmaceutical companies. Those currently in clinical development include treatments for Duchenne muscular dystrophy (Pfizer), hemophilia (Takeda), and spinal muscular atrophy (AveXis), which was approved by the FDA last year, and is awaiting marketing approval by the EMA.

Image from Shutterstock

More:
AskBio Takes Over French Gene Therapy Company to Treat Alzheimers and... - Labiotech.eu

CDMO Fujifilm Diosynth Biotechnologies (FDB) says partnering with OXGENE could reduce the lead time of its customers gene therapy projects by up to 25%.

The technology, licensed from UK-based OXGENE for an undisclosed fee, consists of Helper, Rep/Cap and Gene of Interest plasmids, used in combination with a clonal suspension a HEK293 cell line.

The AAV system is expected to reduce the length of the supply chain gene therapy customers, according to contract development and manufacturing organization (CDMO) FDB, with the standard lead-time from the start of process development to the first GMP manufacture potentially reduced by three to six months.

Image: iStock/PashaIgnatov

FDB will manufacture and stock a supply of Helper and Rep /Cap plasmids for clients engaged in process development and GMP manufacture of gene therapy programs. Gene of Interest (GOI) plasmid manufacture will be performed in-house using FDBs existing microbial capabilities and facilities at its site in College Station, Texas with cGMP manufacture planned to commence in Q4 2020.

The site is subject to numerous investments by the CDMO, the latest a $35 million expansion adding cell culture and high throughput manufacturing suites.

OXGENEs AAV system is superior to off-the-shelf plasmid systems for AAV titers, said Andy Topping, chief scientific officer at FDB, adding the agreement gives the CDMO plasmid to drug product capability for AAV systems and allows clients to avoid delays associated with GMP production of plasmids.

UK-based OXGENE was founded as a plasmid catalog business eight years ago. Sophie Lutter, scientific marketing and communications manager, told this publication how the firms technology works at the Phacilitate conference in January.

We start with custom plasmid design and engineering we have plasmid sets optimized for AAV and lentiviral production and then we pair that with our GMP-banked clonal suspension HEK293 cell lines and engineered derivatives, she explained. We take them through to process development, where we can support scales of up to 10 L.

With downstream purification as part of its platform, the company offers a full viral-vector package. This leaves the customer not only with the final viral vector, but also [with] the processes and protocols to take that through to GMP manufacture.

Read more:
Fujifilm licenses AAV tech to speed gene therapies - Bioprocess Insider - BioProcess Insider

Global CNS Gene Therapy Market Analysis

Persistence Market Research, in a recently published market study, offers valuable insights related to the overall dynamics of the CNS Gene Therapy market in the current scenario. Further, the report assesses the future prospects of the CNS Gene Therapy by analyzing the various market elements including the current trends, opportunities, restraints, and market drivers. The COVID-19 analysis section within the report offers timely insights regarding the impact of the global pandemic on the market. The presented study also offers data regarding the business and supply chain continuity strategies that are likely to assist stakeholders in the long-run.

As per the report, the CNS Gene Therapy market is set to grow at a CAGR of ~XX% over the forecast period (2019-2029) and exceed a value of ~US$ XX by the end of 2029. Some of the leading factors that are expected to drive the growth of the market include, focus towards research and development, innovations, and evolving consumer preferences among others.

Request Sample Report @ https://www.persistencemarketresearch.co/samples/27514

Regional Outlook

The report scrutinizes the prospects of the CNS Gene Therapy market in different geographical regions. The scope of innovation, consumer behavior, and regulatory framework of each region is thoroughly analyzed in the presented study.

Distribution-Supply Channel Assessment

The report provides a thorough analysis of the different distribution channels adopted by market players in the global CNS Gene Therapy market along with the market attractiveness analysis of each distribution channel. The impact of the COVID-19 pandemic on the different distribution channels is enclosed in the report.

Product Adoption Analysis

key players and product offerings

Request Report Methodology @ https://www.persistencemarketresearch.co/methodology/27514

The report aims to address the following pressing questions related to the CNS Gene Therapy market:

Key Takeaways from the CNS Gene Therapy Market Report

For any queries get in touch with Industry Expert @ https://www.persistencemarketresearch.co/ask-an-expert/27514

Why Companies Trust PMR?

Read more:
CNS Gene Therapy Market to Slip Due to Delays in Production Amidst Coronavirus Outbreak Cole Reports - Cole of Duty

BOSTON and LONDON, May 01, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company will host a conference call and live webcast on Thursday, May 7, 2020, at 8:00 a.m. ET to report its first quarter 2020 financial results and other business updates.

A live webcast will be available under "News & Events" in the Investors & Media section of the company's website at orchard-tx.com. The conference call can be accessed by dialing (866) 987-6504 (U.S. domestic) or +1 (602) 563-8620 (international) and referring to conference ID 8348144. A replay of the webcast will be archived on the Orchard website following the presentation.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy product candidate pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders, where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

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

Contacts

Investors

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

Excerpt from:
Orchard Therapeutics to Webcast Conference Call of First Quarter 2020 Financial Results - GlobeNewswire

Documenting the Industry Development of Gene Therapy Market concentrating on the industry that holds a massive market share 2020 both concerning volume and value With top countries data, Manufacturers, Suppliers, In-depth research on market dynamics, export research report and forecast to 2029

As per the report, the Gene Therapy Market is anticipated to gain substantial returns while registering a profitable annual growth rate during the predicted time period.The global gene therapy market research report takes a chapter-wise approach in explaining the dynamics and trends in the gene therapy industry.The report also provides the industry growth with CAGR in the forecast to 2029.

A deep analysis of microeconomic and macroeconomic factors affecting the growth of the market are also discussed in this report. The report includes information related to On-going demand and supply forecast. It gives a wide stage offering numerous open doors for different businesses, firms, associations, and start-ups and also contains authenticate estimations to grow universally by contending among themselves and giving better and agreeable administrations to the clients. In-depth future innovations of gene therapy Market with SWOT analysis on the basis Of type, application, region to understand the Strength, Weaknesses, Opportunities, and threats in front of the businesses.

Get a Sample Report for More Insightful Information(Use official eMail ID to Get Higher Priority):https://market.us/report/gene-therapy-market/request-sample/

***[Note: Our Complimentary Sample Report Accommodate a Brief Introduction To The Synopsis, TOC, List of Tables and Figures, Competitive Landscape and Geographic Segmentation, Innovation and Future Developments Based on Research Methodology are also Included]

An Evaluation of the Gene Therapy Market:

The report is a detailed competitive outlook including the Gene Therapy Market updates, future growth, business prospects, forthcoming developments and future investments by forecast to 2029. The region-wise analysis of gene therapy market is done in the report that covers revenue, volume, size, value, and such valuable data. The report mentions a brief overview of the manufacturer base of this industry, which is comprised of companies such as- Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc, Advantagene.

Segmentation Overview:

Product Type Segmentation :

Ex vivo, In Vivo

Application Segmentation :

Cancer, Monogenic, Infectious disease, Cardiovascular disease

To know more about how the report uncovers exhaustive insights |Enquire Here: https://market.us/report/gene-therapy-market/#inquiry

Key Highlights of the Gene Therapy Market:

The fundamental details related to Gene Therapy industry like the product definition, product segmentation, price, a variety of statements, demand and supply statistics are covered in this article.

The comprehensive study of gene therapy market based on development opportunities, growth restraining factors and the probability of investment will anticipate the market growth.

The study of emerging Gene Therapy market segments and the existing market segments will help the readers in preparing the marketing strategies.

The study presents major market drivers that will augment the gene therapy market commercialization landscape.

The study performs a complete analysis of these propellers that will impact the profit matrix of this industry positively.

The study exhibits information about the pivotal challenges restraining market expansion

The market review for the global market is done in context to region, share, and size.

The important tactics of top players in the market.

Other points comprised in the Gene Therapy report are driving factors, limiting factors, new upcoming opportunities, encountered challenges, technological advancements, flourishing segments, and major trends of the market.

Check Table of Contents of This Report @https://market.us/report/gene-therapy-market//#toc

Get in Touch with Us :

Mr. Benni Johnson

Market.us (Powered By Prudour Pvt. Ltd.)

Send Email:[emailprotected]

Address:420 Lexington Avenue, Suite 300 New York City, NY 10170, United States

Tel:+1 718 618 4351

Website:https://market.us

Our Trending Blog:https://foodnbeveragesmarket.com/

Get More News From Other Reputed Sources:

Temporary Pacemaker Market Attractiveness, Competitive Landscape, Traders/Distributors, Key Buyers, Forecasts 2020-2029 | BioSpace

Thymol Market Economic Forecasting By Key Players 2029 | J & K SCIENTIFIC and Meryer (Shanghai) Chemical Technology

The rest is here:
Gene Therapy Market | on(impact of COVID-19) 2020-2029 Analysis on Growth, Future Demand 2020 - Jewish Life News

You can't lump all clinical-stage biotechs together. TakeCara Therapeutics (NASDAQ:CARA) andSangamo Therapeutics (NASDAQ:SGMO), for example. The two biotechs are about as different as night and day. One thing that both Cara and Sangamo have in common, though, are promising pipeline candidates.

Investors have placed a greater value on Sangamo's pipeline based on the company's higher market cap. But which of these biotech stocks is the better pick for long-term investors? Here's how Cara and Sangamo stack up against each other.

Image source: Getty Images.

All of Cara Therapeutics' hopes ride on one drug -- Korsuva. But that one drug has multiple formulations and multiple opportunities.

The most promising of these opportunities is for Korsuva injection in treating chronic kidney disease-associated pruritis (CKD-aP) in patients on hemodialysis. The itching (pruritis) experienced by many patients with CKD is very problematic. So far, there haven't been any approved treatments for CKD-aP.

That could soon change. Last week, Cara and its partner, Vifor Fresenius Medical Care Renal Pharma, announced positive results from a global late-stage study of Korsuva injection in treating CKD-aP in patients on hemodialysis. These results reinforced positive results reported last year from a U.S. study. The two companies plan to file for U.S. regulatory approval for Korsuva injection in the second half of 2020, followed closely by filing for European approval of the drug.

Cara anticipates completing an interim statistical analysis within the next couple of months for a phase 2 study evaluating an oral version of Korsuva in treating pruritis in patients with atopic dermatitis. The biotech is also conducting another phase 2 study of oral Korsuva in treatingpatients with pruritus and hepatic impairment due to primary biliary cholangitis (PBC).

Oral Korsuva should advance to a late-stage study in treating CKD-aP in patients who aren't on hemodialysis later this year. Cara reported results from a phase 2 study in December with the drug meeting its primary endpoint but failing to meet two secondary endpoints.

Korsuva injection could achieve peak annual sales topping $500 million. An oral version of the drug could boost that number considerably. With Cara Therapeutics' market cap currently below $750 million, the stock should have plenty of room to run if Korsuva wins regulatory approvals and reaches its commercial potential.

Unlike Cara Therapeutics, Sangamo isn't hanging its hat on only one candidate. The biotech's pipeline includes five different therapies in clinical testing. However, none of these programs are yet in late-stage testing.

Sangamo specializes in gene editing using a technique known as zinc finger nuclease (ZFN) technology and in developing gene therapies. The company's approach has captured attention from several big drugmakers.

Pfizeris partnering with Sangamo on developing SB-525, a gene therapy targeting hemophilia A. Sanofiteamed up with Sangamo on two gene-edited cell therapies in clinical studies. ST-400 targets treatment of rare blood disease beta-thalassemia, while BIVV-003 targets treatment of sickle cell disease.

Sangamo also has two wholly owned programs in phase 1/2 studies. Gene therapy ST-920 targets Fabry disease. ZFN gene-editing therapy SB-913 targets rare genetic diseasemucopolysaccharidosis type II (MPS II).

In addition to its clinical programs, Sangamo has 11 therapies in preclinical testing. Gilead Sciencesis collaborating with Sangamo on two of these preclinical programs. Pfizer and Japanese drugmaker Takeda have teamed up with the biotech on one preclinical program each.

In February, Sangamo announced a dealwith Biogen to develop gene therapies targeting neurological diseases. Three of Sangamo's preclinical programs are included in this agreement.

Sangamo's market cap stands at a little under $1 billion. While the company has a long way to go before any of its candidates could potentially win approval, it also has multiple shots on goal. Successes for only one or two of its experimental therapies would enable this small biotech to become much larger.

I think that both of these stocks could be big winners over the long run. However, my view is that Cara Therapeutics offers a better risk-reward proposition.

Although it's not a slam dunk that Cara wins FDA and EU approvals for Korvuva injection, I think the odds appear to be pretty good. Assuming those approvals are secured, Cara should be set to begin pulling in some solid revenue by late 2021.

Having said that, it wouldn't surprise me if Sangamo achieves greater success by the end of this decade. I think that investors should keep the biotech on their radar screens.

Visit link:
Better Buy: Cara Therapeutics vs. Sangamo Therapeutics - Motley Fool

The research report on Gene Therapy Market provides comprehensive analysis on market status and development pattern, including types, applications, rising technology and region. Gene Therapy Market report covers the present and past market scenarios, market development patterns, and is likely to proceed with a continuing development over the forecast period. The report covers all information on the global and regional markets including historic and future trends for market demand, size, trading, supply, competitors, and prices as well as global predominant vendors information.

Get Sample copy: https://www.reportspedia.com/report/life-sciences/global-gene-therapy-market-research-report-2014-2026-of-major-types,-applications-and-competitive-vendors-in-top-regions-and-countries/44997 #request_sample

This market research report on the Gene Therapy Market is an all-inclusive study of the business sectors up-to-date outlines, industry enhancement drivers, and manacles. It provides market projections for the coming years. It contains an analysis of late augmentations in innovation, Porters five force model analysis and progressive profiles of hand-picked industry competitors. The report additionally formulates a survey of minor and full-scale factors charging for the new applicants in the market and the ones as of now in the market along with a systematic value chain exploration.

An outline of the manufacturers active within the Gene Therapy Market, consisting of

Sibiono GeneTech,Advantagene,Spark Therapeutics,Shanghai Sunway Biotech Co. LtdBluebird Bio,UniQure NVAvalanche Bio,Celladon,Sangamo,Dimension Therapeutics

The Gene Therapy Market Segmentation by Type:

Viral vectorNon-viral vector

The Gene Therapy Market Segmentation by Application:

Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Market Segment by Regions, regional analysis covers

Get Upto 40% discount on this report Click Here @ https://www.reportspedia.com/discount_inquiry/discount/ 44997

The competitive landscape of the Gene Therapy Market is discussed in the report, including the market share and new orders market share by company. The report profiles some of the leading players in the global market for the purpose of an in-depth study of the challenges faced by the industry as well as the growth opportunities in the market. The report also discusses the strategies implemented by the key companies to maintain their hold on the industry. The business overview and financial overview of each of the companies have been analyzed.

This report provide wide-ranging analysis of the impact of these advancements on the markets future growth, wide-ranging analysis of these extensions on the markets future growth. The research report studies the market in a detailed manner by explaining the key facets of the market that are foreseeable to have a countable stimulus on its developing extrapolations over the forecast period.

Key questions answered in this research report:

Table of Contents:

Global Gene Therapy Market Research Report

Chapter 1 Gene Therapy Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

.CONTINUED FOR TOC

Get Full Table of content @ https://www.reportspedia.com/report/life-sciences/global-gene-therapy-market-research-report-2014-2026-of-major-types,-applications-and-competitive-vendors-in-top-regions-and-countries/44997 #table_of_contents

If you have any special requirements, please let us know and we will offer you the report as you want.

See the article here:
Global Gene Therapy Market Size, Share, Growth, Revenue, Global Industry Analysis and Future Demand And Forecast To 2020-2026 Cole Reports - Cole of...