Category Archives: Gene therapy

Dive Brief:

Only a handful of gene therapies for inherited diseases are approved worldwide. Bluebird, holding two of them, has been one of the leading developers.

So Bluebird's struggles in Europe are notable for the dozens of other biotech companies advancing gene-based treatments for uncommon diseases like cerebral adrenoleukodystrophy or severe beta thalassemia.

The company's decision also reflects the differences in how therapies approved in Europe are paid for, with decisions on reimbursement left up to the governments of individual EU member states. Compared to the U.S., European countries can be more aggressive in demanding lower prices and, as many have single-payer healthcare systems, are better able to negotiate for larger discounts.

While Bluebird set a $1.8 million price for Zynteglo, the company proposed having countries reimburse for treatment over five years. Payments were linked to continued patient benefit.

Germany, however, countered with an initial price of $790,000 per patient, rising to roughly $950,000 if patients were benefiting from treatment several years later, according to an April report from STAT News.

That proved unpalatable to Bluebird, which pulled Zynteglo from Germany before later announcing a broader withdrawal from Europe.

"Bluebird's decision to focus on the U.S. market is driven by the challenges of achieving appropriate value recognition and market access for Zyntegloin Europe, which makes bringing its transformative gene therapies like Zyntegloand Skysona to patients and physicians in Europe untenable for a small innovative company at this time, said Andrew Obenshain, head of severe genetic diseases at Bluebird, in an August statement.

Along with the withdrawal of Skysona from Europe, Bluebird is also pulling back an application for approval in the U.K., according to a Thursday filing with U.S. securities regulators. Withdrawal of Zynteglo from the EU and U.K. will be complete by early 2022.

Bluebird said it will continue long-term follow-up of patients treated in clinical trials in Europe, but will not carry out further study of the treatments there.

While specific numbers aren't available, few patients ever received Zynteglo. The first patient ever treated commercially only received the therapy in February of this year, after manufacturing difficulties had delayed Bluebird's launch of the drug.

Neither Zynteglo or Skysona were expected to be widely used given the small patient populations they were approved to treat. But clinical testing had shown both to be effective therapies, meaning their withdrawal leaves patients in Europe with one less treatment option.

Bluebird recently asked the Food and Drug Administration for approval of Zynteglo in the U.S. and plans to do the same for Skysona by the end of this year.

The company is also in the midst of splitting in two, with its cancer research and programs set to be spun out into a new independent company called 2seventy bio.

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Bluebird, winding down in Europe, withdraws another rare disease gene therapy - BioPharma Dive

27 October 2021

Morrison & Foerster LLP

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On September 30, 2021, the U.S. Food and Drug Administration(FDA)announcedfinal guidance titled "Interpreting Sameness of Gene Therapy Productsunder the Orphan Drug Regulations." The guidance finalizesthe January 2020 draft guidance and provides FDA's currentperspective on certain criteria that help determine sameness ofhuman gene therapy products for orphan drug designation andexclusivity purposes. In the Federal Register notice announcing thefinal guidance, FDA noted that it received comments on the draftguidance that "generally supported the approach described inthe guidance." FDA considered these comments and requests foradditional clarification when finalizing the guidance, "addingclarification and examples, as feasible."

The Orphan Drug Act (ODA) seeks to incentivize the developmentof drugs for rare diseases, defined in the ODA as those affectingfewer than 200,000 people in the United States. The incentivesinclude a 25% tax credit on applicable research and developmentexpenditures, waived user fees when submitting applications to FDA,and the potential for a seven-year period of orphan drugexclusivity for the approved indication. Manufacturers must requestand be granted designation before they are eligible for anyincentives.

When FDA grants marketing approval for a designated orphan drugfor a use or indication within the designation disease orcondition, FDA will determine if the drug is eligible for orphandrug exclusivity. Orphan drug exclusivity is product and indicationspecific, meaning FDA cannot approve a drug containing the sameactive moiety for the same intended use or indication during theperiod of marketing exclusivity.

FDA uses different criteria in determining sameness formacromolecule and small molecule drugs 1.The FDAdefines "same drug" for macromolecule drugs as "adrug that contains the same principal molecular structural features(but not necessarily all of the same structural features) and isintended for the same use as a previously approved drug, exceptthat, if the subsequent drug can be shown to be clinicallysuperior, it will not be considered to be the same drug."2

If a sponsor requests orphan drug designation for a drug that isthe same as a drug already approved for the same use or indication,the sponsor must provide a plausible hypothesis that its drug isclinically superior to the already approved drug. Designation asclinically superior is based on greater efficacy, safety, or amajor contribution to patient care. In order to receive theseven-year market exclusivity, a sponsor must demonstrate that itsproduct is actually clinically superior.

Human gene therapy products may qualify for orphan drugdesignation if they are intended for the treatment of a raredisease or condition and the sponsor sufficiently establishes abasis for expecting the drug to be effective in treating the raredisease. The existing regulations do not describe how the"same drug" definition applies specifically to genetherapy products for orphan drug designation and exclusivity.Mirroring the January 2020 draft guidance, FDA's final guidanceprovides some insight into the current interpretation of how the"sameness" criteria applies to gene therapies.

Assuming that two gene therapy products are intended for thesame use or indication, FDA will consider the "principalmolecular structural features" of the gene therapy productswhen determining "sameness." In the final guidance, FDAstates its intention to generally "consider certain keyfeatures such as transgenes and vectors used in gene therapyproducts to be 'principal molecular structural features'under this regulation." However, FDA does not intend toclassify two gene therapy products as different based solely onminor differences in the transgenes and/or vectors, and willdetermine whether differences are minor differences on acase-by-case basis.

For two gene therapy products intended for the same use orindication, if the products express different transgenes, FDAgenerally intends to consider them to be different drugs becausethey will not contain the same principal molecular structuralfeatures. This would be the case regardless of whether the two genetherapy products at issue have or use the same vector. FDA alsointends to consider vectors from a different viral group to bedifferent for purposes of determining "sameness."Additionally, FDA clarified in the final guidance that it willconsider two gene therapy products from the same viral group to bedifferent "when the differences between the vectors impactfactors such as tropism, immune response avoidance, or potentialinsertional mutagenesis." FDA intends to determine whethervariants of a vector from the same viral group are the same ordifferent on a case-by-case basis.

In a case where two gene therapy products express the sametransgene and have/use the same vector, FDA may also consideradditional features of the final product when determining"sameness," such as regulatory elements (e.g., promotersor enhancers). In these instances, FDA generally intends todetermine "sameness" of gene therapy products on acase-by-case basis.

Footnotes

1. 21 CFR 316.3(b)(14).

2. 21 CFR 316.3(b)(14)(ii).

Because of the generality of this update, the informationprovided herein may not be applicable in all situations and shouldnot be acted upon without specific legal advice based on particularsituations.

Morrison & Foerster LLP. All rights reserved

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On October 13th our friends over at STAT broke the news [sorry, Paywall] about a "warranty" pilot program from Pfizer that offers both patients and health plans (including Medicare Part D plans) the opportunity to receive a refund ...

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Life sciences company Catalent said Tuesday that it is planning to invest $230 million to expand its campus for manufacturing gene therapy treatments near BWI Airport.

With this investment, Catalent plans to add three viral-vector manufacturing suites to its facility in the Anne Arundel County town of Harmans, adding capacity for the company to produce treatments that transfer genetic material into cells to fight rare diseases.

At the facility, Catalent Cell and Gene Therapy offers contract manufacturing for firms that develop and commercialize new treatments in an area of medicine that is coming of age. With FDA clearance, it is producing treatments at commercial scale that are designed to be used in late-stage trials and in the clinic.

An initial building at the site that opened in 2019 has 10 manufacturing suites. In 2020, the company invested $130 million to expand a second building, which has five manufacturing suites. With the latest investment, three more suites with room for multiple bioreactors will be added to that building to maximize capacity for production. Catalent also plans to add storage capacity, including space for just-in-time inventory and low-temperature freezers. In all, the company expects to have 18 suites operational on the 350,000-square-foot campus at the end of 2022.

It will also mean hiring. Currently, Catalent employs 900 people at the site. The company plans to add 700 new employees in scientific, technical and operational roles over the next six years. With the employee growth, it is also adding facilities like an onsite cafeteria and parking garage with the latest investment.

Catalent is committed to continuous improvement and growing with our customers futures in mind. This necessitates that we consistently incorporate our own learnings and the latest developments in [Current Good Manufacturing Practice] manufacturing into our new and existing facilities and operations, to help assure quality and de-risk processes, said Dr. Manja Boerman, president of Catalent Cell & Gene Therapy, in a statement. By applying the expertise we have gained from the last three years of operating our flagship gene therapy commercial facility, we are able to continue to expand our campus with a design layout that is innovative, efficient, and provides ultimate flexibility for our customers.

Somerset, New Jersey-based Catalent provides development and manufacturing services to pharma, biotech and other health companies producing new treatments at facilities around the world. Its presence in the area dates to 2018, when it acquired Baltimore-founded Paragon Bioservices in a deal worth $1.2 billion. The Harmans campus opened right around the time that the acquisition was announced, and Paragon became part of what is now Catalent Cell and Gene Therapy. It continues to have a facility at the University of Maryland BioPark in downtown Baltimore where it works on manufacturing for developmental phases, and added a facility in Rockville in 2019.

Together, the Maryland facilites form a network under Catalent Cell and Gene Therapy, which has grown since the Paragon acquisition in 2019. With innovation continuing to progress rapidly and companies bringing new treatments to market, Catalent is growing its footprint as its customers grow, too.

It shows continued investment in the manufacturing portion of the states biotech footprint. Maryland has a concentration of researchers and scientists at universities and federal labs that make the discoveries that lead to new treatments, and start companies. The state is also increasingly a place where companies are opening facilities to manufacture treatments after they are developed and approved for market.

Along with Catalents investment, this year also brought news of forthcoming production facilities in Frederick from home COVID-19 test maker Ellume, as well as a new site for multiple companies helmed by VaLogic. Kite Pharma also opened a new biomanufacturing facility in Frederick Countys Urbana.

Funding from investors is a well-tracked metric of a communitys growth, but these moves indicate its also worth considering what the growth investments made directly by companies show about the innovation economy.

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Catalent is investing $230M to expand its gene therapy manufacturing campus near BWI - Technical.ly

Angelman syndrome affects roughly 1 in every 20,000 children and it has no approved treatment.

Researchers recently published results of early tests of a gene therapy strategy for Angelman syndrome, a rare neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities.

Angelman syndrome affects roughly 1 in every 20,000 children. In United States there may be more than 15,000 people with the condition and it has no specific treatment.

The genetics of Angelman syndrome are more complicated than classic single-gene disorders such as cystic fibrosis and sickle cell anemia. Humans inherit 1 maternal and 1 paternal copy of most genes. Angelman syndrome arises in children whose maternal UBE3A copy has somehow been mutated or deleted.

For reasons that arent fully clear, mature neurons normally express only the maternal copy of UBE3A; the paternal copy is effectively silenced. Thus, when the maternal copy is lost, the genes function is absent in neurons. Because UBE3A encodes a protein that helps regulate the levels of other important proteins, its absence severely disrupts brain development.

Compounding the complexity, neurons express 2 different variants or isoforms of UBE3A that vary slightly in lengtha short form and a long formin a ratio of about 3 short forms for every 1 long form.

Researchers created a version of UBE3A that, when expressed by neurons, yields short and long forms of the UBE3A protein at a near-normal ratio. The scientists inserted their therapeutic UBE3A gene into a virus-based vector engineered for reliable delivery to neurons. They injected a solution of this vector into hollow spaces, called ventricles, in the brains of newborn Angelman syndrome model mice, which lack the maternal copy of the mouse Ube3a gene. Like humans with Angelman syndrome, these mice fail to express UBE3A protein in their neurons and develop motor deficits, seizures, and other neurological symptoms in the first months of life.

The scientists verified that vector-borne UBE3A became active in neurons throughout the Angelman model mouse brain just days after injection, at a level similar to that of the normal gene. This treatment restored motor skill-learning and the essential mouse behaviors of digging, burrowing, and nest-building. Untreated mice developed the usual Angelman-like impairments. The treated mice also did not become as susceptible as their untreated counterparts to experimentally induced epileptic seizures, and importantly, did not suffer any obvious negative side effects.

This was a proof-of-concept study, but if these early results were translated to the clinic, they would represent big improvements in the quality of life for individuals with Angelman syndrome, said study lead author Matt Judson, PhD, a research associate in the Philpot Lab at the University of North Carolina School of Medicine.

Results were published in the journal JCI Insight.

The researchers plan to further develop their strategy in additional animal models to optimize dose and delivery methods, and ultimately human clinical trials. If such a therapy were available, the researchers expect it might be able to deliver benefits to individuals of any age, but perhaps with varying benefits.

Reference

Judson MC, Shyng C, Simon JM, et al. JCI Insight. Published online October 22, 2021. doi:10.1172/jci.insight.144712.

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Early Results of Gene Therapy for Angelman Syndrome Look Promising - AJMC.com Managed Markets Network

NEW YORK and DURHAM, N.C., Oct. 21, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of Type I (early infantile-onset) and Type II (late infantile-onset and juvenile-onset) GM1 gangliosidosis. The Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

Receiving Fast Track Designation is a critical step in our mission to develop the first potential treatment for all pediatric forms of this rare, terminal disease. This designation joins both the Orphan Drug Designation and Rare Pediatric Disease Designation assigned to AXO-AAV-GM1 by the FDA, which we believe further demonstrates the potential impact of this work on the patient community, said Pavan Cheruvu, M.D., Chief Executive Officer of Sio Gene Therapies. Building on the recently presented data at ESGCT demonstrating normalization of key disease biomarkers in the high-dose cohort with no serious adverse events attributed to AXO-AAV-GM1, this designation will help us accelerate clinical development of this promising investigational therapy for children and families.

The current Phase 1/2 study (NCT03952637) is designed to evaluate the safety, tolerability, and potential efficacy of AXO-AAV-GM1 gene therapy delivered intravenously in children with early infantile, or Type I, and late infantile and juvenile, or Type II, GM1 gangliosidosis. Stage 1 of the study is a dose-escalation study in which the low-dose cohort is evaluating 1.5x1013 vg/kg and the high-dose cohort is evaluating a dose of 4.5x1013 vg/kg. Stage 2 of the trial will then evaluate the efficacy and safety of the optimal dose identified in Stage 1.

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GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene that cause impaired production of the -galactosidase enzyme. Currently, there are no FDA-approved treatment options for GM1 gangliosidosis.

About AXO-AAV-GM1

AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to achieve a broad central and peripheral biodistribution. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to increase -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA and is the only gene therapy in clinical development for all pediatric forms of GM1 gangliosidosis.

In 2018, Sio licensed exclusive worldwide rights from UMass Chan Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

About Sio Gene Therapies

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visit http://www.siogtx.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as "expect," "estimate," "may" and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities, funding requirements and/or runway to meet its upcoming clinical milestones, and timing and outcome of its upcoming clinical and manufacturing milestones are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations; the actual funds and/or runway required for our clinical and product development activities and anticipated upcoming milestones; actual costs related to our clinical and product development activities and our need to access additional capital resources prior to achieving any upcoming milestones; the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the occurrence of adverse safety events during our current and future trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing; the outcome of interactions with regulatory agencies and expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 12, 2021, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Contacts:

Media

Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.com info@siogtx.com

Investors and Analysts

Parag V. MeswaniSio Gene Therapies Inc.Chief Commercial Officerinvestors@siogtx.com

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Sio Gene Therapies Announces Granting of FDA Fast Track Designation for Investigational AXO-AAV-GM1 (AAV9-GLB1) Gene Therapy in Patients with GM1...

SOUTH SAN FRANCISCO, Calif., October 21, 2021--(BUSINESS WIRE)--Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, announced today that members of the management team will participate in a fireside chat at the Jefferies Gene Therapy/Editing Summit on Thursday, Oct. 28, 2021, at 4:30 p.m. ET.

The fireside chat will be webcast live from Graphite Bios website at http://www.graphitebio.com in the Investors section. A replay of the webcast will be archived and available for one month following the event.

About Graphite Bio

Graphite Bio is a clinical-stage, next-generation gene editing company harnessing high efficiency targeted gene integration to develop a new class of therapies to potentially cure a wide range of serious and life-threatening diseases. Graphite Bio is pioneering a precision gene editing approach that could enable a variety of applications to transform human health through its potential to achieve one of medicines most elusive goals: to precisely "find & replace" any gene in the genome. Graphite Bios platform allows it to precisely correct mutations, replace entire disease-causing genes with normal genes or insert new genes into predetermined, safe locations. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, M.D., and Matthew Porteus, M.D., Ph.D.

Learn more about Graphite Bio by visiting http://www.graphitebio.com and following the company on LinkedIn.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211021005108/en/

Contacts

Company: Stephanie YaoVP, Communications and Investor Relations443-739-1423syao@graphitebio.com

Investor Relations: Stephanie AscherStern IR, Inc.212-362-1200ir@graphitebio.com

Media: Christy CurranSam Brown, Inc.615-414-8668media@graphitebio.com

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Graphite Bio Announces Participation in Upcoming Jefferies Gene Therapy/Editing Summit - Yahoo Finance

The construction of a gene therapy facility in Sheffield, UK will advance scientific discoveries into treatment options for patients, says University.

The Gene Therapy Innovation and Manufacturing Centre (GTIMC) is one of three hubs, which are part of an 18 million ($24 million) network funded by the Medical Research Council (MRC), LifeArc, and supported by the Biotechnology and Biological Sciences Research Council (BBSRC).

The other two hubs are located at Kings College London and NHS Blood and Transplant in Bristol and they will work together by sharing skills and resources to advance gene therapy research.

Image: Stock Photo Secrets

The facility will manufacture clinical grade adeno-associated viruses (AAV) and work towards human trials at NHS trusts and Advanced Therapies Treatment Centres within the GTIMC and national network.

This exciting development and partnership will speed up the pull through of new gene therapies into early phase clinical trials and offer hope to patients with neurological and other rare diseases that can be addressed in this way, Pam Shaw, director of the NIHR Sheffield Biomedical Research Centreand co-applicant on the GTIMC application said.

The support given to this initiative will greatly accelerate the translational potential of genetic therapies in the UK and bring benefits in key areas of unmet medical need.

According to the University of Sheffield, the center will be the first of its kind in the north of England and will provide translational and regulatory support alongside training and skills programs to address the shortage of skills in Good Manufacturing Practices (GMP) manufacturing.

Construction of the facility has already begun.

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Sheffield University to build gene therapy facility - BioProcess Insider - BioProcess Insider

Dive Brief:

Despite a recent series of safety concerns, gene therapies continue to draw interest and investment from the world's most powerful drug companies. In just the last month or so, both Pfizer and AbbVieformed collaborations with small gene therapy developers. And Novartis, which already spent nearly $9 billion back in 2018 to acquire AveXis, also announced in late September that it was buying another biotech focused on genetic treatments, Arctos Medical.

Takeda and Poseidaare now adding to this recent spree of deals. For Takeda, which has been slower than some of its peers in establishing a foothold in cell and gene therapy research, the partnership offers another way to catch up.

In particular, Takedaseems enticed by Poseida'sengineering platform, which is designed to very precisely edit genetic material and then be able to deliver large amounts of it into cells. The goal is to develop treatments that don't use viruses as delivery vehicles, an approach first-generation gene therapies rely on, but which has notable limitations. Poseida's technology is part of a growing wave of research into newer methods that can expand gene therapy's reach.

Madhu Natarajan, Takeda'shead of rare disease drug discovery, said in a statement that Poseida's technologies should complement the company's existing collaborations. Over the past year and a half, Takedahas placed bets on multiple other gene therapy biotechswith newer gene therapy technologies, including Ensoma, SelectaBiosciences and Carmine Therapeutics.

Working with Poseida"reinforces Takeda's commitment to investing in next-generation gene therapy approaches that have the potential to deliver functional cures to patients with rare genetic and hematologic diseases," Natarajan said. Among the six programs initially covered in the deal is one for hemophilia A, a disease that has been a prime target for many leading gene therapy developers.

Moving forward,Poseida is in charge of research activities for each program until the candidate selection stage, after which Takeda is responsible for further testing and commercializing the therapy. Currently, all of Poseida's gene therapy programs are in preclinical or earlier stages of development.

Posieda's share price rose nearly 6% following announcement of the Takeda deal,but quickly backtracked to break even. Shares traded at $6.38 apiece late Tuesday morning,reflecting about two-thirds of the value they held at the start of the year.

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Takeda takes aim at a biotech's gene therapy work - BioPharma Dive

Charles River Laboratories is parting ways with two divisionsfor about $115 million, including a Swedish contract developer and manufacturer site snagged in the $875 million acquisition of Cognate Bioservices in March.

The Japanese research models and services unit was sold to Jackson Laboratory for about $63 million in cash, while the gene therapy CDMO site in Sweden was handed over to a private investor group for about $52 million in cash as of Tuesday, according to a statement. The CDMO deal could grow by another $25 million upon potential contingent payments, Charles RiversaidTuesday.

The research and services operations has 260 employees and bolstered Charles River's 2020 revenue by $46 million. Charles River and Jackson Laboratory have been distribution partners on the unit for more than two decades. Following the divestiture, the companies have a technology license agreement for Jackson to produce and distribute the research models in Japan.

RELATED:Charles River jumps into cell and gene therapy manufacturing with $875M Cognate buy

The gene therapy manufacturing plant in Sweden was picked up through Charles River's acquisition of Cognate in the spring. That plant, which has 130 employees,produces plasmid DNA and helped add $10 million to the books last year.

While the plant is no longer a part of Charles River, the CRO still has access to cell and gene therapy CDMO capabilities at operations in the U.S. and the U.K., the company said.

Charles River expects revenue to drop nearly $20 million as a result of the sales.

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Charles River offloads Japanese research model site, Swedish gene therapy CDMO for $115M - FierceBiotech

The new site is five miles from Avids existing operations in Tustin, CA.

Avid has also appointed Drew Brennan, an experienced CDMO business development executive, as general manager of viral vector technologies to lead its expansion into the cell and gene therapy market.

The cell and gene therapy market has seen continued strong growth; and yet the CDMO industry still lacks proven, high-quality CGMP manufacturing expertise and capacity for viral vectors, says Avid. This has prompted its decision to expand into viral vector development and manufacturing.

The company expects the complete new facility build out to take up to 18 months, costing $65m - $75m. The analytical and process development laboratories are expected to come online earlier, and could be operational within six to eight months.

This investment into viral vector services follows Avids recent moves to grow its existing biologics manufacturing capacity through ongoing expansions at its Myford manufacturing facility. The expansion into viral vector services, combined with the ongoing Myford expansion, has the potential to bring the companys total annual revenue generating capacity to more than$350m.

As the new general manager of viral vector technologies, Brennan will be responsible for overseeing all business activities related to Avids expansion into the cell and gene therapy market. He comes to Avid after more than a decade in senior sales and operations positions at Novasep, a provider of equipment and services in the fields of both small molecule and biologics production and purification for the life science and chemical industries.

Nicholas Green, president and chief executive officer ofAvid Bioservices, said: We believe that the addition of viral vector services is a natural extension of our existing traditional biologics offering and provides another avenue for growth by supporting the development and manufacturing of these innovative therapies. We are also thrilled to add Drew to our team to lead our business efforts in the cell and gene therapy market. His impressive track record in this area, combined with the deep relationships that he has developed throughout the industry, will strongly position this new service offering for success.

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Avid Bioservices expands into manufacturing services for cell and gene therapy - BioPharma-Reporter.com