header logo image


Page 20«..10..19202122..3040..»

Archive for the ‘Gene therapy’ Category

Technological Advancements in Manufacturing Boost the Cell and Gene Therapy Market, Says Frost & Sullivan – PRNewswire

Friday, December 4th, 2020

"The need for reproducible, scalable, and economical production of cell and gene therapies is creating a demand for digital bioprocessing technologies," said Nitin Naik, Global Life Sciences Vice President at Frost & Sullivan. "These technologies are critical to realize the true commercial potential of cell and gene therapies in the next two to three years and serve as a conduit to improve market access and control the total cost of therapy."

Naik added: "From a market segment perspective, while the stem cell market is lucrative, the highest growth is expected to be in gene-modified cell therapies, with a pipeline of 269 products,* followed by gene therapies, which account for 182 assets in the pipeline.* Further, although allogeneic stem cell therapies dominate the marketed product catalogs, interest in disease-modifying CAR-T therapies, which are largely autologous, is driving demand for the evolution of manufacturing technologies, models, and capacity expansion investment by CDMOs." (*as of August 2020)

To tap into the growth prospects exposed by the CGT market, companies must focus on:

Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026 is the latest addition to Frost & Sullivan's Healthcare research and analyses available through the Frost & Sullivan Leadership Council, which helps organizations identify a continuous flow of growth opportunities to succeed in an unpredictable future.

About Frost & Sullivan

For six decades, Frost & Sullivan has been world-renowned for its role in helping investors, corporate leaders and governments navigate economic changes and identify disruptive technologies, Mega Trends, new business models and companies to action, resulting in a continuous flow of growth opportunities to drive future success.Contact us: Start the discussion.

Supply Chain Optimization and Decentralized Manufacturing to Expand the Contract Cell and Gene Therapy Manufacturing Market, 20202026

MF98

Contact:Mariana Fernandez Corporate Communications P: +1 210 348 10 12 E: [emailprotected] http://ww2.frost.com

SOURCE Frost & Sullivan

Excerpt from:
Technological Advancements in Manufacturing Boost the Cell and Gene Therapy Market, Says Frost & Sullivan - PRNewswire

Read More...

Bayer establishes new cell and gene therapy platform – PharmaTimes

Friday, December 4th, 2020

Bayer has launched a Cell and Gene Therapy (C>) Platform, aiming to accelerate its presence in this area.

Bayer is planning to strengthen its capabilities for its internal C> activities, while also pursuing external strategic collaborations, technology acquisitions and licensing.

The overall aim is to establish robust platforms with broad application against a range of therapeutic areas.

The G> Platform will combine multiple functions to provide support across the entire value chain for the research and development of cell and gene therapies.

Bayer has made a series of acquisitions over the last few years to bolster its presence in C>, including its recent acquisition of Asklepios Biopharmaceutical which closed on 1 December, and its acquisition of BlueRock Therapeutics in 2019.

These two companies will be the first to be integrated into the new C> Platform, Bayer has announced in a statement.

The German pharma company added that it has also established a C> pipeline consisting of five advanced assets and over 15 preclinical candidates.

This is a defining moment for Bayer. Cell and gene therapies are leading innovation in healthcare, and it is our goal to be at the forefront of this revolution in science, said Stefan Oelrich, member of the board of management, Bayer AG and president of its pharmaceuticals division.

The C> field is growing at an unprecedented pace. With the establishment of Bayers own C> Platform our company will propel its presence in this area. This will complement our existing C> pipeline which already includes five advanced assets with at least three investigational new drugs annually for the next years, he added.

Go here to see the original:
Bayer establishes new cell and gene therapy platform - PharmaTimes

Read More...

A little cowboy saved by groundbreaking gene replacement therapy – Wink News

Friday, December 4th, 2020

SALT LAKE CITY (Ivanhoe Newswire)

Gene replacement therapy: Its a game-changer when it comes to treating life-threatening illnesses. It can replace disease-causing genes with healthy genes, knock out a gene thats not working right, or add a new gene to the body to help fight disease. To date, the FDA has approved four types of gene therapy including one that was given the OK just in time to save one little boys life.

No doubt about it, Cinch Wight is going to be a cowboy just like his dad.

He loves the dog and the horses and the cows, shared Cinchs dad, Alex Wight.

But it has been a wild ride for this young bronco. A mandatory newborn screening test at birth revealed Cinch had spinal muscular atrophy or SMA.

Cinchs mom, Amber Wight recalled, That was the first time Id ever even heard the term and what it was. And so, it was very scary.

A neuromuscular disorder that can paralyze a baby in the first few weeks of life.

My first thought was, hes never going to be able to ride broncs or anything like that, expressed Alex.

But just one day after Cinch was born, the FDA approved a new gene therapy.

We were pretty excited to get a phone call from the department of health, you know, and have this baby here who we can use this treatment on after its approval, explained Russell Butterfield, MD,pediatric neurologist at University of Utah Health and Intermountain Primary Childrens Hospital.

A critical gene in little Cinch was missing. Pediatric neurologist Russell Butterfield used an infusion to deliver a virus carrying a new copy of the gene into Cinchs nerve cells.

Its like a delivery truck to deliver genes to where you want them to go. What that does do, is it stops the disease right where it is, elaborated Dr. Butterfield.

Just a few years ago, most children born with SMA didnt make it to their second birthday. Now?

The hardest is holding a baby in one hand and holding that drug in the other and really feeling the weight of that. And understanding that how different this childs life will be with his new medicine, expressed Dr. Butterfield.

It took a lot of courage for this family to get this far. Thats why Alex wrote a book for his son. A true story about how real cowboys never give up.

I wanted to let him know that no matter how hard it gets, as long as he keeps going, hell be all right, shared Alex.

Doctors dont know if the one-time infusion will last a lifetime or will have to be repeated and there could be a possible risk of inflammation to the liver that doctors will closely monitor. The gene replacement therapy costs 2.1 million dollars. Insurance paid for most of it, but Alex hopes sales from his childrens book will help pay the rest. You can find the book, A Cowboy and His Horse, at https://www.amazon.com/COWBOY-HIS-HORSE-ALEX-WIGHT/dp/B08CWG46ZX.

Contributors to this news report include Cyndy McGrath, Executive Producer; Marsha Lewis, Field Producer; Rusty Reed, Videographer; Roque Correa, Editor.

Read the original:
A little cowboy saved by groundbreaking gene replacement therapy - Wink News

Read More...

Sio Gene Therapies Announces First Patient Dosed in High-Dose Cohort of AXO-AAV-GM1 Clinical Trial in Patients with GM1 Gangliosidosis – BioSpace

Friday, December 4th, 2020

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Dec. 02, 2020 (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 first patient has been dosed in the high-dose cohort of the Phase 1/2 (Stage 1) study for Type I (infantile) and Type II (late infantile and juvenile onset) GM1 gangliosidosis.

AXO-AAV-GM1 is the only gene therapy in the clinic targeting patients with Type I and Type II GM1 gangliosidosis, a devastating and fatal pediatric disease, said Gavin Corcoran, M.D., Chief R&D Officer of Sio. The initiation of the high-dose cohort builds on evidence of extension of survival in naturally-occurring GM1 disease animal models and encouraging clinical data from an expanded access study conducted by a National Human Genome Research Institute (NHGRI) team led by our principal investigator, Dr. Cynthia Tifft at the National Institutes of Healths (NIH) Clinical Center. Our team and academic partners are dedicated to improving the lives of children affected by this devastating disease, and we look forward to reporting topline data from the low-dose cohort before year end.

The Phase 1/2 study (NCT03952637) is designed to evaluate the safety, tolerability, and potential efficacy of AXO-AAV-GM1 delivered intravenously in patients with Type I and Type II GM1 gangliosidosis.

AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation and is the only gene therapy in clinical development for both Type I and Type II GM1 gangliosidosis.

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 approved treatment options for GM1 gangliosidosis. In 2019, Sio reported clinically meaningful improvements from baseline to six-month follow-up for the first GM1 Type II child dosed with low-dose AXO-AAV-GM1 gene therapy under an expanded access protocol.

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 broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

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.

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

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 will, expect, believe, estimate, and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities 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 initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for AXO-Lenti-PD; the scaling up of manufacturing, the 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 November 13, 2020, 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.

Contacts:

Media

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

Investors and Analysts

David NassifSio Gene Therapies Inc.Chief Financial Officer and General Counsel(646) 677-6770investors@siogtx.com

See original here:
Sio Gene Therapies Announces First Patient Dosed in High-Dose Cohort of AXO-AAV-GM1 Clinical Trial in Patients with GM1 Gangliosidosis - BioSpace

Read More...

Ottawa baby a pioneer of gene therapy for rare disease – Windsor Star

Friday, December 4th, 2020

When I first started at CHEO as a neuromuscular expert in 2010, there were absolutely no treatments available, he said, noting that children diagnosed then with SMA were given the necessary nutritional and respiratory supports, and assisted devices such as wheelchairs, to help them live the best life possible.

But research at CHEO, led by Dr. Alex MacKenzie, who discovered a gene NAIP which may contribute to the severity of the disease, helped develop treatment therapies.

One of them, which Aidan was first given when he was about three weeks old, is known as Spinraza. Approved in Canada, it corrects how the faulty gene produces certain proteins, but requires repeated treatments via spinal taps throughout the persons life.

A second treatment, a gene-replacement therapy called Zolgensma, only needs to be administered once, but is not yet approved in Canada and is prohibitively expensive: more than $2 million in the U.S.

Our immediate families volunteered to remortgage their houses and help fundraise, recalls Adam, while the couple planned to sell their house and move in with family.

Fortunately, they didnt have to. Through the manufacturers managed access program, the drug was made available on a compassionate basis to Aidan, who received it two weeks after his first treatment.

Theres no definitive test to say how well Aidans treatment has worked, but, according to Dr. McMillan, many of the newborns who initially received Zolgensma are now five and six years old, and they continue to show robust strong effects to the gene-replacement therapy, and theyve had no symptoms of clinical deterioration and no signs of any wearing-off effects.

For Sully and Deschamps, the proof so far is in seeing their son approach his first birthday with no signs of the disease. We see him as a physically active child who climbs two sets of stairs with ease and cruises around the house like he owns it. He dances to music, he throws and chases balls, and he chases our cats. We are so thankful.

bdeachman@postmedia.com

More:
Ottawa baby a pioneer of gene therapy for rare disease - Windsor Star

Read More...

Tweaking synonymous sites for gene therapy and vaccines – Drug Target Review

Friday, December 4th, 2020

Professor Laurence D Hurst explains why understanding the nucleotide mutations in viruses, including SARS-CoV-2, can have significant implications for vaccine design.

With 61 codons specifying 20 amino acids, some can be encoded by more than one codon and it is often presumed that it does not matter which one a gene uses. When I first studied genetics, some books I read taught that mutations between such alternative codons (eg, GGA->GGC, both giving glycine) were called synonymous mutations, while others referred to them as silent mutations. However, are synonymous mutations really silent meaning they are identical in terms of fitness and function? Although they may specify the same amino acid, does that mean they are all the same?

Figure 1: Intronless GFP transgene expression is higher for variants of GFP with higher GC content at synonymous sites5

Perhaps one of the biggest surprises over recent years has been the discovery that versions of the same gene, differing only at synonymous sites, can not only have different properties, but effects that are not modest.1-5 For example, two versions of green fluorescent protein (GFP) differing only at synonymous sites can have orders of magnitude differences in their expression level.4 We similarly recently discovered that for an intronless transgene to express in human cell lines it needs to be GC rich, which can be achieved by altering the synonymous sites,5 as seen in Figure 1. It is no accident, we suggest, that the well-expressed endogenous intronless genes in humans (such as histones) are all GC rich and that our functional retrogenes tend to be richer in GC content than their parental genes.

The realisation that synonymous sites matter has clear relevance to the design of transgenes or other artificial genes, be these for experiments, gene therapy, protein production (eg, in bacteria) or for vaccine design. In the case of vaccines, we might wish to modulate a viral protein to be effectively expressed in human cells to illicit a strong and robust immune response.6 Conversely to the design of attenuated vaccines, we seek to produce a tuned down version of the virus that can function but is weak.7

The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered. One approach is mass randomisation try many alternatives and see what works.4,8,9 In principle this is fine, but this approach requires many randomisations, which is still technically difficult for long attenuated viruses. An alternative strategy that we have been exploring is to let nature tell us; we can apply tools and ideas from population genetics to better understand what natural selection favours and disfavours and in turn to estimate the strength of selection.

it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus

Estimation of the strength of selection is possible from knowledge of the site frequency spectrum, (ie, how common variants are) from which we can infer the distribution of fitness effects (DFE). If a site is under strong purifying selection, then mutations may occur in the population but these are rapidly eliminated, so variants are always rare. By contrast, if they are selectively neutral, we expect some variants to be quite common. We recently applied this methodology to show that synonymous mutations in human genes that disrupt exonic splice enhancer motifs are often under strong selection and affect many synonymous sites in our genes.10 This has implications for both diagnostics and for transgene design for gene therapy, as we often remove introns in heterologous genes, so freeing up these residues from their role in specifying exons ceases.11

The same DFE methodology cannot easily be applied to viruses, as the methods assume free recombination (ie, we assume one mutation does not impact the fate of others in the same genome). However, other population genetical tools can still be applied. Recently, we examined SARS-CoV-2 and identified the profile of mutations that we see at four-fold degenerate sites.12 From this profile we could estimate what the synonymous site composition would be, assuming that the only forces are mutational biases and neutral evolution (ie, no selection). We observed that in this genome there is a strikingly strong C->U mutation bias and a G->U one. In the raw data this is not so obvious as G and C are quite rare. However, the mutability of the sites per occurrence of the site reveals the underlying patterns.

Figure 2: The rate of mutational flux from one dinucleotide to another in the coding sequence of SARS-CoV-2. The direction of flux is indicated by the indentation of the connecting links: the inner layer represents flux out while the outermost layer represents flux into the node. The frequency of the flux exchange is represented by the width of any given link where it meets the outer axis. Dinucleotide nodes are coloured according to their GC-content. Hence, it is evident that there is high flux away from GC-rich dinucleotides whereas AU-rich dinucleotides are largely conserved.12

With knowledge of the mutational bias we then asked what the equilibrium frequency of the four nucleotides would be using four simultaneous equations. This is the nucleotide content at which for every mutation changing a particular base there is an equal and opposite one creating the same base somewhere else in the genome, ensuring overall unchanged nucleotide content. Given the strong C->U and G->U mutational biases, it is no surprise that the equilibrium content is very U rich (we estimate equilibrium U content should be about 65 percent). However, while the four-fold sites are indeed U rich, they are not that U rich, being closer to 50 percent. A clue as to why the mutation bias is so skewed to generating U comes from analysis of equilibrium UU content: UU residues are predicted to be very common, with CU residues being particularly mutable generating UU (Figure 2) this is expected due to human APOBEC proteins attacking and mutating/editing the virus.13

One probable explanation for this difference between predicted and observed nucleotide content is selection against U content. There may be many U residues appearing in the population, but many are pushed out of the population owing to purification selection, ie, because of the deleterious effects of the mutations. That such selection is happening in the SARS-CoV-2 genome is also clear from the sequence data. We estimate that for every 10 mutations that appear in the sequence databases, another six are lost because of selection prior to genome sequencing. Indeed, UU content is about a quarter of that predicted (Figure 3).

Figure 3: The predicted (under neutral mutational equilibrium) and observed dinucleotide content of SARS-CoV-2. Note the very high predicted levels of UU given the strong mutational flux to UU residues (see Figure 2) and the net underrepresentation in actual sequence.9

This leaves two problems: why is selection operating on SARS-CoV-2 and what can we do with this information? In some cases, we have a good idea as to why: many mutations to U at codon sites generate stop codons. However, we have observed that U destabilises the transcripts and is associated with lower-reported transcript levels;12 a full explanation of the causes of selection on nucleotide content therefore requires manipulation of the sequences.

The second question, what to do with this information, is perhaps more urgent. It has previously been noted that nucleotide content manipulation is a viable means to attenuate viruses.7 Currently there are three groups investigating this route to make a vaccine for SARS-CoV-2: Indian Immunologicals Ltd/Griffith University, Codagenix/Serum Institute of India and Acbadem Labmed Health Services/Mehmet Ali Aydinlar University. In prior attempts, attention has been paid to CpG levels and UpA levels (which we find to be correlated between SARS genes and between different viruses).12 CpGs attract the attention of zinc antiviral protein (ZAP) and UpA attracts an RNAase L. Not surprisingly, some viruses, including SARS-CoV-2, therefore have low levels of both dinucleotide pairs given the levels of the underlying nucleotides.

The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered

In the past, attenuation strategies have focused on modulating synonymous sites to increase CpG and UpA, making the virus more visible to antiviral proteins.14 We in turn suggest a general strategy to utilise this method and to increase U content as well.12 Given the evidence that selection on the virus is to reduce U content, while our antiviral proteins are mutating it to increase U content, it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus. This is an unusual circumstance in which we predict that we should build in more of the already most common synonymous site nucleotides (U in this case) to degrade the virus. More generally, it is assumed that the most used codons are those that tend to increase the fitness of the organism. In the face of such a severe mutation bias, however, this simpler logic no longer holds.

Laurence D Hurst is Professor of Evolutionary Genetics and Director of the Milner Centre for Evolution at the University of Bath. He is currently also the President of the Genetics Society. He completed his D.Phil in Oxford, after which he won a research fellowship and then moved to Cambridge University as a Royal Society Research Fellow. While on the fellowship he assumed his current Chair at Bath University. In 2015 he was elected a Fellow of the Academy of Medical Sciences and a Fellow of the Royal Society. He is a recipient of the Genetics Society Medal and the Scientific Medal of the Zoological Society of London.

Related topicsDisease research, DNA, Gene Therapy, Genetic analysis, Genomics, Protein, Proteogenomics, Proteomics, Research & Development, RNAs, Vaccine

See the article here:
Tweaking synonymous sites for gene therapy and vaccines - Drug Target Review

Read More...

Shape Therapeutics Unveils AAVid Capsid Discovery Platform and Identification of Novel Tissue-Specific AAV Variants, Solving a Fundamental Delivery…

Friday, December 4th, 2020

SEATTLE--(BUSINESS WIRE)--Shape Therapeutics, Inc. (ShapeTX), a next-generation gene therapy company with an industry-leading RNA targeting technology platform, announces today the unveiling of the AAVidTM capsid discovery platform and results from its first AAV5 variant library in a non-human primate selection campaign.

The AAVidTM capsid discovery platform uses non-random mutational fitness to create massive capsid libraries of billions of unique AAV variants for direct-to-NHP in vivo biological selection. By combining cutting-edge DNA synthesis, advanced synthetic biology, next-generation sequence barcoding and machine learning algorithms, ShapeTX generates industry-leading library size and diversity to enable the development of best-in-class human therapeutics.

Wildtype first-generation AAVs are enabling the recent advances in gene therapy, but they have been plagued by toxicities in the clinic due in part to a lack of tissue specificity, resulting in the need for high doses. Our AAVidTM platform solves the issue by creating novel capsid variants with specific tissue-tropism, said Francois Vigneault Ph.D., President and CEO at ShapeTX. Weve stayed quiet for the past three years while developing a superior AAV platform technology and are excited to announce that we have best-in-class AAV variants in hand. Today, we are announcing our novel liver-tropic AAV5 variants stay tuned for more to come.

David J. Huss, Ph.D., Vice President and Head of Research added, The vast structural space for exploration at the AAV capsid/target cell interface necessitates enormous library size and diversity, which until now, has only been probed with capsid library sizes in the tens of thousands to millions. At ShapeTX, we set out to create a superior AAV capsid discovery platform with library sizes in the billions of unique variants, thereby maximizing the opportunity for novel virus/target cell interactions. Dr. Huss presented the details of the platform at the 2nd RNA Editing Summit on Dec. 2, 2020.

About Shape Therapeutics, Inc

Shape Therapeutics is a biotechnology company developing next-generation RNA-targeted therapies to treat the worlds most challenging diseases. The ShapeTX technology platform includes RNAskip, a proprietary suppressor tRNA technology that enables premature stop codon readthrough; RNAfixTM, a precision RNA editing technology using endogenous Adenosine Deaminase Acting on RNA (ADAR); and AAVidTM, a next-generation engineered adeno-associated virus (AAV) platform producing highly specific, tissue-tropic AAVs. The power of the ShapeTX platforms resides in redirecting the cellular machinery already present in our cells, thereby bypassing the risks of immunogenicity and DNA damage seen with other contemporary editing technologies. ShapeTX is committed to data-driven scientific advancement, passionate people and a mission of providing life-long cures to patients. Shape Life!

Excerpt from:
Shape Therapeutics Unveils AAVid Capsid Discovery Platform and Identification of Novel Tissue-Specific AAV Variants, Solving a Fundamental Delivery...

Read More...

Landmark Study in Non-Muscle Invasive Bladder Cancer Evaluating Breakthrough Investigational Gene Therapy Nadofaragene Firadenovec Published in The…

Friday, December 4th, 2020

CAMBRIDGE, Mass.--(BUSINESS WIRE)--FerGene Inc., today announced that The Lancet Oncology published the Phase 3 data from the landmark U.S. clinical trial evaluating an investigational gene therapy, nadofaragene firadenovec (rAd-IFN/Syn3), for the treatment of patients with high-grade, Bacillus Calmette-Gurin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC). In the study, patients received nadofaragene firadenovec, an intravesical therapy given once every three months that is believed to target the patients own bladder wall cells to enhance the bodys natural defenses to fight cancer.1

The Phase 3 study of 157 patients from 33 U.S. sites met its primary endpoint with more than half (53.4%) of CIS Ta/T1 patients (carcinoma in situ; with or without concomitant high-grade Ta or T1 disease) achieving a complete response (CR), all by three months. Of the patients who achieved a CR, 45.5% continued to remain free of high-grade recurrence at 12 months. In the study, nadofaragene firadenovec was administered directly into the patients bladder once every three months by a healthcare professional. The long-term follow-up phase of the four-year study is ongoing, and patients are continuing to be monitored.1

The most common adverse events (AEs) observed in the study that occurred in patients in order of decreasing frequency were: instillation site discharge, fatigue, bladder spasm, micturition urgency, and hematuria. The discontinuation rate due to AEs was 1.9%.1

Once patients with high-grade, non-muscle invasive bladder cancer no longer benefit from their initial BCG treatments, patients often make an informed decision to decline cystectomy a highly complex and life-altering bladder removal surgery or are often medically ineligible for this complex operation, leaving them with limited options, said Colin P. N. Dinney, MD, Chairman, Department of Urology, Division of Surgery, University of Texas MD Anderson Cancer Center and senior author of the publication. These data published in The Lancet Oncology show that nadofaragene firadenovec, a first-of-its-kind therapy, may be an effective treatment option for BCG-unresponsive non-muscle invasive bladder cancer patients.

As a practicing urologist, Im encouraged by these efficacy and safety data which demonstrate the potential for a novel treatment option that fits within the urology practice and gives patients the choice of receiving treatment once every three months which may be a particularly important consideration in this evolving healthcare environment, said Gennady Bratslavsky, MD, President of the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC). Our organization is proud to have played a key role in the mid- and late-stage clinical studies for nadofaragene firadenovec.

Bladder cancer is the sixth most common cancer in the U.S., with NMIBC representing approximately 75% of all new bladder cancer cases.2,3 BCG remains the first-line standard of care for people living with high-grade NMIBC, however up to 50% of high-grade patients who receive initial treatment with BCG will experience recurrence and disease progression within one year becoming BCG-unresponsive.4,5

We believe the important clinical findings highlighted in The Lancet Oncology with this novel gene therapy may fulfill a significant unmet need for patients and have the potential to be practice-changing, said Vijay Kasturi, MD, Vice President of Medical Affairs at FerGene Inc. We are extremely grateful to the investigators, the patients who participated in the study, FKD Therapies and the SUO-CTC for the important findings highlighted in this publication.

A Biologics License Application (BLA) for nadofaragene firadenovec is currently with the U.S. Food and Drug Administration (FDA).

About Nadofaragene Firadenovec

Nadofaragene firadenovec (rAd-IFN/Syn3) is an investigational gene therapy being developed as a treatment for patients with high-grade, BCG-unresponsive NMIBC. It is a non-replicating adenovirus vector-based gene therapy containing the gene interferon alfa-2b, administered by catheter into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene to do its work. The internal gene/DNA machinery of the cells picks up the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby turns the patients own bladder wall cells into interferon microfactories, enhancing the bodys natural defenses against the cancer. Nadofaragene firadenovec has been studied in a clinical trial program that includes 221 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.4 It is estimated that there will be approximately 81,000 new cases of bladder cancer in the U.S. in 20206; 75% of these cases present as NMIBC.3 In patients with high-grade NMIBC, intravesical BCG is the recommended treatment; however, up to 50% of high-grade patients will experience disease recurrence within one year.4,5 The outcome for BCG-unresponsive patients is poor, with chemotherapy and radiation or total cystectomy (complete removal of the bladder) often being the recommended next treatment options.7

About the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC)

Created, owned and operated by its members, the SUO-CTC is a clinical research investigator network of over 500 members from more than 200 clinical sites in the U.S. and Canada. This national alliance of leading academic and community based uro-oncologists is committed to furthering urology research. The SUO-CTC is a registered 501c3 not-for-profit corporation and has a cooperative relationship with the Society of Urologic Oncology (SUO). The SUO-CTC pursues clinical trials, in concert with sponsors, to investigate therapeutic interventions which address urological cancers including, but not restricted to: Bladder Cancer, Prostate Cancer and Renal Cancer. Together with industry, the SUO-CTC offers enhanced research options for ultimately delivering better quality of life to our patients.

About FerGene Inc.

FerGene Inc. is a gene therapy company committed to revolutionizing the treatment of bladder cancer through its innovative science and unparalleled commitment to patient care. Founded in 2019, as a result of a collaboration between Blackstone Life Sciences and Ferring Pharmaceuticals, FerGene Inc. is singularly focused on evolving the bladder cancer treatment landscape through its novel approach to gene therapy. A trusted partner to medical and advocacy communities, FerGene Inc. is dedicated to bringing new hope to a patient population which has seen little improvement in their standard of care over the past twenty years. For more information, please visit http://www.fergene.com or engage with us on Twitter at @FerGeneBio or on LinkedIn.

2020 FerGene Inc. 20/09 US- ADST-2000114

1 Boorjian, S., Alemozaffar, M., Konety, B., Shore, N., Gomella, L., Kamat, A. et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet Oncol. 2020;2045(20)30540-4. doi:10.1016/S1470.2 National Cancer Institute SEER Program. Cancer Stat Facts: Common Cancer Sites. https://seer.cancer.gov/statfacts/html/common.html. Accessed March 5, 2020.3 Burger M, Catto JW, Dalbagni G, et al. Epidemiology and risk factors of urothelial bladder cancer. Eur Urol. 2013;63(2):234-41. doi:10.1016/j.eururo.2012.07.033.4 Sanli, O., Dobruch, J., Knowles, M. et al. Bladder cancer. Nat Rev Dis Primers. 3,17022 (2017) doi:10.1038/nrdp.2017.22.5 Kamat AM, Li R, ODonnell MA, et al. Predicting response to intravesical Bacillus Calmette-Gurin immunotherapy: Are we there yet? A systematic review. Eur Urol. 2018;73(5):738-748. doi:10.1016/j.eururo.2017.10.0036 American Cancer Society. Key Statistics for Bladder Cancer. https://www.cancer.org/cancer/bladder-cancer/about/key-statistics.html. Updated 2020. Accessed March 5, 2020.7 Marqueen K, et al. Identifying high surgical risk in muscle-invasive bladder cancer (MIBC) patients undergoing radical cystectomy (RC). JNCI Cancer Spectr. 2018 Oct; 2(4): pky075.

See the article here:
Landmark Study in Non-Muscle Invasive Bladder Cancer Evaluating Breakthrough Investigational Gene Therapy Nadofaragene Firadenovec Published in The...

Read More...

Genprex to Present at the Benzinga Global Small Cap Conference on December 8 – Business Wire

Friday, December 4th, 2020

AUSTIN, Texas--(BUSINESS WIRE)--Genprex, Inc. (Genprex or the Company) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that the Company will present at the Benzinga Global Small Cap Conference taking place virtually December 8-9. Genprexs President and Chief Executive Officer, Rodney Varner, will virtually deliver a company overview, including recent progress made on its upcoming Acclaim-1 clinical trial, to participating investors.

Event: Benzinga Global Small Cap ConferencePresentation Date: Tuesday, December 8Presentation Time: 2:30 p.m. ESTRegistration Link: https://bit.ly/2Jf5TcN

The Benzinga Global Small Cap Conference will provide investors with direct access to small caps for insights on their leadership, business fundamentals and plans for expansion. Attending investors will also have the opportunity to meet with management of presenting companies on a one-on-one basis.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Companys lead product candidate, REQORSA (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). REQORSA has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for REQORSA for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso) for patients with EFGR mutations whose tumors progressed after treatment with osimertinib alone.

For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding the Companys future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of REQORSA immunogene therapy drug, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including GPX-002, our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships, including those relating to manufacturing of our product candidates; the timing and success at all of obtaining FDA approval of REQORSA and our other potential product candidates including whether we receive or benefit from fast track or similar regulatory designations; costs associated with developing our product candidates, whether we identify and succeed in acquiring other technologies and whether patents will ever be issued under patent applications that are the subject of our license agreements or otherwise. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Here is the original post:
Genprex to Present at the Benzinga Global Small Cap Conference on December 8 - Business Wire

Read More...

News briefing: Merck’s Roger Perlmutter buys his first solid tumor TriNKET from Dragonfly; ViGeneron to expand production of eye gene therapy -…

Friday, December 4th, 2020

A little more than 2 years after Mercks Roger Perlmutter signed off on a deal that would pay Dragonfly up to $695 million for each drug program it picked off for solid tumors, the pharma giant is stepping up with their first opt-in.

We dont know exactly how much this deal costs Merck in the upfront, or which immunotherapy theyre getting, but its a major step forward for Bill Haneys Waltham, MA-based biotech, which built its TriNKET technology platform with the help of Tyler Jacks, an MIT professor, HHMI investigator and director of the David H. Koch Institute for Integrative Cancer Research as well as Berkeleys David Raulet, whose background as an expert in NK cells and tumor immunology helped spotlight some of the big ideas Dragonfly is pursuing.

This latest pact marks the latest in a flurry of BD deals for the pharma giant, just one last step before Perlmutter hangs it up as head of R&D and passes the reins to Dean Li. John Carroll

A little over a year since announcing its Series A, gene therapy biotech ViGeneron has entered into a new deal.

The German company is partnering with WuXi Advanced Therapies, a contract testing, development and manufacturing organization under WuXi AppTec based out of Philadelphia, to ramp up production of ViGenerons lead candidate VG901 for ophthalmic disorders. VG901s current target is for retinitis pigmentosa, also known as rod cone dystrophy, a degenerative eye disease that causes severe vision impairment as early as childhood.

Manufacturing for the candidate should begin before the year is out, ViGeneron said in a statement. The company added that the program came out of its proprietary vgAAV vector platform, which allows for better transduction of retinal cells as well as a less invasive treatment administration.

There is currently no cure for the disease, though there are some methods that can help manage symptoms like the use of low vision aids and portable lighting. Patients often experience worsening peripheral vision and trouble seeing at night. Max Gelman

Lentiviral vector manufacturer iVexSol has raised $13 million in Series A financing, bringing the total haul to $15.2 million from Casdin Capital and BioLife Solutions and a third undisclosed lead investor.

Founded on the promise to change the way this critical raw material is made using next-generation manufacturing tech, the company said it can produce LVVs at significantly greater quantities than traditional transient transfection processes. The companys name is short for intelligent vector solutions.

Much like adeno-associated viral vectors, or AAV, these delivery vehicles are crucial for cell and gene therapies such as CAR-T, iVexSol added, and their shortage means developers often have to wait 12 to 18 months for production slots.

Details on exactly how it plans to revolutionize the space are scant, but CEO Rod Rietze and CSO Mike Greene both bring technical experience from shops like Novartis and Pfizer.

Its new funding will help establish a facility in Lexington, MA housing stable LVV producer cell line master banks and commercial-grade LVV. Amber Tong

Read the rest here:
News briefing: Merck's Roger Perlmutter buys his first solid tumor TriNKET from Dragonfly; ViGeneron to expand production of eye gene therapy -...

Read More...

CDMOs Are the Unsung Heroes of the Gene and Cell Therapy… – Labiotech.eu

Tuesday, November 24th, 2020

Cell and gene therapy is booming amid the ongoing pandemic, and this is driving up demand for synthetic DNA products, often produced and delivered by CDMOs.

Karen Fallen started her career at Celltech Biologics in the UK, which was acquired in 1996 by Swiss giant Lonza, a world leader in the pharmaceutical manufacturing space. She stayed there for 31 years before moving on to the role of CEO at Touchlight DNA Services in October.

The market in DNA and nucleic acid medicine is just exploding. Cell and gene therapies have the biggest growth of all the biologics out there and DNA is the essential starting material, Fallen told me.

For several years, UK-based company Touchlight Genetics has been producing a form of synthetic DNA, called doggyboneDNA, that is designed to simplify the manufacturing of DNA vaccines and gene therapy.

To meet the ever-increasing demand for its products, Touchlight Genetics created a contract development and manufacturing organization (CDMO) arm of the business called Touchlight DNA Services, which Fallen now leads.

Traditionally, the DNA necessary for these treatments has been produced by bacteria. Touchlight is developing a method to create this DNA enzymatically, which removes the need to include certain genes required by the bacteria and thus makes the final product simpler and safer.

Its really quick to actually produce the DNA, much faster than the bacterial methodology of doing it, Fallen noted. Its also very scalable.

The increasing need for fast, cheap and accurate DNA synthesis in recent years has led a number of biotechs in Europe to try and meet the demand, such as DNAScript, Evonetix, Nuclera, and Camena Bioscience, among others.

The demand has further increased with the Covid-19 pandemic, since many of the companies developing vaccines or treatments for Covid-19 require DNA as a starting material. So do many tests to diagnose Covid-19 infections.

Theres a lot of potential RNA and [DNA] vaccines out there, Fallen said. The demand for DNA was already outstripping supply, and that was just with cell and gene therapy demands. The Covid-19 impact has just accentuated that.

While CDMOs are crucial to the drug development industry, they often dont receive the same level of public and media attention as biotechs and big pharma. But Fallen thinks their importance is only increasing as cutting-edge treatments become commonplace, as many biotechs working on advanced therapies do not have the capacity for advanced manufacturing.

I think that the CDMO industry over the last few years has really come into its own At least 70% of the innovator molecules out there reside with biotechs that dont always have the capability to develop or manufacture them, Fallen told me.

The supply chain is imperative. Its all very well developing a molecule, but if you cant manufacture it or deliver it, then youre going to go nowhere.

Starting a new job as a CEO is a challenge for most people, but particularly so at the moment with the pandemic hanging over everyone and most people working completely remotely.

I met all the people by Zoom, which isnt often the easiest thing to do. But that gives you an idea of the culture of the company.

Thanks to her experience running a business unit at Lonza, Fallen has learned a lot about how to be a good leader. Ive always found that people follow you because they want to, not because youre in a position of power You need to listen to people, see what motivates them, and then you can get people on board.

Youve also got to make tough decisions quickly as well. You cant have an endless amount of data to make decisions on, youve just got to go with it and have the confidence to do that.

Fallen is a strong believer in supporting others. I have become, surprisingly, even to myself, a role model. I spent a fair amount of time talking with some of the more junior people [at Lonza] mentoring them and giving them my perspective on the world. I have shared what Ive learned as Ive been going along and given some advice on how to handle certain situations.

Although she has been lucky in that respect, Fallen recognizes that diversity can be a problem in CDMOs, biotechs, and pharma companies, particularly at higher management levels.

Youve got to try and get rid of unconscious biases. I know a lot of companies are putting people into positions just to get the numbers up, but Im not convinced thats the right way of doing it. I think you just have to have an open mind and give people the opportunity. From my experience, that is mostly successful.

Youve got to take a risk with people sometimes. Ive been in that position of people taking risks with me. Youve got to then support them, youve got to develop them in the right way, dont just talk them into something. But if you do that, I think you get what you want out of people.

Cover illustration by Elena Resko, picture provided by Touchlight DNA Services.

See more here:
CDMOs Are the Unsung Heroes of the Gene and Cell Therapy... - Labiotech.eu

Read More...

Health Beat: Boy saved by groundbreaking gene replacement therapy – WFMZ Allentown

Tuesday, November 24th, 2020

SALT LAKE CITY - No doubt about it, Cinch Wight is going to be a cowboy just like his dad.

"He loves the dog and the horses and the cows," shared Cinch's dad, Alex Wight.

But it has been a wild ride for this young bronco. A mandatory newborn screening test at birth revealed Cinch had spinal muscular atrophy, or SMA.

"That was the first time I'd ever even heard the term and what it was, and so it was very scary," recalledCinch's mom, Amber Wight.

A neuromuscular disorder can paralyze a baby in the first few weeks of life.

"My first thought was he's never going to be able to ride broncs or anything like that," expressed Alex.

But just one day after Cinch was born, the FDA approved a new gene therapy.

"We were pretty excited to get a phone call from the department of health, you know, and have this baby here who we can use this treatment on after its approval," explained Dr. Russell Butterfield, a pediatric neurologist at University of Utah Health and Intermountain Primary Children's Hospital.

A critical gene in little Cinch was missing. Butterfield used an infusion to deliver a virus carrying a new copy of the gene into Cinch's nerve cells.

"It's like a delivery truck to deliver genes to where you want them to go. What that does do is it stops the disease right where it is," Butterfield explained.

Just a few years ago, most children born with SMA didn't make it to their second birthday. Now?

"The hardest is holding a baby in one hand and holding that drug in the other and really feeling the weight of that, and understanding that how different this child's life will be with this new medicine," Butterfield said.

It took a lot of courage for this family to get this far. That's why Alex wrote a book for his son, a true story about how real cowboys never give up.

"I wanted to let him know that no matter how hard it gets, as long as he keeps going, he'll be all right," shared Alex.

Doctors don't know if the one-time infusion will last a lifetime or will have to be repeated, and there could be a possible risk of inflammation to the liver that doctors will closely monitor.

The gene replacement therapy costs $2.1 million. Insurance paid for most of it, but Alex hopes sales from his children's book will help pay the rest. You can find the book, "A Cowboy and His Horse," on Amazon.

See the original post here:
Health Beat: Boy saved by groundbreaking gene replacement therapy - WFMZ Allentown

Read More...

Singapore: Public consultation on Proposed Regulations of Cell, Tissue and Gene Therapy Products – Lexology

Tuesday, November 24th, 2020

In brief

From 6 to 27 November 2020, the Health Sciences Authority (HSA) will be carrying out a public consultation seeking feedback on the proposed Health Products Act ("Amendment of First Schedule") Order 2020 ("HPA Amendment Order"), and the proposed Health Products (Cell Tissue and Gene Therapy Products) Regulations 2020 ("CTGTP Regulations") (collectively, the "Proposed Regulations"), which seek to regulate cell, tissue and gene therapy products (CTGTP). The Proposed Regulations seek to flesh out (i) a definition of CTGTP, (ii) a risk-based regulatory approach for CTGTPs, and (iii) introduce requirements unique to CTGTP.

Comments

CTGTP are a notably emerging and cutting edge field of medicine, providing great potential for the development of new treatment opportunities. Apart from being able to engineer the growth of healthy and functional tissues to reconstruct, regenerate or repairdamaged tissues or organs, CTGTP may also be used in therapies to treat or cure disease through the modification of individual genes.

The HSA has noted that the CTGTP regulations are reportedly intended to facilitate patient access to therapies that meet appropriate safety, efficacy and quality standards, and to provide a fit-for-purpose regulatory framework to support product development and facilitate product commercialisation. Given the novelty of this legislation, it may be beneficial for businesses to participate in the public consultation given their expertise and experience in this area of medicine. This could also assist toensure that any eventual laws appropriately account for the unique nature of CTGTP while not being excessively onerous on suppliers, importers, and manufacturers.

In more detail

From 6 to 27 November 2020, the Health Sciences Authority (HSA) will be carrying out a public consultation seeking feedback on the proposed Health Products Act ("Amendment of First Schedule") Order 2020 ("HPA Amendment Order"), and the proposed Health Products (Cell Tissue and Gene Therapy Products) Regulations 2020 ("CTGTP Regulations") (collectively, the "Proposed Regulations"), which seek to regulate cell, tissue and gene therapy products (CTGTP) as a new category of health products. The Proposed Regulations are intended to provide (i) a definition of CTGTP, (ii) a risk-based regulatory approach for CTGTPs, and (iii) introduce requirements unique to CTGTP.

Part (i) will be addressed via the HPA Amendment Order, which seeks to insert the following definition of CTGTP under the Health Products Act: "a substance consisting of human cells or tissues that are either autologous obtained from the same individual) or allogeneic (obtained from another individual), viable animal cells or tissues, or recombinant nucleic acids (i.e. modified DNA or RNA as carriers of a therapeutic gene); that is intended for diagnosis, treatment or prevention of any human disease or medical condition".

The CTGTP Regulations in turn, addressparts (ii) and (iii). The proposed regulatory framework is generally split into the following areas:

The draft HPA Amendment Order and CTGTP Regulations can be foundhereandhere. Feedback on the regulations may be providedhere.

See the original post:
Singapore: Public consultation on Proposed Regulations of Cell, Tissue and Gene Therapy Products - Lexology

Read More...

Genenta to Appoint Stephen Squinto, Experienced Biotech Executive and Investor, as Chairman – BioSpace

Tuesday, November 24th, 2020

MILAN, Italy and NEW YORK, Nov. 23, 2020 (GLOBE NEWSWIRE) -- Genenta Science, a clinical-stage biotechnology company pioneering the development of a hematopoietic stem progenitor cell gene therapy for cancer (Temferon), announced that highly experienced biotech executive and investor Stephen Squinto, PhD joint Genenta and will be appointed as Chairman of its Board of Directors.

The appointment will be effective with the approval of the Genenta Shareholders Meeting. Pierluigi Paracchi, currently Chairman and Chief Executive Officer of Genenta, will continue to serve as CEO.

Dr Squinto is currently Executive Partner of the healthcare investment company OrbiMed Advisors and has more than 25 years experience in the biotech industry, including as Chief Executive Officer of the gene therapy company Passage Bio. He was a co-founder of Alexion Pharmaceuticals, where he served as Chief Global Operations Officer and Global Head of Research, and previously held several senior leadership positions at Regeneron Pharmaceuticals.

Dr Squinto currently serves on the Board of Directors of a several biotech and healthcare companies and has received numerous honors and awards from academic and professional organizations for his scientific work. He received his PhD in Biochemistry and Biophysics from Loyola University of Chicago.

Pierluigi Paracchi, CEO of Genenta, said: I am delighted to welcome Steve Squinto to Genenta as our new Chairman. Steve will provide valuable input and guidance to the development on Genenta, based on his outstanding and extensive time in biotech industry. In particular, his time as CEO of the gene therapy company Passage Bio provides a clear parallel to Genenta, as we continue to develop the stem cell gene therapy Temferon.

Stephen Squinto said: It as honor to be invited to be Chairman of Genenta, a truly exciting company which has the potential to revolutionize the way we treat cancer through its novel immuno-gene therapy Temferon. I am looking forward to working with the outstanding team already in place to progress this treatment, which has potential against a broad range of tumors both as first line and as combination therapy, further through clinical trials and towards market.

About Genenta Science

Genenta (www.genenta.com) is a clinical-stage biotechnology company pioneering the development of a proprietary hematopoietic stem cell gene therapy for cancer. Temferon is based on ex-vivo gene transfer into autologous hematopoietic stem/progenitor cells (HSPCs) to deliver immunomodulatory molecules directly via tumor-infiltrating monocytes/macrophages (Tie2 Expressing Monocytes - TEMs). Temferon, which is under investigation in a Phase I/II clinical trial in newly diagnosed Glioblastoma Multiforme patients, is not restricted to pre-selected tumor antigens nor type and may reach solid tumors, one of the main unresolved challenge in immuno oncology. Based in Milan, Italy, and New York, USA, Genenta has raised 33.6 million (~$40 million ) in three separate rounds of financing.

Continue reading here:
Genenta to Appoint Stephen Squinto, Experienced Biotech Executive and Investor, as Chairman - BioSpace

Read More...

Orchard Therapeutics to Present at Piper Sandler 32nd Annual Virtual Healthcare Conference – GlobeNewswire

Tuesday, November 24th, 2020

BOSTON and LONDON, Nov. 24, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that its chief executive officer, Bobby Gaspar, M.D., Ph.D. will participate in a pre-recorded fireside chat as part of the Piper Sandler 32nd Annual Virtual Healthcare Conference. Management will also be available for one-on-one meetings on Tuesday, December 1, 2020.

The fireside chat is available for on-demand viewing under "Events" in the Investors & Media section of the company's website at http://www.orchard-tx.com and will remain available for approximately 90 days.

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. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, 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 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.

Availability of Other Information About Orchard

Investors 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 (TwitterandLinkedIn), 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

Investors

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

Read more here:
Orchard Therapeutics to Present at Piper Sandler 32nd Annual Virtual Healthcare Conference - GlobeNewswire

Read More...

Reactions from the 2020 SYNGAP1 Scientific Conference – Spectrum

Tuesday, November 24th, 2020

Bridging the gap: SYNGAP1 protein is located mostly at synapses, the junctions between neurons (green).

Spectrum is covering the 2020 International SYNGAP1 Scientific Conference, which took place virtually because of the coronavirus pandemic. Here were highlighting researchers reactions to noteworthy presentations.

Drug test: A new assay allows researchers to test thousands of candidate drugs for their ability to boost expression of the autism gene SYNGAP1. The tool may help researchers identify and screen potential treatments for people with mutations that silence the gene. Gavin Rumbaugh, professor of neuroscience at Scripps Research in Jupiter, Florida, presented the unpublished results on 18 November.

The assay uses neurons from mice with one intact and one mutated copy of SYNGAP1. The researchers genetically engineer the mice so that SYNGAP1 protein made from the intact copy is tagged with luciferase the enzyme that gives fireflies their glow.

They then grow these neurons in tiny wells and add a different candidate drug to each well. The amount of SYNGAP1 protein in the dish gives a proportionate amount of light in your well, Rumbaugh says.

Rumbaugh and his team plan to use the platform to run through more than 100,000 different experimental compounds in 2021, he says.

Thats going to be really exciting for drug discovery efforts for SYNGAP1. I think thats going to be a game changer, says Karun Singh, senior scientist at the University Health Network in Toronto, Canada, who was not involved in the work.

It will be very exciting to see if they are able to uncover any useful hits with their novel approach, says Helen Bateup, associate professor of neurobiology at the University of California, Berkeley, who was not involved in the work.

Treatment across ages: A leading theory of autism is that the condition is characterized by a signaling imbalance: too much excitation or too little inhibition in the brain. One of the key players in creating this imbalance is thought to be inhibitory interneurons, which employ the neurotransmitter gamma-aminobutyric acid (GABA). And mutations to SYNGAP1 may disrupt GABAs function, said James Clement, assistant professor of neuroscience at the Jawaharlal Nehru Centre for Advanced Scientific Research in Bangalore, India, in a presentation on 18 November.

GABA is excitatory early on in brain development and inhibitory later on a switch that seems to be impaired in mice with SYNGAP1 mutations, he says. He and his team have tested a new compound that restores the GABA switch in mice and eases almost all SYNGAP1-related traits including seizures, learning issues and motor impairment in the mice. It works in newborn and adolescent mice. Due to a pending patent application, Clement and his lab are not revealing the compounds name.

I think its important to test efficacy at multiple ages, as they have done, to understand which phenotypes or problems can be improved with early treatment and which might still be responsive to treatment even if its administered later in life, says Bateup, who was not involved in the work. The idea that GABA may remain depolarizing for longer in SYNGAP1 mutant mice is quite interesting.

Clements lab was the only other lab that was presenting at this meeting that presented data from a very early age, says Shilpa Kadam, associate professor of neurology at the Kennedy Krieger Institute in Baltimore, Maryland, who was not involved in the work.

Motor coordination: For mice, the loss of SYNGAP1 function in the striatum impairs their goal-directed learning and seems to lead to inflexible behavior, Bateup said in a presentation on 18 November.

Helen Bateups work looking at striatal function as it relates to motor coordination and motor learning is also pretty exciting and may shed light not only on the motor-coordination difficulties but also the repetitive or habitual motor behaviors, says Constance Smith-Hicks, child neurologist and research scientist at the Kennedy Krieger Institute, who was not involved in the work.

Bateups presentation also demonstrated that SYNGAP1 deletion seems to affect neurons differently depending on which type of dopamine receptor they express.

We know SYNGAP1 is at most excitatory synapses, so why shes seeing some functional effects in one type of cell and not the other, I find that interesting, says Richard Huganir, professor of neuroscience and psychological and brain sciences at Johns Hopkins University in Baltimore, Maryland, who was not involved in the work.

Its exciting to be able to kind of pinpoint which pathway might be involved and get a better understanding of the circuits that are disrupted, says Singh, who was not involved in the work.

Protein levels: People with a nonfunctional copy of SYNGAP1 have about half the typical amount of SYNGAP1 protein. Increasing the activity of the intact copy of the gene could help restore typical functioning, Huganir said in a presentation on 18 November.

He and his team tested this idea on two unique mouse models in unpublished work. Instead ofhaving one intact and one missing copyof the SYNGAP1 gene, as is typical for SYNGAP1 mouse models, each mouse model carriesone intact copy of the gene and one with a mutation seen in people. Both mice produce about half the typical amount of SYNGAP1 protein and show the same behaviors as the classical knockout mouse, despite having different types of mutations.

These new mouse models are crucial because they can directly correlate to what is happening in the humans, says Clement, who was not involved in the work.

Huganir and his team are testing different types of gene therapies to increase SYNGAP1 protein up to the typical levels, and have found that there are two SYNGAP1 protein isoforms, or slight variations of the protein.

One of the isoforms can restore synaptic plasticity in the animal model for SYNGAP1, so I think thats really exciting because even though theres multiple isoforms, it seems that one might be more important from a gene therapy point of view, says Singh, who was not involved in the work. Its pretty exciting to have a specific target now.

Read more reports from the2020 International SYNGAP1 Scientific Conference.

Link:
Reactions from the 2020 SYNGAP1 Scientific Conference - Spectrum

Read More...

George Church backs a startup solution to the massive gene therapy manufacturing bottleneck – Endpoints News

Friday, November 20th, 2020

George Church and his graduate students have spent the last decade seeding startups on the razors edge between biology and science fiction: gene therapy to prevent aging, CRISPRed pigs that can be used to harvest organs for transplant, and home kits to test your poop for healthy or unhealthy bacteria. (OK, maybe theyre not allon that razors edge.)

But now a new spinout from the Department of Genetics second floor is tackling a far humbler problem one that major company after major company has stumbled over as they tried to get cures for rare diseases and other gene therapies into the clinic and past regulators: How the hell do you build these?

Theres a lot happening for new therapies but not enough attention around this problem, Lex Rovner, who was a post-doc at Churchs lab from 2015 to 2018, told Endpoints News. And if we dont figure out how to fix this, many of these therapies wont even reach patients.

This week, with Church and a couple other prominent scientists as co-founders, Rovner launched 64x Bio to tackle one key part of the manufacturing bottleneck. They wont be looking to retrofit plants or build gene therapy factories, as Big Pharma and big biotech are now spending billions to do. Instead, with $4.5 million in seed cash, they will try to engineer the individual cells that churn out a critical component of the therapies.

The goal is to build cells that are fine-tuned to do nothing but spit out the viral vectors that researchers and drug developers use to shuttle gene therapies into the body. Different vectors have different demands; 64x Bio will look to make efficient cellular factories for each.

While a few general ways to increase vector production may exist, each unique vector serotype and payload poses a specific challenge, Church said in an emailed statement. Our platform enables us to fine tune custom solutions for these distinct combinations that are particularly hard to overcome.

Before joining Churchs lab, Rovner did her graduate work at Yale, where she studied how to engineer bacteria to produce new kinds of protein for drugs or other purposes. And after leaving Churchs lab in 2018, she initially set out to build a manufacturing startup with a broad focus.

Yet as she spoke with hundreds of biotech executives on LinkedIn and in coffee shops around Cambridge, the same issue kept popping up: They liked their gene therapy technology in the lab but they didnt know how to scale it up.

Everyone kept saying the same thing, Rovner said. We basically realized theres this huge problem.

The issue would soon make headlines in industry publications: bluebird delaying the launch of Zynteglo, Novartis delaying the launch of Zolgensma in the EU, Axovant delaying the start of their Tay-Sachs trial.

Part of the problem, Rovner said, is that gene therapies are delivered on viral vectors. You can build these vectors in mammalian cell lines by feeding them a small circular strand of DNA called a plasmid. The problem is that mammalian cells have, over billions of years, evolved tools and defenses precisely to avoid making viruses. (Lest the mammal they live in die of infection).

There are genetic mutations that can turn off some of the internal defenses and unleash a cells ability to produce virus, but theyre rare and hard to find. Other platforms, Rovner said, try to find these mutations by using CRISPR to knock out genes in different cells and then screening each of them individually, a process that can require hundreds of thousands of different 100-well plates, with each well containing a different group of mutant cells.

Its just not practical, and so these platforms never find the cells, Rovner said.

64x Bio will try to find them by building a library of millions of mutant mammalian cells and then using a molecular barcoding technique to screen those cells in a single pool. The technique, Rovner said, lets them trace how much vector any given cell produces, allowing researchers to quickly identify super-producing cells and their mutations.

The technology was developed partially in-house but draws from IP at Harvard and the Wyss Institute. Harvards Pam Silver and Wysss Jeffrey Way are co-founders.

The company is now based in SoMa in San Francisco. With the seed cash from Fifty Years, Refactor and First Round Capital, Rovner is recruiting and looking to raise a Series A. Theyre in talks with pharma and biotech partners, while they try to validate the first preclinical and clinical applications.

Gene therapy is one focus, but Rovner said the platform works for anything that involves viral vector, including vaccines and oncolytic viruses. You just have to find the right mutation.

Its the rare cell youre looking for, she said.

Read more from the original source:
George Church backs a startup solution to the massive gene therapy manufacturing bottleneck - Endpoints News

Read More...

Avrobio tracks improvements in first patient treated with Gaucher gene therapy – FierceBiotech

Friday, November 20th, 2020

Avrobio has shared data on the first Gaucher disease patient to receive its gene therapy AVR-RD-02. The patient, who was stable on enzyme replacement therapy at baseline, experienced a 22% drop in a toxic metabolite after receiving AVR-RD-02 and stopping taking the standard of care.

Gaucher, like the Fabry disease targeted by Avrobios lead prospect, is currently treated using enzyme replacement therapies sold by Sanofi and Takeda, which entered the market through its takeover of Shire. However, a significant minority of patients experience physical limitations despite treatment. Negative outcomes include bone pain and spleen enlargement. Johnson & Johnsons Zavesca offers an oral alternative, but there remain unmet medical needs.

Avrobio is developing AVR-RD-02 to address those needs. The data shared as part of Avrobios R&D day mark the start of the effort to show AVR-RD-02 performs as hoped in the clinic.

Start using real-world data to advance your clinical research

Much has been written about the promise of real-world data (RWD) in life sciences, but how does it work in practice? We address this question in a new whitepaper that demonstrates the potential benefits of new RWD technologies with a proof of concept study to show how RWD can be incorporated into clinical research.

The first patient to receive AVR-RD-02 discontinued enzyme replacement therapy one month before taking the gene therapy. Three months after receiving the gene therapy, levels of Gaucher biomarker lyso-Gb1 had fallen 22%. The patients level of plasma chitotriosidase, a biomarker of cells associated with severe organ damage, was down 17%. Hemoglobin and platelets were in the normal range.

AVR-RD-02 triggered those changes without causing serious adverse events. The data drop offers an early indication that Avrobio may be able to improve outcomes by harvesting hematopoietic stem cells, adding a gene that encodes for glucocerebrosidase and reinfusing the cells back into the same patient. With enzyme replacement therapies costing healthcare systems up to $400,000 a year per patient, there is scope for AVR-RD-02 to cut the cost of treating Gaucher disease.

Avrobio shared the early look at clinical data on AVR-RD-02 alongside updates about other assets. There is now more than three years of data on some Fabry patients treated with Avrobios lead asset, putting the company in a position to plot a path to accelerated approval. Avrobio plans to submit its briefing book to the FDA by the end of the year to align on an accelerated approval strategy.

The update also covered cystinosis candidate AVR-RD-04. The first patient to receive the candidate is off oral and eye drop cysteamine 12 months after receiving the gene therapy. The number of crystals in the patients skin are down 56%, leading Avrobio to posit they may have gained the ability to make their own functional cystinosin protein.

The rest is here:
Avrobio tracks improvements in first patient treated with Gaucher gene therapy - FierceBiotech

Read More...

Lilly, Precision Biosciences team up on Duchenne gene therapy in $135M deal – FierceBiotech

Friday, November 20th, 2020

Once wary of inking deals around gene therapy, Eli Lilly is jumping headfirst into developing treatments that edit genes within the body. Its teaming up with cell and gene therapy biotech Precision BioSciences to develop in vivo gene therapies for three gene targets, starting with Duchenne muscular dystrophy.

Lilly is forking over $100 million upfront and investing $35 million in Precision BioSciences, but its on the hook for up to $420 million in development and commercialization milestones per product it takes forward. Under the deal, Precision will lead preclinical research and IND-enabling studies and pass the baton to Lilly for clinical development and commercialization.

RELATED: Gilead axes $445M Precision BioSciences gene therapy hep B pact

Start using real-world data to advance your clinical research

Much has been written about the promise of real-world data (RWD) in life sciences, but how does it work in practice? We address this question in a new whitepaper that demonstrates the potential benefits of new RWD technologies with a proof of concept study to show how RWD can be incorporated into clinical research.

Beyond Duchenne, the duo did not disclose the targets of the deal. Precisions stock jumped 10% on the news when the market opened Friday. The partnership is a boost for Precision, which lost Gilead Sciences as a partner in July when the company pulled the plug on a hepatitis B collaboration worth up to $445 million.

Lilly started 2019 with an $8 billion takeover of Loxo Oncology, signaling an appetite for new cancer drugsan area in which the company had been late to the game, CEO David Ricks told Reuters at the time. It would stay away from the fields of CAR-T and gene therapy, though, because, in their current iteration, they dont reach a lot of patients.

Almost everything I am aware of is single gene edit defects, which ultimately leads you to pretty ultra-rare conditions, which are not our area of interest, Ricks told Reuters.

Since then, the Indianapolis-based pharma has changed its tune.

RELATED: Off-the-shelf CAR-T and gene-editing player Precision Bio files $100M IPO

"This collaboration with Precision BioSciences represents another milestone in the realization of our vision to create medicines with transformational potential, using new therapeutic modalities such as gene editing to tackle targets and indications which were previously undruggable, said Andrew Adams, Ph.D., vice president of new therapeutic modalities at Eli Lilly, in a statement Friday.

Lilly and Precision will use the latters ARCUS genome editing platform to develop new gene therapies. The technology is based on a natural enzyme called a homing endonuclease that can insert or delete a piece of DNA before shutting itself off using a built-in safety switch. The switch is designed to prevent unwanted, off-target edits elsewhere in the genome.

Duchenne is caused by mutations in the dystrophin gene that stop it from producing a protein of the same name. Without it, muscle fibers, including those in the heart, eventually weaken and die.

Sarepta markets two drugs for Duchenne muscular dystrophy, Exondys 51 and Vyondys 53, for patients whose disease is amenable to skipping exons 51 and 53, respectively. Together, the drugs work for about one-fifth of patients with the disease. A gene therapy that addresses the dystrophin mutation could reach many more patients.

RELATED: Pfizer's DMD gene therapy looks good in data refresh, but safety concerns persist

Pfizer is working on a gene therapy for Duchenne, while Sarepta is working on a micro-dystrophin gene therapy using a truncated version of dystrophin. Ultragenyx recently jumped into the fray, teaming up with Solid Biosciences on gene therapies for Duchenne and other muscular dystrophies stemming from a lack of the dystrophin protein.

Read the original here:
Lilly, Precision Biosciences team up on Duchenne gene therapy in $135M deal - FierceBiotech

Read More...

Helixmith starts new trial of diabetic neuropathy gene therapy – – pharmaphorum

Friday, November 20th, 2020

Just over a year after its first phase 3 trial of its Engensis gene therapy for painful diabetic peripheral neuropathy (DPN) bombed, Helixmith has started dosing patients in a new study.

The South Korean biotech says the DNA plasmid-based therapy has been administered to a patient at Innovative Research of West Florida. The aim is to enrol 152 DPN patients at 15 clinical sites across the US in the study, called REGAiN-1A.

Helixmith has previously suggested that if positive, the new study could support marketing applications for Engensis. It is due to generate results in December 2021, which could lead to filings in 2022.

While most gene therapies in late-stage development target rare diseases, DPN is a relatively common condition and is likely to become even more prevalent as diabetes is becoming more common around the world. It has a lifetime prevalence of around 50% in people with diabetes, with around half of these having pain.

In DPN, prolonged exposure to higher than normal blood sugar levels damages nerves, most commonly in the legs and feet but also in the arms and hands.

At the moment treatment is limited to drugs like gabapentin and pregabalin, which are only palliative and dont tackle the underlying cause of the condition.

Engensis also known as VM202 is a DNA plasmid-based gene therapy that is administered as an intramuscular injection into the calves, delivering a gene coding for human hepatocyte growth factor (HGF).

The hope is that delivery of Engensis to the lower limbs might promote nerve system regeneration and alleviate the pain that often accompanies DPN, whilst also promoting blood vessel growth in the extremities.

In its first 500-patient phase 3 trial, called DPN 3-1, Engensis was no better than placebo at reducing pain scores over the first 90 days of the trial. However Helixmith (formerly known as ViroMed) said that was due to a major mix-up in the study protocol, which undermined the results.

Analysis of samples taken from patients in the placebo group found traces of the VM202 plasmid, suggesting that clinicians may have inadvertently administered the gene therapy to the control group.

There was also a wide variation in the amount of plasmid DNA among the treatment group, which might suggest inaccuracies in the administration of the gene therapy. On the plus side, safety results were in keeping with a benign profile seen in earlier-stage clinical trials.

Helixmith then started a phase 3 extension study in 101 subjects from DPN 3-1 conducted under a separate protocol to look at long-term safety and efficacy at 12 months.

That backed up the safety data for the therapy, and also found significant pain reductions compared to placebo after six, nine and 12 months, as well as a trend towards reduced pain at three months.

The primary measure in REGAiN-1A will be a comparison of the average daily pain scores from seven days prior to the first injection, to seven days prior to the six-month visit between both the Engensis and placebo groups.

Secondary efficacy measures include pain reduction at six months compared to placebo, as well as the proportion of patients experiencing a 50% reduction in pain at six months. The therapy will be administered by injection into the calf at day zero, 14, 90 and 104.

See the original post here:
Helixmith starts new trial of diabetic neuropathy gene therapy - - pharmaphorum

Read More...

Page 20«..10..19202122..3040..»


2025 © StemCell Therapy is proudly powered by WordPress
Entries (RSS) Comments (RSS) | Violinesth by Patrick