Category Archives: Genetic Medicine

LEXINGTON, Mass., Nov. 12, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery today announced senior member of management will be presenting at upcoming investor conferences

Links to all presentations will be available under the investors tab at http://www.logicbio.com upon their availability.

About LogicBio Therapeutics

LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration withTakedato research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

Contact:

Matthias Jaffe Chief Financial Officer mjaffe@logicbio.com (617) 245-0399

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LogicBio Therapeutics to Present at Upcoming Investor Conferences - GlobeNewswire

The researchers measured the visceral adipose tissue (VAT) and liver fat (LF) from magnetic resonance images of 12,276 participants from the UK Biobank, the researchers assessed and divided individuals into four groups defined by sex-specific median values of VAT and LF: low VAT-low LF, low VAT-high LF, high VAT-low LF, and high VAT-high LF. Participants were followed for 1.3 years (on average) to detect VAT-LF groups' associations with CHD incidence.

In total, 176 CHD events were recorded, revealing that high visceral adiposity increased risk for CHD; this effect was exacerbated in those who also had low liver fat elevating the risk for CHD greater than two-fold compared with low VAT-low LF. High liver fat in combination with low visceral adiposity (low VAT-high LF), did not increase the risk for CHD. After adjusting for age and body mass index, high VAT-high LF association with CHD diminished, but the increased risk of CHD among those with high VAT-low LF persisted.

"We believe knowledge of patient risk for disease is limited when assessment is restricted to single, isolated fat depots. When developing NAFLD treatments, a decrease in liver fat alone may not be sufficient to lower patients' cardiometabolic risk. In fact, the research shows that decreasing liver fat without resolving visceral obesity may put the patient at greater risk of heart disease. This is what we want to investigate further,"stated Jennifer Linge, Lead Scientist, Personalized Medicine at AMRA Medical.

The results suggest that heterogeneity of body fat distribution affects CHD risk. Specifically, risk for CHD increased among those with high visceral adiposity, which intensifies in the presence of low liver fat indicating that liver triglyceride regulation plays a vital role in cardiovascular health in the context of visceral obesity.

Learn more about these findings by viewing the recorded presentation. The corresponding abstract "Can Low Liver Fat Be Bad for Your Heart? The High Visceral Fat, Low Liver Fat Phenotype: A Risk Factor for Coronary Heart Disease" (number 89) can be found in the journal HEPATOLOGY.

About UK Biobank

UK Biobankis large-scalebiomedical database and research resource, containing in-depth genetic andhealth information from halfa million UK participants. The database, which isregularly augmented with additional data, is globally accessible to approvedresearchersand scientists undertaking vital research into the most common andlife-threatening diseases. UK Biobank's researchresource is a majorcontributor to the advancement of modern medicine and treatment and has enabledseveral scientific discoveriesthat improve human health.

About AMRA Medical

AMRAis a ground-breaking international digital health company at the forefront of medicalimaging and precision medicine. The company has developed a new global standard in bodycomposition assessment, the ability to automatically produce multiple fat and musclebiomarkers with unrivaled precision and accuracy, as well as contextual disease insights allfrom a single, rapid, whole-body MRI.

SOURCE AMRA Medical

AMRA Medical | Medical Imaging and Body Composition Analysis

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AMRA Medical Research Reveals New MRI-based Data Connected to Risk for Coronary Heart Disease at The Liver Meeting Digital Experience - PRNewswire

Ryan Butler was 19 and playing guitarin two touring bands on the heavier end of the musical spectrum Wellington and Unruh when he lost his mother to Wilson's disease, agenetic disorderin which excesscopperbuilds up in the body and eats through the liver.

He had just turned 43, the age at which his motherdied, when a doctor confirmed what Butler had already long suspected.

He, too, had contracted a disease so rare a liver doctor told him he would more than likely be the only Wilson's patient the doctor'sstaff would ever see.

This was in August 2019, after six years of regular visitstoa hematologist for what hesays were "really low platelets," a sign of a liver disorder.

As the guitarist recalls, "I told him over and over 'My mom passed from Wilson's and I know it can be passed down genetically, even though it's rare. Please check me.' 'Oh, no, you don't have Wilson's.' Over and over with this guy. And he could never figure out what was wrong with me."

Then, the eye problems started.

"I got acute angle glaucoma, out of nowhere, and lost the vision in one eye," Butler says. "I was getting all kinds of weird sicknesses and fevers. And the eye doctor was like 'There's something wrong with you. You need to figure out what's going on.'"

That's when a trip to thegastroenterologist confirmed his worst fears.

As Butler recalls, "That doctorwas like, 'You have Wilson's disease. I'm gonna send you to a liver specialist right away."

Ryan Butler on stage at Club Red for the Unruh reunion in 2015.(Photo: Valerie Littlejohn, No Ceiling Photography)

It's been a month since Butler's liver transplant.

"I feel pretty lucky to have gotten this far through it," he says of life with Wilson's.

"The month before I got the transplant, theystopped having me take medicine. None of it was working anymore because my liver couldn't metabolize it. It was just so shot. So it's pretty intense. You degrade slowly over time. And then it just hits you."

Normal platelet levels are about 200, Butler says. Before the transplant, his were in the 50s. Now they're back in the 170s and 180s.

"I'm pretty much couchbound," he says. "In my head, I feel so much better but then I try to move around and it's like, 'Oh yeah, I'm not feeling good.'"

As he continues to recover, Butler will be facing major medical expenses.

"I haven't worked in months because I've been so sick," he says.

"I've given almost everything I've made these last two years to doctors and a dentist to get ready for the transplant. And my wife hasn't worked in months because she's been taking care of me. And the bills just keep coming when you go through something like this."

He was told that the cost ofthe medication alone will be enough for him to meet his $7,000 deductible every January.

"I don't exactly have that just sitting around for fun," he says. "So I'm gonna have to figure that out every year from now on."

In the meantime, his friends in the music community have come to Butler's aid with a virtual benefit concert they've called Butler Fest at 3 p.m. Saturday, Nov. 21, to help him cover medical expenses.

Among the artists who came forward with exclusive filmed performances are Jim Adkins of Jimmy Eat World, Exhumed, Dropdead, Fall Silent, Papa M, Austin Lunn of Panopticon, Bobb Bruno of Best Coast, Goya, Lago, Sorrower and Broloaf.

Broloaf is the last band Butler did a show with, playing Cro Mags covers at the Yucca Tap Room as part of their Halloween weekend festivities in 2019.

"And I had had my biopsy on my liver a few days before that," Butler recalls. "And I was puking in the trash can before our set. I was actually talking with one of the guitarists from Broloaf about starting a new band. And after that, I was like, yeah, I'm not gonna be playing for a while."

Another former bandmate,Danny Marianino of North Side Kings, is contributing a set of standup comedy.

Ryan Butler and other members of Landmine Marathon.(Photo: Provided by the artist)

And it was yet another former bandmate, Matt Martinez of Landmine Marathon, whoapproached him about the idea of putting a benefit show together. He enlisted the aid and technical support ofMichelle Donovan at the Nile Theatre in Mesa, where many of the sets are being filmed.

"Matt kind of just surprised me with the lineup," Butler says. "He was like, I'm gonna do a benefit to stream online. And I was like, 'OK.'"

Then he showed him the lineup.

"And I was like, 'Are you kidding me?,' Butler recalls with a laugh. "So it was a real shocker. And it really kind of reflects my musical tastes, honestly. It's all over the map."

It's been an overwhelming experience for Butler to see these people come together in his hour of need.

"A guy like Jim Adkins or Papa M, it's hard to believe they would taketime out of their day to film a set for me," he says.

"Exhumed is a big death metal band. They had to fly their drummer out to do it. It's just pretty amazing that they're coming together just to help me out. It really feels special. I'm just glad I'm well enough that I'll be able to kind of enjoy it."

Martinez figures he's known Butler almost 30 years.

"Just through punk rock and playing in bands when we were in high school, crossing paths," he says. "Our previous bands in the '90s played together a lot. And even before he was in Landmine, he recorded Landmine in his studio."

Getting a benefit together for his friend was pretty much a given for Martinez, who also started a GoFundMe for Butler in late August.

"I've experienced extreme life-changing medical events, and I know how our healthcare system works," he says.

"I knew there was gonna be a huge financial burden associated with not only the procedure itself but the aftereffects, the healing process, not being able to go to work immediately. So I networked with Michelle and we brainstormed ways to make a virtual benefit happen."

A lot of the filming was done at the Nile.

"Michelle has a crew that's really adept and in tune with the technology side of it and making that happen," Martinez says. "We've definitely faced a lot of challenges going through the process and adapted to them. So it's been a fun learning experience"

Martinez was beyond impressed by the willingness of the artists he approached about sharing their talent and time to help his friend.

"Especially with the national artists, you ask with theideathat they'll probably say no," he says.

"They're either not gonna be available or it's just not gonna be their thing. But 95% of this lineup we envisioned said yes immediatelywithout question. It was pretty humbling and kind of a testament to Ryan's friendships that he forms."

These are friendships Butler started forming while still in his formative years at Arcadia High School, having hit the road hisjunior year with Lyburnum, a screamo band.

From there, he started Wellington, a doom-metal trio that released one album and a handful of EPs.

His next group, Unruh, toured the hardcore circuit in the States and Europe and released two albums and some EPs before breaking up.

After a brief stint in Structure of Lies, he was invited to joinNorth Side Kings as they were gearing up to tour with H2O and Madball, joining Landmine Marathon, whose debut album he'd recorded in his studio, Arcane Digital Recording, in 2006.

He spent the next 10 years in Landmine Marathon, which did more touring and recording than those other groups, while still playing in North Side Kings.

Butler hasn't done much playing since Landmine ended in late 2015. There's been a North Side Kings reunion show and that ill-fated Broloaf show with the trash can.

This past spring, he says, "Mobility was becoming an issue. By summer I was completely on a cane and not really getting around much at all. Plus the coronavirus. I couldn't be around that at all with my immunocompromised condition."

Ryan Butler with his wife, Amy.(Photo: Provided by the artist)

Playing in bands isa side of his life he's missed these past few years.

"When Landmine stopped, I had been touring and playing in multiple bands for so long, I was like, 'I need a break,'" he recalls.

"So I didn't pick up anything. And North Side Kings talked about playing more after that reunion. But my dad passed away and I had to deal with all kinds of stuff. So we never ended up playing any more shows. AndI got really sick shortly after that."

Being laid up in the hospital and even now that he's back home recovering has given Butler lots of time to think.

"The second I came out of the transplant a month ago, I felt awful because of the surgery, but Iknew instantly that my symptoms were gone," he says.

"So to be sitting there with nothing but time on your hands, you just start thinking about the future. And I'm definitely gonna be tuning up those guitars soon."

Details: 3 p.m. Saturday, Nov. 21. theniletheater.com.$10. You also can donate at Butler's GoFundMe.

Reach the reporter at ed.masley@arizonarepublic.com or 602-444-4495. Follow him on Twitter @EdMasley.

Support local journalism.Subscribe to azcentral.com today.

Read or Share this story: https://www.azcentral.com/story/entertainment/music/2020/11/17/phoenix-musician-rare-genetic-liver-disorder-healthcare/6273638002/

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A rare disease almost killed him but this Phoenix rocker's friends are helping him survive - The Arizona Republic

- Expanded leadership team with appointment of Sachiyo Minegishi as CFO and promotion of Jennifer Wellman to COO -

- Continued progress towards 2021 IND submission for AK-OTOF, a gene therapy intended for the treatment of hearing loss due to mutations in the OTOF gene -

- Execution on build of internal manufacturing capabilities and infrastructure to support future research and clinical trials is on track -

BOSTON, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Akouos,Inc. (Nasdaq: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reported financial results for the third quarter ended September 30, 2020 and provided updated business highlights.

We continue to advance AK-OTOF, a gene therapy intended for the treatment of hearing loss due to mutations in the OTOF (otoferlin) gene, towards a 2021 IND filing. Today, individuals with OTOF-mediated hearing loss have no therapeutic options. Our novel gene therapy candidate has the potential to restore hearing for these individuals, who typically have no functional hearing at birth, said Manny Simons, Ph.D., founder, president and CEO of Akouos. Also, despite the challenging environment around us, the team is executing on our plan to establish internal manufacturing capabilities and infrastructure to support IND-enabling studies and clinical trials. Our progress towards our long-term mission, making healthy hearing available to all, is a testament to our deeply committed, experienced team and our strategic partners.

Business and Pipeline Highlights

Third Quarter 2020 Financial Results

About Akouos

Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, our expectations regarding our manufacturing capabilities, and the period over which we believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our plans to develop and, if approved, subsequently commercialize our product candidates; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our expectations regarding our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing and manufacturing capabilities and strategy; our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates; our intellectual property position; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations; our competitive position and expectations regarding developments and projections relating to our competitors and any competing therapies that are or become available; developments and expectations regarding developments and projections relating to our competitors and our industry; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the Risk Factors included in the Companys Quarterly Report on Form 10-Q for the three months ended June 30, 2020 filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Condensed Consolidated Balance Sheet Data(Unaudited)

(in thousands)

Condensed Consolidated Statements of Operations and Comprehensive Loss(Unaudited)

(in thousands, except share and per share data)

Contacts

Media:

Katie Engleman, 1ABkatie@1abmedia.com

Investors:

Courtney Turiano, Stern Investor RelationsCourtney.Turiano@sternir.com

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Akouos Reports Third Quarter 2020 Financial Results and Provides Business Highlights - GlobeNewswire

Here's a look at which companies are raking in cash this week in the biopharma industry.

Apollomics, Inc.

Committed tocombatting cancer with precision,Apollomics plans to use the$124.2 million Series Cfinancing to focusclinicalefforts on its lead programs: APL-101 and APL-106. APL-101 is an oral c-MET inhibitorcurrently involved in several ongoing clinical trials. TheSPARTA trialis in Phase II, targeting non-small cell lung cancer,glioblastomamultiforme and solid tumors with MET amplifications.APL-106 is a first-in-class targeted inhibitordesignedtoblockE-selectin, an adhesion molecule on cells in bone marrow,from binding with blood cancer cells.It has received Breakthrough Therapy Designation from the FDA in relapsed and refractory acute myeloid leukemia.In 2019,Apollomicsraised$100 million in a Series B round.

Decibel Therapeutics

Decibel Therapeutics made some noise this week with an oversubscribed Series D financing, raking in$82.2 million. The company will use the funds to advance DB-OTO, a gene therapy to restore hearing in children with congenital deafness due to a deficiency in the otoferlin gene.Clinical testing is expected to initiate in2022.DB-020 is already in a phase Ib study with cancer patients as apreventative treatment for the ototoxicityassociated with cisplatin-based chemotherapy.Hearing and balance disorders have historically been overlooked by the biopharma industry, even though they exact a devastating toll on the lives of hundreds of millions of people around the globe. At Decibel, we are dedicated to restoring hearing and balance with precision therapeutics designed to deliver the right genetic medicine specifically to the right cells in the ear, said Laurence Reid, Ph.D., Chief Executive Officer of Decibel.

Adagio Therapeutics

After launchingin Junewith $50 million, Adagio has pocketed another$80 million in a Series Bfinancing round to take its COVID-19 antibody into the clinic next year. Adagios neutralizing monoclonal antibodies are expected to provide broad protection against not onlyagainst SARS-CoV-2 and SARS-CoV-1, but also additional bat coronaviruses that have yet to cross the species barrier. Adagio got the green light from the FDA to proceed with their first-in-human study in early 2021.We were impressed by the thoughtful approach that Adagio took. By dealing with the broader coronavirus problem, we expect ADG20 to be more resistant to escape mutations and potentially cover future coronavirus pandemics, said Krishna Yeshwant, Managing Partner at GV. As a preventative agent, ADG20 holds the promise of providing the efficacy necessary to deliver greater protection against COVID-19. Given its unique combination of attributes, ADG20 could complement and supplement vaccines by providing rapid, durable antibody protection against current and future coronaviruses.

InmageneBiopharmaceuticals

Drug development companyInmagenehas its eye on being number one in immunology in China.The$21 million Series Bclosed this week will be pumped into conducting global clinical trials, research and development, and product in-licensing activities.Currently candidate IMG-020 is in a Phase II psoriasis trial, with a strong safety profile and clear clinical benefits giving it best-in-class potential. Manufactured inan E. coli system, IMG-020 is less than 1/20thof the average manufacturing cost of a typical antibody drug.The candidate is about to enter global registration trials formultiple indications.

Locus Biosciences

Its been a busy season for this CRISPR-engineer.In September,Locussigneda$144 million contract with BARDAto develop theirproduct targeting E. coli bacteria causing recurrent urinary tract infections. This week they closed a$14.4 million deal with CARB-Xto advance development of LBP-KP01, another CRISPR Cas3-enhanced bacteriophage (crPhage) product, targeting K. pneumoniae.The initial indication will be to target recurrent UTIs, then development for targeting lung infections (pneumonia), intra-abdominal infections (IAIs) and bacteremia.Together, the two cocktails have the potential to treat more than 90% of UTIs.Auniquedual mechanismof bacteria-hunting bacteriophages along with the DNA-targetingCRISPR-Cas3makesLocuscandidates significantly more effective at killing the targeted bacteria cells, regardless of whether they are resistant to antibiotics.Both the U.S. Centers for Disease Control and Prevention (CDC) and World Health Organization (WHO) have identified antibiotic-resistant K. pneumoniaeas an urgent and serious public health threat requiring development of new treatments.

Memo Therapeutics

Swiss innovator Memo Therapeutics has raised over$15.3 million in a Series Bprimarilyto advance its COVID-19 antibody treatment.The company entered into a partnership with NorthwayBiotechpharmain August to manufactureMTX-COVAB, which is currently going through a fast-tracked development path as an immunotherapy and a preventative of the novel coronavirus. Memo plans to begin clinical studies in 2021. Proceeds will also be used to advance its neutralizing antibody MTX-005 against BK virus infection in renal transplant patients into Phase II studies."We believe Memo Therapeutics AG has taken innovation in the field of antibody discovery to the next level. Their ability to exploit the power of microfluidic single-cell molecular cloning could not only serve to move one step closer to conquer the COVID pandemic but also potentially other infectious diseases and cancer, said Dr. Robert Schier, Investment Director atSwisscantoInvest.

Trailhead Biosystems

Pushing the boundaries of dimensional testing, Trailhead is increasing the speed, lowering the cost and reducing the risk of developing cell therapies. A$6.6 millioninfusion of cash will expand the companys High Dimensional Design of Experiments platform (HD-DoE) to support the generation of multiple specialized human cells with therapeutic properties and theirpilotscalemanufacturing.Trailheads aim is to rapidly develop the capability to create highly pure, specialized human cell types for regenerative medicine and therapeutic purposes at an industrial scale. "Biology is complex, but conventional science is not," saysJan Jensen, Ph.D., Chief Executive Officer and founder of Trailhead Biosystems. "We created Trailhead Biosystems to address key limitations in the scientific process, unlocking a deeper understanding of biology that will enable us to better control it."

Originally posted here:
Biopharma Money on the Move: November 4-10 - BioSpace

PHILADELPHIA, Nov. 11, 2020 (GLOBE NEWSWIRE) -- Passage Bio Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today commends the newly published research of the University of Pennsylvanias (Penn) Gene Therapy Program (GTP) regarding a novel targeted approach to prevent a selective neurotoxicity seen in the sensory neurons of dorsal root ganglia (DRG) after gene therapy treatment. As previously published, this DRG toxicity has been observed after both systemic and central nervous system (CNS) delivery of gene therapy and across a variety of vectors in pre-clinical models, but clinical manifestations have not been observed.1

As part of its unique collaboration agreement with Penn, Passage Bio has certain rights to this novel DRG technology for the indications the company progresses with Penn.

Although our safety studies for our programs have not shown any clinical manifestations of DRG toxicity, we are excited about the promising approach developed by Penns GTP, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. As part of our mission to develop transformative therapies for patients, we remain committed to advancing the field of gene therapy. If in the future this new approach shows clinical benefit for patients, we will be in a strong position to incorporate it into our programs. Our relationship with Penns GTP is an important distinguishing characteristic of Passage Bio. Through our collaboration, we have ready access to world-class expertise and groundbreaking research that we can rapidly apply, if appropriate, to our therapeutic programs.

GTPs research on preventing DRG toxicity published online this week in Science Translational Medicine. According to the researchers, DRG toxicity is the result of over expression of an introduced gene, known as a transgene, in cells in the DRG, a cluster of neural cells on the outside of the spinal cord responsible for transmission of sensory messages. To correct this over expression, the GTP research team modified a transgene with a microRNA target designed to reduce the level of the transgene expression in DRG neurons as well as toxicity in DRG neurons, without affecting transduction elsewhere in the brain. That alteration eliminated more than 80 percent of the transgene expression in DRG neurons and reduced the related DRG toxicity in preclinical studies with primates.

James M. Wilson, M.D., Ph.D., director of Penns GTP and a chief scientific advisor at Passage Bio, served as a senior author of the published manuscript. Juliette Hordeaux, DVM, Ph.D., senior director of Translational Research in Penns GTP is first author. They reported that their microRNA target approach may be a straightforward way to potentially make AAV therapy for the central nervous system more safe.

As previously reported, results from preclinical toxicology studies for Passage Bios lead therapeutic programs, PBGM01 (GM1 gangliosidosis), PBKR03 (Krabbe disease), PBFT02 (FTD-GRN), were consistent with this overall AAV platform observation, and showed no clinical manifestations in detailed neurological examinations or daily observations. To proactively determine whether there is appearance of clinical signs of DRG toxicity in our clinical programs, Passage Bio will implement monitoring of patients, consisting of both nerve-conduction studies and neurological exams focused on sensory and peripheral nerve functions.

Passage Bio is advancing six programs, which include the lead programs for GM1 gangliosidosis (GM1), Krabbe disease, and frontotemporal dementia (FTD), as well as three additional programs for amyotrophic lateral sclerosis (ALS), metachromatic leukodystrophy (MLD) and Charcot-Marie-Tooth disease Type 2a (CMT2a). The company anticipates that the initial three clinical candidates will be in clinical trials in 2021. Through its collaboration agreement with Penn, Passage Bio has the option to license a total of 17 programs focused on rare, monogenic disorders of the CNS.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvanias Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with unparalleled access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at http://www.passagebio.com.

Penn Financial Disclosure

Dr. Wilson is a Penn faculty member and also a scientific collaborator, consultant and co-founder of Passage Bio. As such, he holds an equity stake in the Company, receives sponsored research funding from Passage Bio, and as an inventor of certain Penn intellectual property that is licensed to Passage Bio, he may receive additional financial benefits under the license in the future. The University of Pennsylvania also holds equity and licensing interests in Passage Bio.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including our planned IND submissions, initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators and partners ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with theSecurities and Exchange Commission(SEC), and other reports as filed with theSEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:Sarah McCabe and Zofia MitaStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.comZofia.mita@sternir.com

Media:Gwen FisherPassage Bio215-407-1548gfisher@passagebio.com

1 Juliette Hordeaux, Elizabeth L. Buza, et al. Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology, Human Gene Therapy, published online June 25, 2020.

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Passage Bio Highlights University of Pennsylvania's Gene Therapy Program's Newly Published Research to Prevent Toxicity Associated with Gene Therapy -...