Category Archives: Gene Medicine

The CRISPR gene-editing tool can be used to silence an important hepatitisB virus gene, a proof-of-concept invitro study suggests.

"It's the first time we've seen CRISPR editing done in a hepatitisB model," said Douglas Dieterich, MD, director of the Institute of Liver Medicine and professor of medicine at the Icahn School of Medicine at Mount Sinai in New York City.

HepatitisB can lead to liver disease and is the primary cause of hepatocellular carcinoma. In 2015, more than 250million people around the world were infected with the virus, according to the World Health Organization.

For their study, investigator Hao Zhou, from The First Hospital of Jilin University in China and the Department of Medicine at the University of Minnesota in Minneapolis, and colleagues targeted the Sgene. Zhou presented the findings at the Liver Meeting 2019 in Boston.

The Sgene gives rise to the hepatitisB surface antigen, the presence of which indicates that a person is infected with the virus. "The question is whether it's the right target," Dieterich told Medscape Medical News.

Reducing the amount of the hepatitisB surface antigen is a "good idea" because that's what is believed to inhibit the immune system from clearing the virus. Doing so might help the immune system recover and clear the virus, "with a little help from some antivirals," explained Dieterich, who was not involved in the study.

However, "the surface is not the only DNA that's integrated into the host genome," he pointed out. "I think maybe a broader application might be necessary to actually get the hepatitisB genome out of the hepatocytes."

Zhou's team used a newer CRISPR approach, called CRISPR-STOP, for their gene-editing procedure.

"The idea is that CRISPR-STOP can be as efficient as standard CRISPR editing, but it's safer," said Kiran Musunuru, MD, PhD, associate professor of cardiovascular medicine and genetics at Penn Medicine in Philadelphia, who was not involved in the study. Musunuru is cofounder of and senior scientific advisor at Verve Therapeutics, a company using gene editing to prevent cardiovascular disease.

The standard CRISPR-Cas9 approach requires a double-strand break in the genome, and the problem with that is it introduces the possibility for "mischief," he explained. "If you have more than one double-strand break occurring in the human genome at the same time, you have the potential for different parts of different chromosomes coming together in the wrong ways and then causing problems."

Instead of creating a double-strand break, CRISPR-STOP uses a base editor to chemically modify the DNA base from one base to another and introduce a stop codon into the target gene sequence, effectively hamstringing the ability of the target gene to produce a functional protein.

This is a very nice, clean way to turn off a gene effectively.

"This is a very nice, clean way to turn off a gene effectively," Musunuru told Medscape Medical News.

For their CRISPR-STOP procedure, Zhou's team first transduced liver cells infected with the hepatitisB virus using a base editor called AncBE4max. Next, to activate the base editor so that gene editing could begin, they transduced the cells with one of two lentivectors: one encoded for single-guide RNA that targets the Sgene; and an empty one, which served as the control.

With the gene-editing approach, 71% of the liver cells that expressed the base editor gained the desired stop codon in the target gene.

"That's a very robust number," said Musunuru.

In addition, hepatitisB surface antigen secretion was reduced by 92% with the gene-editing approach.

The investigators report a high degree of conservativity for hepatitisB genotypesB, C, F, and H. Specifically, 94% of the Sgene sequence was conserved for genotypeB, 92% for genotypeC, 91% for genotypeF, and 71% for genotypeH.

The Liver Meeting 2019: American Association for the Study of Liver Diseases (AASLD): Abstract86. Presented November10, 2019.

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CRISPR Used to Silence Crucial Hepatitis B Gene - Medscape

If certain vegetables have always made you gag, you may be more than a picky eater. Instead, you might be what scientists call a super-taster: a person with a genetic predisposition to taste food differently.

Unfortunately, being a super-taster doesnt make everything taste better. In fact, it can do the opposite.

Super-tasters are extremely sensitive to bitterness, a common characteristic of many dark green, leafy veggies such as broccoli, cauliflower, cabbage and Brussels sprouts, to name a few.

The person who has that genetic propensity gets more of the sulfur flavor of, say, Brussels sprouts, especially if theyve been overcooked, said University of Connecticut professor Valerie Duffy, an expert in the study of food taste, preference and consumption.

So that [bitter] vegetable is disliked, and because people generalize, soon all vegetables are disliked, Duffy said. If you ask people, Do you like vegetables? They dont usually say, Oh yeah, I dont like this, but I like these others. People tend to either like vegetables or not.

In fact, people with the bitter gene are 2.6 times more likely to eat fewer vegetables than people who do not have that gene, according to a new study presented Monday at the annual meeting of the American Heart Association.

We wanted to know if genetics affected the ability of people who need to eat heart-healthy foods from eating them, said study author Jennifer Smith, a registered nurse who is a postdoc in cardiovascular science at the University of Kentucky School of Medicine.

While we didnt see results in gene type for sodium, sugar or saturated fat, we did see a difference in vegetables, Smith said, adding that people with the gene tasted a ruin-your-day level of bitterness.

Our sense of taste relies on much more than a gene or two. Receptors on our taste buds are primed to respond to five basic flavors: salty, sweet, sour, bitter and umami, which is a savory flavor created by an amino acid called glutamate (think of mushrooms, soy sauce, broth and aged cheeses).

But its also smelling through the mouth and the touch, texture and temperature of the food, Duffy said. Its very difficult to separate out taste from the rest. So when any of us say the food tastes good, its a composite sensation that were reacting to.

Even our saliva can enter the mix, creating unique ways to experience food.

When we come to the table, we dont perceive the food flavor or the taste of food equally, Duffy said. Some people live in a pastel food world versus others who might live in a more vibrant, neon food world. It could explain some of the differences in our food preference.

While there are more than 25 different taste receptors in our mouth, one in particular has been highly researched: the TAS2R38, which has two variants called AVI and PAV.

About 50% of us inherent one of each, and while we can taste bitter and sweet, we are not especially sensitive to bitter foods.

Another 25% of us are called non-tasters because we received two copies of AVI. Non-tasters arent at all sensitive to bitterness; in fact food might actually be perceived as a bit sweeter.

The last 25% of us have two copies of PAV, which creates the extreme sensitivity to the bitterness some plants develop to keep animals from eating them.

When it comes to bitterness in the veggie family, the worst offenders tend to be cruciferous vegetables, such as broccoli, kale, bok choy, arugula, watercress, collards and cauliflower.

Thats too bad, because they are also full of fiber, low in calories and are nutrient powerhouses. Theyre packed with vitamins A and C and whats called phytonutrients, which are compounds that may help to lower inflammation.

Rejecting cruciferous or any type of vegetable is a problem for the growing waistline and health of America.

As we age as a population, vegetables are very important for helping us maintain our weight, providing all those wonderful nutrients to help us maintain our immune system and lower inflammation to prevent cancer, heart disease and more, Duffy said.

Food scientists are trying to develop ways to reduce the bitterness in veggies, in the hopes we can keep another generation of super-tasters from rejecting vegetables.

Theres been some success. In fact, the Brussels sprouts we eat today are much sweeter than those our parents or grandparents ate. Dutch growers in the 90s searched their seed archives for older, less bitter varieties, then cross-pollinated them with todays higher-yielding varieties.

People who already reject vegetables might try to use various cooking methods that can mask the bitter taste.

Just because somebody carries the two copies of the bitter gene doesnt mean that they cant enjoy vegetables, Duffy said. Cooking techniques such as adding a little fat, a little bit of sweetness, strong flavors like garlic or roasting them in the oven, which brings out natural sweetness, can all enhance the overall flavor or taste of the vegetable and block the bitterness.

More:
Your hatred of heart-healthy veggies could be genetic - WTVR CBS 6 News

Amended Celgene collaboration to focus on engineered alpha-beta T cell medicines with a $70 million payment to Editas Medicine

Appointed Judith R. Abrams, M.D., as Chief Medical Officer

EDIT-101 (AGN-151587) for LCA10 first patient dosing expected by early 2020

EDIT-301 for hemoglobinopathies in vivo pre-clinical data to be presented at ASH

CAMBRIDGE, Mass., Nov. 12, 2019 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (EDIT), a leading genome editing company, today reported business highlights and financial results for the third quarter of 2019.

"Our momentum in 2019 remains strong in advancing our pipeline of in vivo CRISPR and engineered cell medicines," said Cynthia Collins, Chief Executive Officer of Editas Medicine. We announced this morning an amended agreement with Celgene to further expand and accelerate our oncology pipeline. In hemoglobinopathies, we look forward to presenting in vivo pre-clinical data for EDIT-301 at ASH that supports its potential as a best-in-class medicine. Finally, we eagerly anticipate first patient dosing with EDIT-101 for LCA10 in the coming months.

Recent Achievements and OutlookIn VivoCRISPR Medicines

Engineered Cell Medicines

Corporate

Upcoming Events

Editas Medicine will participate in the following investor events:

Editas Medicine will present pre-clinical data for EDIT-301 to address sickle cell disease and beta-thalassemia in at the 61st American Society of Hematology Annual Meeting & Exposition. Details are as follows:

Abstract Number: 4636Title: EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCDPresenter: Edouard De Dreuzy, Ph.D.Session: 801. Gene Therapy and Transfer: Poster III Time: Monday, December 9, 2019: 6:00 PM-8:00 PMLocation: Hall B, Orange County Convention Center, Orlando, FL

Third Quarter 2019 Financial Results

Cash, cash equivalents, and marketable securities at September 30, 2019, were $332.6 million, compared to $369.0 million at December 31, 2018. The $36.4 million decrease was primarily attributable to operating and capital expenses related to our on-going preclinical and clinical activities, patent costs and license fees, and employee-related costs, partially offset by $42.1 million in proceeds from financing activities.

For the three months ended September 30, 2019, net loss was $32.9 million, or $0.66 per share, compared to $15.2 million, or $0.32 per share, for the same period in 2018.

Conference Call

The Editas Medicine management team will host a conference call and webcast today at 8:00 a.m. ET to provide and discuss a corporate update and financial results for the third quarter of 2019. To access the call, please dial 844-348-3801 (domestic) or 213-358-0955 (international) and provide the passcode 6577216. A live webcast of the call will be available on the Investors & Media section of the Editas Medicine website at http://www.editasmedicine.com and a replay will be available approximately two hours after its completion.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

About EDIT-101 (AGN-151587)EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.

About Leber Congenital AmaurosisLeber congenital amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 2030 percent of all LCA patients.

Story continues

About the Editas Medicine-Allergan AllianceIn March 2017, Editas Medicine and Allergan Pharmaceuticals International Limited (Allergan) entered a strategic alliance and option agreement under which Allergan received exclusive access and the option to license up to five of Editas Medicines genome editing programs for ocular diseases, including EDIT-101 (AGN-151587). Under the terms of the agreement, Allergan is responsible for development and commercialization of optioned products, subject to Editas Medicines option to co-develop and share equally in the profits and losses of two optioned products in the United States. In August 2018, Allergan exercised its option to develop and commercialize EDIT-101 globally for the treatment of LCA10. Additionally, Editas Medicine exercised its option to co-develop and share equally in the profits and losses from EDIT-101 in the United States. Editas Medicine is also eligible to receive development and commercial milestones, as well as royalty payments on a per-program basis. The agreement covers a range of first-in-class ocular programs targeting serious, vision-threatening diseases based on Editas Medicines unparalleled CRISPR genome editing platform, including CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a).

Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Companys plans with respect to the Brilliance Phase 1/2 clinical trial for EDIT-101 (AGN-151587), including the Companys expectations regarding the timing of dosing a patient by early 2020. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of the Companys product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make 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 statements, whether because of new information, future events or otherwise.

Investor ContactMark Mullikin(617) 401-9083mark.mullikin@editasmed.com

Media ContactCristi Barnett(617) 401-0113cristi.barnett@editasmed.com

See more here:
Editas Medicine Announces Third Quarter 2019 Results and Update - Yahoo Finance

By Sandee LaMotte, CNN

(CNN) If certain vegetables have always made you gag, you may be more than a picky eater. Instead, you might be what scientists call a super-taster: a person with a genetic predisposition to taste food differently.

Unfortunately, being a super-taster doesnt make everything taste better. In fact, it can do the opposite.

Super-tasters are extremely sensitive to bitterness, a common characteristic of many dark green, leafy veggies such as broccoli, cauliflower, cabbage and Brussels sprouts, to name a few.

The person who has that genetic propensity gets more of the sulfur flavor of, say, Brussels sprouts, especially if theyve been overcooked, said University of Connecticut professor Valerie Duffy, an expert in the study of food taste, preference and consumption.

So that [bitter] vegetable is disliked, and because people generalize, soon all vegetables are disliked, Duffy said. If you ask people, Do you like vegetables? They dont usually say, Oh yeah, I dont like this, but I like these others. People tend to either like vegetables or not.

In fact, people with the bitter gene are2.6 times more likely to eat fewer vegetables than people who donot have that gene, according to a new study presented Monday at the annual meeting of the American Heart Association.

We wanted to know if genetics affected the ability of people who need to eat heart-healthy foods from eating them, said study author Jennifer Smith, a registered nurse who is a postdoc in cardiovascular science at the University of Kentucky School of Medicine.

While we didnt see results in gene type for sodium, sugar or saturated fat, we did see a difference in vegetables, Smith said, adding that people with the gene tasted a ruin-your-day level of bitterness.

Our sense of taste relies on much more than a gene or two. Receptors on our taste buds are primed to respond to five basic flavors: salty, sweet, sour, bitter and umami, which is a savory flavor created by an amino acid called glutamate (think of mushrooms, soy sauce, broth and aged cheeses).

But its also smelling through the mouth and the touch, texture and temperature of the food, Duffy said. Its very difficult to separate out taste from the rest. So when any of us say the food tastes good, its a composite sensation that were reacting to.

Even our saliva can enter the mix, creating unique ways to experience food.

When we come to the table, we dont perceive the food flavor or the taste of food equally, Duffy said. Some people live in a pastel food world versus others who might live in a more vibrant, neon food world. It could explain some of the differences in our food preference.

While there are more than 25 different taste receptors in our mouth, one in particular has been highly researched: the TAS2R38, which has two variants called AVI and PAV.

About 50% of us inherent one of each, and while we can taste bitter and sweet, we are not especially sensitive to bitter foods.

Another 25% of us are called non-tasters because we received two copies of AVI. Non-tasters arent at all sensitive to bitterness; in fact food might actually be perceived as a bit sweeter.

The last 25% of us have two copies of PAV, which creates the extreme sensitivity to the bitterness some plants develop to keep animals from eating them.

When it comes to bitterness in the veggie family, the worst offenders tend to be cruciferous vegetables, such as broccoli, kale, bok choy, arugula, watercress, collards and cauliflower.

Thats too bad, because they are also full of fiber, low in calories and are nutrient powerhouses. Theyre packed with vitamins A and C and whats called phytonutrients, which are compounds that may help to lower inflammation.

Rejecting cruciferous or any type of vegetable is a problem for the growing waistline and health of America.

As we age as a population, vegetables are very important for helping us maintain our weight, providing all those wonderful nutrients to help us maintain our immune system and lower inflammation to prevent cancer, heart disease and more, Duffy said.

Food scientists are trying to develop ways to reduce the bitterness in veggies, in the hopes we can keep another generation of super-tasters from rejecting vegetables.

Theres been some success. In fact, the Brussels sprouts we eat today are much sweeter than those our parents or grandparents ate. Dutch growers in the 90s searched their seed archives for older, less bitter varieties, then cross-pollinated them with todays higher yielding varieties.

People who already reject vegetables might try to use various cooking methods that can mask the bitter taste.

Just because somebody carries the two copies of the bitter gene doesnt mean that they cant enjoy vegetables, Duffy said. Cooking techniques such as adding a little fat, a little bit of sweetness, strong flavors like garlic or roasting them in the oven, which brings out natural sweetness, can all enhance the overall flavor or taste of the vegetable and block the bitterness.

The-CNN-Wire & 2019 Cable News Network, Inc., a WarnerMedia Company. All rights reserved.

Read the original here:
Attention Vegetable Haters: It Could Be In Your Genes - CBS Boston

Updated results from the Alta trial show that a single infusion with the highest dose of SB-525, an investigational gene therapy, yields dose-dependent and durable increases in clotting factor VIII (FVIII). The trial, in adults with severe hemophilia A , found no bleeding episodes up to 24 weeks following the infusion.

That highest dose of SB-525 31013 vector genomes, vg/kilogram, kg led patients to reach normal FVIII activity. Participants no longer needed replacement therapy following a short preventive course post-SB-525-administration.

With these promising results, Pfizer has initiated a lead-in study (NCT03587116) to support SB-525 advancement to a Phase 3 registrational clinical trial. The six-month study will evaluate the current efficacy and safety of preventive replacement therapy in the usual care setting. It is currently recruiting participants worldwide.

The Alta trials most recent findings will be shared at the upcoming 61st Annual Meeting of the American Society of Hematology (ASH), to be held Dec. 7-10 in Orlando, Fla.

Data will be featured in a poster titled Updated Follow-up of the Alta Study, a Phase 1/2, Open Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 Gene Therapy in Adult Patients with Severe Hemophilia A.

SB-525 is a gene therapy candidate to treat hemophilia A thats being developed by Sangamo Therapeutics in collaboration with Pfizer. It consists of a DNA sequence coding for the production of a working FVIII the clotting factor missing in hemophilia A. That FVIII is carried and delivered to liver cells, where clotting factors are produced, using a harmless adeno-associated viral (AAV) vector.

The goal of the therapy is for patients to regain the ability to continuously produce the coagulation factor, and reduce or eliminate the need for FVIII replacement therapy.

The therapys safety and effectiveness for the treatment of adults with severe hemophilia A are currently being evaluated in the open-label Phase 1/2 Alta trial (NCT03061201).

The study is testing a single infusion into the vein (intravenous) of one of four ascending doses of SB-525: 91011 vg/kg; 21012 vg/kg; 11013 vg/kg; and 31013 vg/kg. Two people have been dosed per group, except for the highest dose group, which was expanded to five patients.

Updated trial data now released show the results for the two patients dosed in the third group those given 11013 vg/kg and the five individuals receiving the highest dose of 31013 vg/kg.

In the third group, a single infusion of SB-525 resulted in stable and clinically relevant increases in FVIII activity.

Stronger results were seen with SB-525s highest dose. Of the five patients treated, data were available for four. For these participants, a single infusion with the highest dose of SB-525 led to normal FVIII levels with no bleeding events reported up to 24 weeks post-administration. These individuals no longer needed replacement therapy after the initial prophylactic period of up to about three weeks after SB-525 dosing.

In addition, preliminary tests from the high-dose group indicate similar activity of SB-525-derived FVIII and the clotting factor provided by Xyntha, Pfizers recombinant therapy for hemophilia A.

As to safety, one patient had treatment-related serious adverse events, namely low blood pressure and fever, occurring about six hours after infusion. These effects resolved with treatment within 24 hours, with no loss of FVIII expression.

Some patients also showed elevated blood levels of liver enzymes(ALT, alanine aminotransferase). However, these were reported to be mild and temporary increases, which were treated in a timely manner with corticosteroids.

Dosing in the fourth group is ongoing. At the upcoming meeting, Sangamo will disclose additional analyses of the trial data, including a follow-up of approximately 4 to 11 months after treatment.

The rapid kinetics of Factor VIII expression, durability of response, and the relatively low intra-cohort variability in the context of a complete cessation of bleeding events and elimination of exogenous Factor VIII usage continues to suggest SB-525 is a differentiated hemophilia A gene therapy, Bettina Cockroft, MD, MBA, chief medical officer of Sangamo said in a press release.

We are pleased with the progress of the program toward a registrational Phase 3 study led by Pfizer, who announced it has enrolled its first patient in the 6-month Phase 3 lead-in study. We have recently completed the manufacturing technology transfer to Pfizer and initiated the transfer of the IND [investigational new drug].

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 121

Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

See original here:
Updated Alta Trial Results Support SB-525 Gene Therapy for Hemophilia A - Hemophilia News Today

When RegenxBio disclosed that the FDA had placed a partial clinical hold on one of its lead gene therapies, execs outlined several customary next steps: continuing assessment and monitoring, delaying a related IND filing, and working with the FDA to address the matter.

As it turned out, they were planning something much less mundane. Two days after announcing the hold in its Q3 update, RegenxBio filed a lawsuit seeking to set it aside, the FDA Law Blog noted.

The lawsuit shed light on the interactions between the biotech and regulators, revealing that there was actually a full clinical hold on the diabetic retinopathy trial in addition to the partial hold on wet age-related macular degeneration and RegenxBio withdrew the IND.

According to the complaint, the company had discussed results from its Phase I/IIa trial with the FDA and was on track to begin the next phase before the end of 2019.

But on October 18, 2019, without notice or explanation, FDA placed RGX-314 on a clinical hold, effectively halting REGENXBIOs development of this potentially life-altering treatment for retinal diseases that are leading causes of adult blindness. Since issuing the clinical hold order, FDA has rebuffed REGENXBIOs repeated attempts to obtain an explanation of the basis for the clinical hold.

Failing to provide advance warning or explanation for the hold, RegenxBio claims, is in violation of the FDAs own regulations. And that led to an arbitrary and capricious final decision, they wrote.

But thats not it.

Since they didnt get a chance to review or rebut the reasons for the hold, which harmed their reputation and property interest in RGX-314, RegenxBio believes the FDA violated the Fifth Amendments Due Process Clause. Furthermore, they charged a particular section of the Food, Drug, and Cosmetic Act represented an unconstitutional vesting of legislative power in the Secretary of Health and Human Services.

Along with the agency, the federal government, HHS Secretary Alex Azar, acting FDA commissioner Brett Giroir (who has taken over from Ned Sharpless while Stephen Hahn goes through the confirmation process), and FDA regulatory project manager Edward Thompson were also named as defendants. Thompson allegedly first notified RegenxBio of the holds citing issues associated with [RGX-314s] delivery systems.

Following multiple exchanges, the FDA apparently told RegenxBio that it would provide a written explanation of the basis for the hold by this Friday, November 15.

Why risk the ire of regulators when an update is due so soon? As a procedural matter to preserve their rights, the company said.

This action was taken on the recommendation from counsel as we continue to work with the FDA to address this matter, and we hope this step will help ensure the FDA will provide RegenxBio with their specific concerns about the unspecified device, it wrote in an email to Endpoints News.

Attorney Deborah Livornese offered this take on the FDA Law Blog:

While it seems unlikely that the merits of the clinical hold will be resolved through the judiciary process in a helpful timeframe, the complaint has likely increased the chances that FDA will respond with a thorough explanation of its reason for the hold when it does provide the written basis.

The biotech added that the plan is still to start the wet AMD trial and file an IND for diabetic retinopathy in Q2 2020, as we believe that there are readily available and suitable alternatives for all of the devices used in our studies.

Originally posted here:
UPDATED: Decrying 'arbitrary and capricious' action, RegenxBio sues FDA over clinical holds on gene therapy - Endpoints News