On February 15, 2022 Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial stage company engaged in the discovery, development and sale of drugs to treat severe metabolic, oncologic and neuropsychiatric disorders by modulating the effects of the hormone cortisol, reported its results for the quarter and year ended December 31, 2021 (Press release, Corcept Therapeutics, FEB 15, 2022, View Source [SID1234608128]).

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Financial Results
•Fourth quarter revenue of $98.8 million, compared to $85.7 million in fourth quarter 2020
•2021 revenue of $366.0 million, compared to $353.9 million in 2020
•2022 revenue guidance of $400 – $430 million
•Fourth quarter GAAP net income of $32.1 million, compared to $26.0 million in fourth quarter 2020
•2021 GAAP net income of $112.5 million, compared to $106.0 million in 2020
•Purchase of ten million shares of Corcept common stock for $207.5 million in fourth quarter 2021
•Cash and investments of $335.8 million
"Korlym is an excellent treatment for Cushing’s syndrome and there are many eligible patients who have yet to receive it. As pandemic restrictions and fears recede, as they already have in certain locations, we expect our growth to continue and are providing 2022 revenue guidance of $400 – $430 million," said Joseph K. Belanoff, MD, Corcept’s Chief Executive Officer.
Corcept’s fourth quarter 2021 revenue was $98.8 million, compared to $85.7 million in the fourth quarter of 2020. 2021 revenue was $366.0 million, compared to $353.9 million in 2020. The company expects 2022 revenue of $400 – $430 million.
GAAP net income was $32.1 million in the fourth quarter of 2021, compared to $26.0 million in the fourth quarter of 2020. For the full year, it was $112.5 million in 2021, compared to $106.0 million in 2020.
Excluding non-cash expenses related to stock-based compensation and the utilization of deferred tax assets, together with related income tax effects, non-GAAP net income in the fourth quarter was $42.6 million, compared to $34.7 million in the fourth quarter of 2020. For the full-year, non-GAAP net income was $149.5 million, compared to $145.6 million in 2020. A reconciliation of GAAP to non-GAAP net income is included below.
Cash and investments of $335.8 million at December 31, 2021 reflects the purchase of ten million shares of Corcept common stock for $207.5 million in the fourth quarter 2021.
Clinical Development
"In addition to successfully managing challenges posed by the pandemic for our commercial business, we significantly advanced our clinical development programs in 2021," said Dr. Belanoff. "Cortisol modulation has the potential to treat many serious diseases. Data generated by our ovarian cancer and non-alcoholic steatohepatitis (NASH) programs last year provided evidence of cortisol modulation’s broad application. In 2022, we will see important results from many of our ongoing clinical programs. We also continue to advance new cortisol modulators to the clinic. Of particular note, we plan to start a Phase 2 trial in patients with amyotrophic lateral sclerosis (ALS) in the second quarter."

Solid Tumors
•Updated overall survival data from Phase 2 trial in patients with recurrent platinum-resistant ovarian cancer expected in first quarter 2022; Phase 3 trial planned to start second quarter 2022
•Selection of the optimum dose of exicorilant or relacorilant plus enzalutamide in patients with castration-resistant prostate cancer (CRPC) expected in second quarter 2022
•Enrollment continues in 20-patient, open-label, Phase 1b trial of relacorilant plus PD-1 checkpoint inhibitor pembrolizumab in patients with adrenal cancer with cortisol excess
"Our 178-patient, randomized, controlled Phase 2 trial of relacorilant in patients with recurrent platinum-resistant ovarian cancer met its objective of improving progression free survival, without increased side effect burden," said Bill Guyer, PharmD, Corcept’s Chief Development Officer. "We and our investigators are excited to advance relacorilant for the potential treatment of ovarian cancer based on these statistically significant and clinically meaningful results. We expect the trial’s updated overall survival data later this quarter and plan to meet with the FDA in the coming months to define the best path forward."
Metabolic Diseases
•Enrollment completed in GRATITUDE II, a 150-patient, double-blind, placebo-controlled Phase 2 trial of miricorilant to reverse long-standing antipsychotic-induced weight gain (AIWG); data expected in fourth quarter 2022
•Completion of enrollment in GRATITUDE, a double-blind, placebo-controlled Phase 2 trial of miricorilant to reverse recent AIWG, expected by mid-2022; data expected in fourth quarter 2022
•Enrollment continues in Phase 1b dose-finding trial of miricorilant in patients with presumed NASH
"We are pleased to have fully-enrolled GRATITUDE II and to be nearing completion of enrollment in GRATITUDE. AIWG is a serious metabolic disorder suffered by millions of patients in the United States, with few treatment options," said Dr. Guyer. "We initiated these double-blind, placebo-controlled, Phase 2 trials to build on the positive data from our studies of both miricorilant and mifepristone in healthy volunteers. We look forward to the data readout from both trials, which we expect in the fourth quarter."
"We and our investigators are very excited by the unprecedented rapidity and magnitude of liver fat reduction experienced by patients taking miricorilant in our previous NASH study," added Dr. Guyer. "The goal of our Phase 1b dose-finding study is to identify a dosing regimen that significantly reduces fat without causing excessive liver irritation."
Cushing’s Syndrome
•Enrollment continues in Phase 3 GRACE trial of relacorilant as a treatment for patients with all etiologies of Cushing’s syndrome; new drug application (NDA) submission expected in second quarter 2023
•Enrollment continues in Phase 3 GRADIENT trial of relacorilant as a treatment for patients with Cushing’s syndrome caused by adrenal adenomas
"Our Phase 3 GRACE and GRADIENT trials continue to accrue patients and generate data. We expect GRACE to serve as the basis for relacorilant’s NDA in Cushing’s syndrome, and we are on track to make this submission in the second quarter of 2023," said Dr. Guyer. "GRADIENT is on track to produce valuable data about an etiology of Cushing’s syndrome that affects many patients, but has not been subject to rigorous, controlled study."
Conference Call
We will hold a conference call on February 15, 2022, at 5:00 p.m. Eastern Time (2:00 p.m. Pacific Time). To participate, click this link (listen-only mode) or dial 1-833-693-0540 from the United States or 1-661-407-1581

internationally approximately 15 minutes before the start of the call. A replay will be available through February 22, 2022 at 1-855-859-2056 from the United States and 1-404-537-3406 internationally. The passcode will be 1861918. A replay will also be available on the Investors / Past Events tab of our website.
Hypercortisolism
Hypercortisolism, often referred to as Cushing’s syndrome, is caused by excessive activity of the hormone cortisol. Endogenous Cushing’s syndrome is an orphan disease that most often affects adults aged 20-50. In the United States, an estimated 20,000 patients have Cushing’s syndrome, with about 3,000 new patients diagnosed each year. Symptoms vary, but most patients experience one or more of the following manifestations: high blood sugar, diabetes, high blood pressure, upper-body obesity, rounded face, increased fat around the neck, thinning arms and legs, severe fatigue and weak muscles. Irritability, anxiety, cognitive disturbances and depression are also common. Hypercortisolism can affect every organ system and can be lethal if not treated effectively. Corcept holds patents directed to the composition of relacorilant and the use of cortisol modulators, including Korlym, in the treatment of patients with hypercortisolism.

On February 15, 2022 HOOKIPA Pharma Inc. (NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, reported it has entered into an amended and restated collaboration and license agreement with Gilead to advance the development of a novel arenaviral immunotherapy as a component of a potential functional curative regimen for human immunodeficiency virus (HIV) (Press release, Hookipa Biotech, FEB 15, 2022, View Source [SID1234608127]).

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In April 2018, Gilead licensed exclusive rights to HOOKIPA’s versatile arenaviral platform to develop immunotherapies for HIV and hepatitis B virus (HBV). Under those terms, the companies agreed to collaborate through a joint research phase, after which time Gilead had rights for further development. Under the amended and restated agreement, HOOKIPA is responsible for advancing the HIV program through the completion of a Phase 1b clinical trial, with funding from Gilead via an upfront payment and equity purchases. At the completion of the Phase 1b trial, Gilead has the exclusive right to assume further development of the program. The HBV portion of the agreement remains unchanged.

"We are pleased to enter into this amended agreement with Gilead which includes provisions that we believe benefit both parties, and we hope ultimately the HIV community," said Joern Aldag, Chief Executive Officer at HOOKIPA. "Gilead is helping to advance our novel arenaviral platform technology, which has the potential to complement Gilead’s overall research strategies for cures of HIV and HBV."

Hookipa earned a one-time $4 million preclinical milestone payment under the original 2018 collaboration and license agreement. Upon signing of the amended agreement, Hookipa will receive a payment of $15 million. In addition, Gilead will make a $5 million equity investment in HOOKIPA at a premium to the current market price, and up to an additional $30 million of equity financing that can be drawn at HOOKIPA’s discretion by December 31, 2023.

HOOKIPA’s research collaboration with Gilead to develop a potential functional cure for Hepatitis B virus (HBV) continues to move forward under the original agreement terms. The project progressed successfully into preclinical development, and Gilead plans to advance the program to the IND-enabling stage in 2022, supporting clinical entry of an alternating two-vector arenaviral therapeutic for the treatment of HBV.

For further details on the amended agreement, refer to our Current Report on Form 8-K filed with the Securities and Exchange Commission on February 15, 2022.

On February 15, 2022 Enveric Biosciences (NASDAQ: ENVB) ("Enveric" or the "Company"), a neuroscience company developing next-generation, psychedelic-inspired mental health and oncology treatments, reported the closing of its previously announced underwritten public offering of 20,000,000 shares of its common stock and warrants to purchase up to 20,000,000 shares of its common stock for gross proceeds of approximately $10 million, before deducting underwriting discounts and commissions and other offering expenses (Press release, Enveric Biosciences, FEB 15, 2022, View Source [SID1234608126]). A.G.P./Alliance Global Partners acted as sole book-running manager for the offering. In addition, Enveric granted the underwriter a 45-day option to purchase up to an additional 3,000,000 shares of common stock and/or warrants to purchase up to an additional 3,000,000 shares of common stock at the public offering price, which the underwriter has partially exercised for warrants to purchase up to 3,000,000 shares of common stock.

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All the securities being sold in the offering were offered by Enveric. At closing, Enveric received net proceeds from the offering of approximately $9.2 million, after deducting underwriting discounts and commissions and estimated offering expenses. The Company intends to use the net proceeds from this offering for working capital and to fund other general corporate purposes.

This offering was made pursuant to an effective shelf registration statement on Form S-3 (No. 333-257690) previously filed with the U.S. Securities and Exchange Commission (the "SEC") that was declared effective by the SEC on July 9, 2021, and to a prospectus supplement and accompanying prospectus. The final prospectus supplement and accompanying prospectus relating to the offering were filed with the SEC and are available on the SEC’s website located at View Source Electronic copies of the final prospectus supplement may be also obtained from A.G.P./Alliance Global Partners, 590 Madison Avenue, 28th Floor, New York, NY 10022 or via telephone at 212-624-2060 or email: [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

On February 15, 2022 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the fourth quarter and full year ended December 31, 2021 (Press release, CRISPR Therapeutics, FEB 15, 2022, View Source [SID1234608125]).

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"2021 was a productive year for CRISPR Therapeutics on all fronts. We and our partner Vertex completed enrollment in the CTX001 clinical trials and are on track for planned regulatory submissions in late 2022," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Concurrently, we advanced our wholly-owned immuno-oncology pipeline, and have initiated dosing of patients in our CTX110 pivotal trial. Further, we and our partner ViaCyte, recently announced the dosing of the first patient in the Phase 1 clinical trial of VCTX210 for the treatment of type 1 diabetes. This investigational therapy has the potential to be a transformative treatment for patients with all insulin-requiring forms of diabetes. Looking ahead, we expect a productive 2022 with continued progress across our portfolio as we enter a new phase of growth for our Company. Given the breadth of our portfolio and our strong capital position, we remain well positioned to advance our pipeline and platform to develop transformative medicines for patients suffering from serious diseases."

Recent Highlights and Outlook

Beta Thalassemia and Sickle Cell Disease Programs

More than 70 patients have been dosed with CTX001 across both trials to date. Target enrollment has been achieved in the ongoing clinical trials for CTX001 in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), with planned regulatory submissions in late 2022.
In June 2021, data from 22 patients with at least three months of follow-up after CTX001 infusion were presented at the Annual European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress (EHA) (Free EHA Whitepaper) and continued to build the profile of a potentially functional cure for patients with TDT and SCD, showing consistent and durable benefit with longer term data from a larger population of patients.

In April 2021, CRISPR Therapeutics and Vertex announced an amendment to their collaboration for CTX001. In connection with the completion of the transaction in June, Vertex made a $900 million upfront payment to CRISPR Therapeutics.

In April 2021, CRISPR Therapeutics and Vertex announced that the European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to CTX001 for the treatment of TDT. CTX001 was granted PRIME designation for the treatment of SCD in 2020.

Immuno-Oncology Programs

CRISPR Therapeutics has begun dosing patients in the pivotal trial of CTX110, its wholly-owned allogeneic chimeric antigen receptor T cell (CAR-T) investigational therapy targeting CD19+ B-cell malignancies. Enrollment is ongoing and the Company expects to report additional data in 2022. The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX110 in November 2021.

In October 2021, CRISPR Therapeutics announced positive results from its ongoing Phase 1 CARBON trial evaluating the safety and efficacy of CTX110. The data showed early evidence of a dose dependent response to CTX110, with overall response rates (ORR), complete response rates (CR) and durability similar to approved autologous CD19 CAR-T therapies on an intent-to-treat (ITT) basis. A single dose of CTX110 at DL2 and above resulted in a 58% ORR and 38% CR rate in large B-cell lymphoma (LBCL) patients on an ITT basis. The pharmacokinetic data provide a strong rationale that consolidation dosing can improve on an already competitive profile for CTX110. Based on the safety and efficacy profile, the Company has expanded the current trial into a pivotal trial that incorporates consolidation dosing and has begun dosing patients in this pivotal arm. The Company expects to report additional data in 2022.

In addition to CTX110, CRISPR Therapeutics has ongoing Phase 1 clinical trials assessing safety and efficacy of several dose levels for the following CAR-Ts: (i) CTX120, its wholly-owned allogeneic CAR-T investigational therapy targeting B-cell maturation antigen for the treatment of relapsed or refractory multiple myeloma; and (ii) CTX130, its wholly-owned allogeneic CAR-T investigational therapy targeting CD70 for the treatment of both solid tumors and certain hematologic malignancies. The Company expects to report top-line data in the first half of 2022.

In May 2021, CRISPR Therapeutics and Nkarta, Inc. announced a strategic partnership to research, develop, and commercialize CRISPR/Cas9 gene-edited cell therapies for cancer. Under the agreement, the companies will co-develop and co-commercialize two CAR-NK cell product candidates, one targeting the CD70 tumor antigen and the other target to be determined. In addition, the companies will bring together their complementary cell therapy engineering and manufacturing capabilities to advance the development of a novel NK+T product candidate harnessing the synergies of the adaptive and innate immune systems.

Regenerative Medicine and In Vivo Programs

Earlier this month, CRISPR Therapeutics and its partner ViaCyte announced the first patient had been dosed in the Phase 1 clinical trial of VCTX210 for the treatment of T1D. VCTX210 is an investigational, allogeneic, gene-edited, stem cell-derived product developed in collaboration by applying CRISPR Therapeutics’ gene-editing technology to ViaCyte’s proprietary stem cell capabilities for the generation of pancreatic cells designed to evade recognition by the immune system. This immune-evasive cell replacement therapy is designed to enable patients to produce their own insulin.

In June 2021, CRISPR Therapeutics and Capsida Biotherapeutics, Inc. announced a strategic partnership to research, develop, manufacture and commercialize in vivo gene editing therapies delivered with engineered AAV vectors for the treatment of familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia. Under the agreement, CRISPR Therapeutics will lead research and development of the Friedreich’s ataxia program and perform gene-editing activities for both programs, and Capsida will lead research and development of the ALS program and conduct capsid engineering for both programs.

The Company continues to make progress with its in vivo approaches for liver gene editing utilizing both viral and non-viral delivery vehicles. The Company expects to move multiple programs utilizing in vivo approaches into the clinic in the next 18 to 24 months.

Other Corporate Matters

Under the June 2019 collaboration agreement with Vertex to discover and develop gene editing therapies for the treatment of Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1), CRISPR Therapeutics received a payment of $12.5 million from Vertex related to the achievement of a research milestone in the DM1 program. CRISPR Therapeutics is eligible to receive additional milestone payments from Vertex of up to $775 million for these two programs.
Fourth Quarter and Full Year 2021 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $2,379.1 million as of December 31, 2021, compared to $1,690.3 million as of December 31, 2020. The increase in our cash position of $688.8 million was primarily driven by an upfront payment of $900.0 million in connection with the Amended and Restated Joint Development and Commercialization Agreement with Vertex. Additionally, cash provided by financing activities was $250.9 million, primarily related to common shares issued in connection with utilizing the Company’s at-the-market (ATM) financing facility as well as option exercises. These increases were offset by continuing operating expenses to support ongoing research and development of the Company’s clinical and pre-clinical programs as well as capital investments in our manufacturing facility.
Revenue: Total collaboration revenue was $12.3 million for the fourth quarter of 2021 compared to $0.2 million for fourth quarter of 2020, and $913.1 million for the year ended December 31, 2021, compared to $0.5 million for the year ended December 31, 2020. The increase in collaboration revenue is primarily attributable to revenue recognized in connection with the aforementioned payments received from Vertex.
R&D Expenses: R&D expenses were $134.5 million for the fourth quarter of 2021 compared to $82.4 million for the fourth quarter of 2020, and $438.6 million for the year ended December 31, 2021, compared to $266.9 million for the year ended December 31, 2020. The increase in expense for the year was driven by development activities supporting the advancement of the hemoglobinopathies program and wholly-owned immuno-oncology programs, as well as increased headcount and supporting facilities-related expenses.
G&A Expenses: General and administrative expenses were $24.1 million for the fourth quarter of 2021 compared to $25.8 million for the fourth quarter of 2020, and $102.8 million for the year ended December 31, 2021, compared to $88.2 million for the year ended December 31, 2020. The increase in general and administrative expenses for the year was driven by headcount-related expense.
Net Income / Loss: Net loss was $141.2 million for the fourth quarter of 2021 compared to a net loss of $107.0 million for the fourth quarter of 2020, and net income was $377.7 million for the year ended December 31, 2021, compared to a net loss of $348.9 million for the year ended December 31, 2020.
About CTX001
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate or eliminate transfusion requirements for patients with TDT and reduce or eliminate painful and debilitating sickle crises for patients with SCD. Earlier results from these ongoing trials were published as a Brief Report in The New England Journal of Medicine in January of 2021.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About the CRISPR-Vertex Collaboration
Vertex and CRISPR Therapeutics entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics.

About CLIMB-111

The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121
The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-131
This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.

About CTX110
CTX110, a wholly owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 19, or CD19. CTX110 is being investigated in the ongoing CARBON trial.

About CARBON
The ongoing Phase 1 single-arm, multi-center, open label clinical trial, CARBON, is designed to assess the safety and efficacy of several dose levels of CTX110 for the treatment of relapsed or refractory B-cell malignancies.

About CTX120
CTX120, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting B-cell maturation antigen, or BCMA. CTX120 is being investigated in an ongoing Phase 1 single-arm, multi-center, open-label clinical trial designed to assess the safety and efficacy of several dose levels of CTX120 for the treatment of relapsed or refractory multiple myeloma. CTX120 has been granted Orphan Drug designation from the FDA.

About CTX130
CTX130, a wholly-owned program of CRISPR Therapeutics, is a healthy donor-derived gene-edited allogeneic CAR-T investigational therapy targeting cluster of differentiation 70, or CD70, an antigen expressed on various solid tumors and hematologic malignancies. CTX130 is being developed for the treatment of both solid tumors, such as renal cell carcinoma, and T-cell and B-cell hematologic malignancies. CTX130 is being investigated in two ongoing independent Phase 1, single-arm, multi-center, open-label clinical trials that are designed to assess the safety and efficacy of several dose levels of CTX130 for the treatment of relapsed or refractory renal cell carcinoma and various subtypes of lymphoma, respectively.

About VCTX210
VCTX210 is an investigational, allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of T1D. VCTX210 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and ViaCyte, Inc.

On February 15, 2022 LUMICKS, a leading life science tools company that develops equipment for dynamic single-molecule and cell avidity analysis, reported that its innovative C-Trap technology was utilized in important new research designed to understand the function of a key protein involved in double-stranded break (DSB) repair (Press release, LUMICKS, FEB 15, 2022, View Source;utm_medium=rss&utm_campaign=simon-boulton-nature-paper-dna-repair [SID1234608124]). DSB repair is fundamental in maintaining the genome integrity and when compromised can lead to cancer predisposition.

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The research, published in Nature (December 22, 2021), focused on protein HELQ, and was titled "HELQ is a dual-function DSB repair enzyme modulated by RPA and RAD51". It was authored by Simon J. Boulton’s team from The Francis Crick Institute in London, Simon Powell’s team from Memorial Sloan Kettering and Weill-Cornell Medical in New York, and David Rueda’s group from Imperial College London and London Institute of Medical Sciences.

In their paper, the researchers disentangled the diversity of molecular mechanisms of HELQ. This revealed that HELQ is involved in many different branches of the DSB repair process and demonstrated how RPA and RAD51 differentially regulate its activity throughout the DNA repair process.

According to Dr. Boulton, "We show that HELQ possesses helicase activity and a previously unappreciated DNA strand annealing function are differentially regulated by RPA and RAD51. Using biochemistry analyses and dynamic single-molecule imaging, we established that RAD51 forms a complex with and strongly stimulates HELQ as it translocates during DNA unwinding. Conversely, RPA facilitates the annealing of complementary DNA strands."

In addition, the team noted, "Our study implicates HELQ in several distinct DSB repair pathways, including HR, SSA and MMEJ, casting light on its role in genome stability and tumor avoidance. These insights help us explain why HELQ loss confers genome instability, infertility and cancer predisposition."

Dr. Boulton added, "This paper is the culmination of nearly a decade’s work trying to understand how HELQ functions in DNA repair."

Key to this understanding was the visualization of HELQ-induced unwinding through LUMICKS’ C-Trap technology with Bluelake operating software, while the python package Pylake was used to analyze the data and reveal the unique insights of dynamic single-molecule experiments.

"Understanding this protein’s function – how it interacts with other proteins and its role in mediating DSB repair – can ultimately help us in understanding mechanisms of genome stability and cancer avoidance," said LUMICKS Chief Scientific Officer, Dr. Andrea Candelli. "Ultimately, studies such as these can offer new and unexplored therapeutic strategies to target DSB vulnerabilities in cancer. We are very pleased that our innovative software played a crucial role in both collecting and analyzing these detailed experiments, conducted at the single-molecule level on HELQ."