R&D Alliance Between Harvard and Deerfield Announces First Project Agreement

Lab1636 commits to advance certain Harvard innovations toward a possible therapeutic for tactile hypersensitivity experienced in autism spectrum disorders

Cambridge, Mass., January 8, 2019 — Lab1636, the R&D alliance between Harvard University and Deerfield Management Company, announced today its first project agreement to advance Harvard researchers’ innovations toward the development of novel therapeutics. Lab1636 has committed to a first project out of the laboratory of David Ginty, PhD, the Edward R. and Anne G. Lefler Professor of Neurobiology in the Blavatnik Institute at Harvard Medical School (HMS) and a Howard Hughes Medical Institute Investigator.

Launched in March 2019, Lab1636 is a major strategic R&D alliance between Harvard and the healthcare investment firm Deerfield to speed the development and translation of biomedical and life-science innovations into transformative treatments to improve life, health, and medical care. Lab1636 was established with a funding commitment of up to $100 million from Deerfield.

Through this first project, Lab1636 will dedicate focused resources to advancing innovations with great therapeutic potential for patients. Recognized for his lab’s elucidation of the peripheral nervous system, Ginty seeks to answer fundamental questions in neuroscience relating to how we perceive and respond to our environment. Over the past few years, in particular, significant strides in understanding tactile hypersensitivity have been led by a postdoctoral researcher in his lab, Lauren Orefice, PhD, who is now Assistant Professor of Genetics at HMS and Massachusetts General Hospital. Together, they identified certain compounds that may point the way to a treatment for the touch hypersensitivity experienced by people with autism spectrum disorders.

The Ginty Laboratory’s research in this area has previously received funding from the federal government and the Simons Foundation, as well as translational research funding from Harvard’s Blavatnik Biomedical Accelerator and Q-FASTR at HMS. Lab1636 is now poised to validate and expand upon the lab’s findings, advancing them through late-stage preclinical development.

“We’re thrilled by the momentum of the Lab1636 alliance so far,” said Vivian Berlin, Managing Director of Strategic Partnerships in Harvard’s Office of Technology Development, which spearheaded the creation of Lab1636 with Deerfield. “The collaboration holds great promise to drive rapid innovation across many fields of biomedical science and translate valuable insights into real-world impact.”

“This is an important milestone for Lab1636 and potentially for people suffering from tactile hypersensitivity,” said James E. Flynn, Managing Partner at Deerfield. “We look forward to continued progress in Professor Ginty’s novel work and other exciting developments on the horizon at Harvard.”

A private company wholly owned by affiliates of Deerfield, Lab1636 supports Harvard R&D projects through various stages of drug discovery and development, for example enabling studies to explicate the biology of disease, validate therapeutic targets, or achieve a proof-of-concept necessary for filing an Investigational New Drug (IND) application. Harvard’s R&D projects funded by Lab1636 are initiated by principal investigators from labs across the University and selected by a joint advisory committee.

See also: Harvard professors David Ginty and Lauren Orefice discuss their translational research in a Q&A published today.


About Harvard Office of Technology Development
Harvard’s Office of Technology Development (OTD) promotes the public good by fostering innovation and translating new inventions made at Harvard University into useful products that are available and beneficial to society. Our integrated approach to technology development comprises sponsored research and corporate alliances, intellectual property management, and technology commercialization through venture creation and licensing. More than 70 startups have launched to commercialize Harvard technologies in the past 5 years, collectively raising more than $2.5 billion in financing. To further bridge the academic-industry development gap, Harvard OTD manages the Blavatnik Biomedical Accelerator and the Physical Sciences & Engineering Accelerator. For more information, please visit https://otd.harvard.edu.


About Deerfield Management Deerfield is a healthcare investment management firm committed to advancing healthcare through investment, information and philanthropy.

Contacts

Harvard Office of Technology Development
Caroline Perry, 617-495-4157
[email protected]

Deerfield Management Company
Karen Heidelberger, 212-551-1600
[email protected]

Broad scientists oust Alzheimer’s risk gene in a lab dish by revising DNA

Photo Courtesy of Stephen Dixon/the McGovern Institute

The CRISPR family enzyme CAS13 (pink) uses a special guide (red) to target RNAs in the cell (blue). Broad Institute scientists used a new model of CRISPR, CAS13, to try to eliminate a genetic risk for Alzheimer’s disease, they report in the journal Science.

With a newly adapted CRISPR tool, researchers out of the Broad Institute of MIT and Harvard, have stamped out an Alzheimer’s threat in cells by revising RNA, rather than permanently editing DNA.

The findings were reported in the July 11, 2019 issue of the journal Science.

Feng Zhang, PhD and colleagues illustrated the promise of the new CRISPR platform, CAS13, by deactivating the APOE4 risk gene and changing it to APOE2, the rarer variant (which is protective and may actually decrease a person’s risk for Alzheimer’s: Science). Long viewed as one of the biggest risk factors for Alzheimer’s – APOE4 is also associated with the most common form of the disease.

Because protein-coding RNA is transcribed from genomic DNA, this technique offers the potential to correct disease-causing mutations at the RNA level without the possible risks of making permanent changes to the genome. In addition, in some cell types, particularly postmitotic cells such as neurons, it is difficult to edit genomic DNA using earlier CRISPR approaches. Therefore, CAS13 represents a potential new strategy to treat devastating diseases that affect the brain, including Alzheimer’s.

The new advance, called RESCUE for RNA Editing for Specific C to U Exchange, builds on REPAIR, a technology developed earlier by Zhang and his team that changes adenine bases into inosine in RNA. The scientists took the REPAIR fusion and evolved it in the lab until it could change cytosine to uridine.“Development of RESCUE demonstrates the power of protein engineering of natural processes,” said Deerfielder Bob Jackson, MD. “The ability of RESCUE to edit from C to U increases the number of pathogenic mutations targetable by RNA editing. It also adds capacity to potentially edit important signaling residues.”

CRISPR refers to Clustered Regularly Interspaced Short Palindromic Repeats that occur in the genome of certain bacteria, from which the system was discovered. Often thought of as “molecular scissors”, the CRISPR technology enables researchers to remove, add or alter specific DNA and RNA sequences in the genome of higher organisms, with the goal of curing disease.

The Broad has been a Deerfield collaborator since 2017: Broad Institute and Deerfield Management launch innovative partnership to tackle serious unmet medical needs

Adapted from MIT news release

Neos Therapeutics

Neos Therapeutics, Inc. is a specialty pharmaceutical company with a late-stage pipeline of innovative extended release products for the treatment of ADHD. Neos has submitted an NDA for its methylphenidate XR-ODT drug candidate for ADHD medication based on its patented XR-ODT technology. Neos’ XR-ODT and XR-Liquid product candidates provide patient-friendly dosage forms for patients who have difficulty swallowing tablets or capsules and can benefit from extended- release formulations.

Aprecia

Aprecia is a specialty pharmaceutical company using its proprietary 3D printing technology to develop and manufacture pharmaceuticals. The company’s ZipDose formulation platform enables rapid dissolving forms of drugs that are beyond the formulation limits of existing fast-melt technologies. The company obtained FDA approval in 2015 for its lead product Spritam, an oral fast-melt form of levetiracetam (Keppra) for epilepsy, and is developing novel formulations of several other epilepsy drugs.

Forecasting the prevalence of status epilepticus and ist subtypes in Europe, 2015-2024

Objectives

To estimate the prevalence of status epilepticus (SE), refractory status epilepticus (RSE), and super-refractory status epilepticus (SRSE) in five major European Union (5EU) markets (France, Germany, Italy, Spain, and the UnitedKingdom) using an incidence-survival model.

Methods

Yearly survival data for each SE etiology (acute symptomatic, progressive symptomatic, remote symptomatic, and idiopathic/cryptogenic) were extracted from published research. Incident cases were calculated for each etiology beginning with 1995, based on market-specific published rates. Applying the survival proportions and incidence estimates to the model for each etiology, we calculated an overall estimate of the prevalence of SE. RSE and SRSE prevalent cases were assessed as proportions of the total number of prevalent SE cases using published values.

Results

We estimated the prevalence of SE to be 18.4 cases per 10,000 population in the5EU, resulting in 590,264 cases in 2015 and increasing to 603,951 in 2024. The calculated prevalence ranged from 17.2 cases per 10,000 (Germany) to 19.7 cases per 10,000 (Italy). The prevalence of RSE in the 5EU was 4.5 per 10,000, resulting in 145,205 cases in 2015, increasing to 148,572 in 2024. SRSE prevalence in the 5EU was 1.8 per 10,000, resulting in 59,027 cases in 2015, increasing to 60,395 in 2024.

Conclusions

To our knowledge, this is the first attempt to calculate the prevalence of SE and its subtypes for all ages in Europe. Estimating the prevalence of SE, RSE, and SRSE using population-based epidemiological methods is challenging because of the variability of SE disease definitions and the unpredictable nature of mortality due to SE. Our incidence-survival model provides an alternative and effective method to assess the prevalent population. Considering the high costs associated with treatment and hospitalization of SE, RSE, and SRSE patients, these estimates are necessary to quantify the burden of disease in Europe.

Deerfield Finances Pioneer in 3D Printing for Pharmaceuticals

New York, New York – January 27, 2015 – Deerfield Management Company announced today that it invested $30 million to lead a $35 million financing in Aprecia Pharmaceuticals. Proceeds from this financing will allow Aprecia to launch SPRITAM®, the world’s first 3D printed medicine approved by the U.S. Food and Drug Administration, as well as accelerate the development of additional 3D printed drug formulations utilizing Aprecia’s novel 3DP technology platform.

Aprecia is a specialty pharmaceutical company using its proprietary 3D printing technology platform to develop and manufacture pharmaceuticals. The company’s ZipDose formulation platform enables rapid dissolving forms of drugs that may exceed the formulation limits of existing fast-melt technologies. SPRITAM® is an oral fast-melt form of levetiracetam for epilepsy.

“Based on extensive market research conducted by the Deerfield Institute, we believe there is a substantial population of epilepsy patients, particularly children, who are not well served by existing formulations and will benefit from SPRITAM® and the other epilepsy drugs in Aprecia’s pipeline,” stated Jonathan Leff, Partner at Deerfield. “We are very pleased to join with the Aprecia team to advance the commercialization of important new therapies based on the company’s unique 3D printing technology.”

“We are excited to have Deerfield as our partner in the launch of SPRITAM®. Deerfield has demonstrated tremendous knowledge of the field as well as creativity and flexibility in structuring the financing. With its desire to advance healthcare and address unmet patient needs, we believe Deerfield is an ideal partner for Aprecia,” stated Don Wetherhold, CEO of Aprecia.

About Aprecia Pharmaceuticals

Aprecia is an emerging pharmaceutical company that expects to use its proprietary ZipDose Technology to transform the way people take medicine. Aprecia believes it is the first and only company in the world to utilize three-dimensional printing (3DP) technology to develop and manufacture pharmaceutical products on a commercial scale. Aprecia plans to introduce multiple highly prescribed, high dose medications utilizing ZipDose Technology in the coming years. The company’s initial focus is on the central nervous system therapeutic area where there is a need for medicines that are easy to take. Aprecia’s mission is to improve real-world outcomes by transforming the way medicine is experienced. The company is privately owned, with affiliates of Prasco, LLC and the Arington family holding a controlling interest.

For more information visit www.aprecia.com.

About Deerfield

Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy.

For more information, please visit www.deerfield.com

Contacts

Deerfield Management Company
Karen Heidelberger, 212-692-7140
[email protected]